Know the Basics Understanding Clinical Trials

hello everyone my name is James Testaverde and I am the senior director of patient services at the Crohn's and Colitis of America welcome and thank you all for attending tonight's webcast know the basics understanding clinical trials offered in partnership with research match research mass org in an online matching service that facilitates customized matching of participants to medical research studies after the presentation we will open up the program for your questions I now have the pleasure of introducing our presenters for today's program Katherine Gregor has ten years of clinical research experience having worked in the multitude of clinical settings and has published and consulted on clinical research operations in both private and academic practice nationwide she had frontline clinical experience having worked as a clinical coordinator on a number of industry sponsored and investigator-initiated trials throughout her tenure Katherine currently works as a project manager for the Randall Institute for clinical and translational research her primary areas of responsibility are a service project lead for research match a disease neutral online volunteer researcher matching service funded by the National Center for advancement of translational science Katherine has an MBA from Belmont University and is certified by both the Society of Clinical Research Associates and the Association of clinical research professionals leslie boone as a project manager with CTSA consortium coordinating center and coordinates the postdoctoral fellows for the Meharry vanderbilt community engaged research program within the institute for medicine and public health at Vanderbilt University Medical Center with in her role as project manager she works with research match to develop a framework for research dissemination ms students academic and personal interests are in health services research developing community initiatives and behavioral research with a focus on sexually transmitted diseases and informed decision making miss Newman has a Master of Public Health degree from Emory University I would now like to turn the program over to Katherine thank you James so just to give you a quick overview of today's objectives we're going to discuss the role of chemical trials in advancing research review the methods and process of a trial including the different phases of a study review common features of trials for inflammatory bowel diseases such as Crohn's and alternative colitis and discuss resources that are available to you where you can find out more information about the things you hear today next slide so I wanted to start this talk by answering the question what is the clinical trial I think a clinical trial is something that we hear a lot about in the news and on TV but sometimes people don't really know what that means so the word to get at the root of what a clinical trial is it's a very simple answer and then it's a research trial carefully designed to answer an important medical question specifically we're looking at how new drugs or combinations of drugs can affect outcomes and patients like yourself if they can prevent or possibly hero' diseases we're looking at new procedures or devices specifically different types of devices to diagnose or treat different types of diseases and we're also looking at new ways of using existing treatments so drugs that were previously approved for one type of therapy might actually have an implication in a different patient population if we look at them in a different capacity will also look at types of care such as improving the quality of life for people with chronic illnesses so what can we do to reduce the amount of daily pain lack of sleep disruption to your daily schedule can we alter the way that we treat diseases with medication or with therapies outside of medicines such as alternative medicine or exercise program and see if that improves the quality of life overall and results in a better day to day existence for people living with chronic diseases sometimes you might hear these clinical trials referred to as an interventional study or a clinical study they're really the same thing and ultimately what we're trying to do is figure out what's safe and what's effective in the population at large next slide so I don't know if any of you noticed but they did a live study about link to clinical trials and how long it takes for a drug to get approved to market we hear a lot in newspapers about new disco every day at research centers like Vanderbilt and Harvard pharmaceutical companies coming up with new molecules that they would like to test that will ultimately become the drug and people wonder why it takes so long from the time you first hear about this drug to the time that you're actually able to get it from your provider and evidence is shown that it takes on average 13 to 20 years from the time something is discovered in a lab to the time that it's actually available for you and your patient care so that you can get it from your doctor so ideally if we looked at something which that was discovered today in our lab here at Vanderbilt it would not be available to you until 2032 also really long time so we were wondering what why is the timeline so long what are the holdups and what can we do to make this timeline shorter next slide so the first thing we can do is take a look at why it takes so long to get to market this infographic that you see here is something that I found a while back and it is just astounding if you think of the fact that it starts off with 10,000 different compounds can come out of a lab at any time in a compound is something as simple as a new molecule and from 10,000 of those 250 of them will actually get into preclinical studies which means they're looking at them in animals to determine what kind of safety profile they have whether or not they show any efficacy at all and from those 250 maybe five of them will actually make it into clinical trials where we're testing them in humans and from that only one of those five will ultimately result in a approved therapy that will be available to you through your doctor so from ten thousand to one that's the odds of making it and it seems like it's pretty daunting and a lot of factors play into that and some of that is that compounds proved not to be as effective as so you thought they were when we actually get them in to animals or into humans another thing is the cost is amount to manufacture the drug that the cost doesn't warrant that continued research and then the other part is that really is it that somebody else has the has a has trouble recruiting I'm sorry minor setter that we can't get patients to participate in clinical trials which is one of the biggest stumbling blocks that we're going to talk to you about here today next slide so I'm going to talk about recruitment and it hath about patience studies from this group called C script which is the Center for information and study on clinical research participation they did a large sampling of a lot of people who have participated in clinical research or who have thought about participating in clinical research as well as the number of stats with actual pharmaceutical companies and some of the effects that they've seen when they actually try to run clinical trials and their stats show that 50% of clinical research sites enroll one or no patients in their studies so that means that a sponsor may have as many as 300 sites so 300 different doctors offices are participating in this clinical study and of there's 300 a hundred and fifty of them may never get a patient to volunteer so why is that another stat that's pretty interesting is that 80% of total trials are delayed at least one month because of unfulfilled enrollment so when a sponsor like Pfizer sets out to conduct a clinical trial they have a target inmate in mind meaning that they want to try and get all of their patients to volunteer and participate by the end of 2017 and if they can't make that deadline that means that it's going to take longer to get them enrolled it's going to cost more money in their late to market which in their mind is money so 80% of these trials take longer than targeted to target of time so think about if that was an airline and 80% of your flights were delayed you would be a hard press to continue to fight on to get more trials through your pipeline each say a drug is delayed two markets want to lose up to eight million dollars for the company so if we can actually get more people involved and help increase the number of people who participate and decrease some of those delays in enrollment we can help get more drugs to market faster next slide so what if we could take the whole process down to five years what if we can go from 2015 to 2020 that's a lot better right well there's a number of people interested in solving this problem and getting it down to five years you see evades one of those groups research matches one of those groups there a number of people have come together to try and think creatively about how we can decrease the amount of time it takes the drug to get from the bench in the lab to the bedside to the patient where they can ultimately make the biggest impact in somebody's life and so one of the ways we thought about that is by talking to people about clinical trials and helping them understand some of the issues that surround them and make them seem more approachable to the average patient and that's part of the emphasis behind why we're talking to you here today next slide so right now I'm actually going to hand this over to my colleague miss Boone Leslie is actually participated in the clinical trial I think you heard James introduces those I've done a lot in terms of talking to patients about simple trials but I have never actually had the honor of participating in one so I wanted to give participation to somebody who actually has participated so on the screen before you there is a poll that we'd like you all to participate in but I'd like to tell you a little bit about my experience and participating in a clinical trial back in the late 90s or the young college students I probably didn't know much about clinical trials but thought it would be an option for me at the time because I did have a medical condition that I was interested in learning more about one thing that I think interested me in the clinical trials that I had a trusting relationship with my physician who knew what I was struggling with and suggested that the trial could be an option for me now mind you it wasn't I didn't have a life-threatening issue but it was something of concern and so this was an option for me to participate and I learned a lot about myself and also about my relationship with my doctor because I learned a lot more information than we had ever shared during a doctor's visit because she had to document so many things for the trials so right now I'll give you a moment to participate in the poll and we want to know have you ever participated or are you currently participating in a clinical trial Wow it seems that 95% of you all who are on the line currently have not participated in a clinical trial so that's very interesting and maybe some of you are caregivers maybe some of you have been thinking about participating in a clinical trial but have not had an interest as of yet or just maybe no one has asked you so we do have a follow-up question for those of you answered no and we'd like to know if you answered no which 95% of you did what would you say is the main reason and I'd like to point out Lesley that actually 95 percent of the population not participating in research is to actually write on part of the national average mm-hmm and in research match we are trying to increase those numbers and we certainly don't think that everyone has to participate in a trial but it could be an option for some and may be an option that has not been considered so we have the results of the poll so 22% of you said not in my study area 11% said you didn't meet criteria 22% of you said you were afraid of the risks involved 17% said that you don't feel knowledgeable and 28% gave another reason and I think part of the reason why we asked this question is because those groups the answers that are up here are fairly common not understanding or feeling comfortable with what's going on in the clinical trial is a very common feeling among patients so we talked to um not being sure of the risk involved and understanding how that's calculated it's also very common concern so hopefully we can give you some tools to address those right and for those of you answers other I know from looking at the literature and from surveying our own population here at research match we know that sometimes transportation can be an issue sometimes it's the time it takes to participate that may also be a barrier for your to participation as well as day-to-day things like childcare taking off from work there are numerous reasons why you may have chosen other at this time but we hope that as we work better with our physicians and doctors offices and other sponsors of research that we can help them understand what their what barriers do exist that help them to create more trials that meet the needs for your lives next slide so why don't people participate I think we've we've listed some of the things that you have identified and I think I just said you know sometimes as transportation you know there if you're lots of day to day things but sometimes it's just lack of opportunities no one has asked you and sometimes it's a lack of knowledge and as you indicated through the poll that you don't know what you're eligible for some healthy volunteers can participate in clinical trials just as well as those who may be dealing with the condition and also lack of comfort if you have not had a lot of experience with the medical professions and you may not be comfortable in a medical setting thinking about what's going to happen to you but the great thing about participation is that if they're really good they will walk you through all of these barriers and help you to feel more comfortable so that you can participate and learn more about yourself in whatever condition that we're trying to solve and ultimately it's important to emphasize that you as the volunteer are crucial to the context of research that was something that I was trying to relay when I talked about some of the barriers to enrollment without people to participate there can be no discovery so really it is the job of the physician and the recruiter who's talking to you to help you feel comfortable and to help you understand what's about to happen in a clinical trial any question you asked should be answered and it should be a message to the extent that you're satisfied so never be afraid to ask a question it's really the people conducting the trial are there to help you alright next slide all right so what are the first things that we wanted to talk about is deciding whether to participate in the clinical trial ultimately you're going to find out when you go in the first document they give you when you're talking about a clinical trial is going to be your informed consent form an informed consent form is a very large document it can be up to 11 pages I've seen some that go as much as 20 pages when you're dealing with some more advanced therapies particularly around cancer but the reason why the document is so long is because we're trying to give you as much information in writing upfront as they can so that you can take the time to ask the questions of the clinical provider that you're dealing with ask the questions of your friends and family if there are opinions on what they think you should do if you feel comfortable asking them and also to take the time to carefully go through and read all the information so that you feel comfortable before making a decision nobody should ever give you an informed consent form and ask you to sign it in the same visit right off the bat you really should have time to go through it all and think about it and ask deliberate questions and make sure that this is something that you feel 100% comfortable with as you move forward some points that we want to point out about the informed consent form is that the FDA requires volunteers are given information before they participate so you have to sign a consent form before anybody does anything related to a clinical trial that means even if tracking data from your medical chart or asking you questions about how you feel about certain things informed consent really is to start this relationship so it should have been prior to any other information gathering informed consent documents include information about the purpose of a clinical trial what are they trying to find out why are you being asked to participate in it a description of what the trial is hoping to accomplish a description of what procedures will be conducted while you're in the clinical trial and they can be very detailed all the way down to the number of blood draws the amount of blood they'll draw how much time you're expected to spend in the clinic so you know I'm not much time to schedule any type of internal exams or vasive exams should all be listed in your Clemson in your consent form a complete description of the risks involved in the clinical trial so that means the risks involved with the drug that means risk to your class reality if you have concerns about that that information needs to be addressed in a consent process risk about blood drives we even have to write down the fact that you may get a bruise if we draw blood or that you could get an infection from a needle when you draw blood which are common risk when you draw blood in general but because we're in a trial and we want to make sure that we dispel as many concerns about risk as we can we lay it all out for you on on the wrist section it should also talk to you about any possible benefits that can come from the trial so whether or not this drug is expected to impact your your life or impact your clinical diagnosis whether or not your participation will result in the benefit of society meaning that you'll help gather more information about something that is unknown which can benefit future generations or if there are no benefits they should tell you there's no expected benefits and this is common when you deal with in stage cancer trials sometimes we're really just trying to find knowledge and there will be no personal benefit to the patient but that is the greatest gift that they can give us and so there's always some language about that as well there should also be information about alternatives so alternative treatment options information letting you know that participate in this clinical trial is not the only option available to you in terms of treating your disease there are other methods available that are what we call standard of care meaning common medical practice or an alternative could be not to participate and not to receive any treatment at all that is your right dissipation as well it should also give you information about compensation are they giving you compensation for the time off that you have to take from work are they reimbursing for travel expenses is there any compensation available if you're treated for if you're injured during the conduct at the trial also any information about who to contact if you are injured in a clinical trial or if you have questions about participating in a clinical trial you should have a 24-hour emergency contact number for a physician who's responsible for the oversight of your care while you're in the front of the trial and often symptoms will also give you a number to something that's called an institutional review board an institutional review board is an external committee that is responsible for making sure that all clinical trials conducted at so at a doctor's office or at a university meet the basic requirements of the Code of Federal Regulations which is the FDA safety regulations for clinical trials and they are there to expect patient complaints they can be done anonymously or in person so if you have a complaint or a question and didn't feel comfortable talking to somebody at the doctor's office where you're being seen you can call the institutional review board and somebody there will get in touch with whoever needs to be spoken to on your behalf without having to make you do it if you don't feel comfortable all the information about the fact that you has a right to refuse treatment on the clinical trial or to withdraw yourself from the clinical trial so if at any point you said yes to a study and you get a couple visits in and you decide this just doesn't feel right I don't see benefit or it's taken too much time or really I just don't want to do this anymore you have the right to walk away at any point without penalty to your treatment or penalty by the provider you do not have to continue to participate all the way through to the end of the study if you decide that it's still longer a benefit if it to you or to your to the person that you're helping also the fact that the doctor can withdraw you for safety reasons so if there's something that comes down from another state that's participating that shows that there's a different risk or a threat to safety of patients they have to tell you that up front and they will draw you from the study also there should be information about confidentiality who is going to have access to your medical information how your information will be shared between the doctors is clinical for the pharmaceutical sponsor and the FDA as well as what would happen if there was a confidentiality breach what are your what information is protected by HIPAA and what is your recourse to come get redress for that also they'll let you know if clinical trials are registered on clinical trials gov clinical is an external website that is run by the government that all clinical trials that meet certain criteria are required to register and provide updates on you can access it at wwm above and it will give you the number and registration ID for that study so that you can go and see what is posted there if you so choose X right so if that wasn't enough information in one document the FDA has also added six additional elements meaning that they want to make sure that there is a statement that there may be risks which are unforeseeable so as I mentioned before sometimes when you get into the conduct of a clinical study after a couple of years you start to notice that there are something an additional safety risk and we may not have known that when we started but part of the idea of clinical trials is to identify those risks so that we know them and we can communicate them to others so there should be a statement about that and then what they'll do when the information arises and how you'll be informed of any additional change in risk under what circumstances the investigator to terminate your participation so like I just said if there was an issue with safety the investigator who is your doctor can remove you from the study any additional cost to you that maybe part of participating in the clinical trial sometimes um up until recently there were some pushback by insurance but actually as of 2010 Medicare decided that they would cover a standard of care cost so comps a complimentary care for people participating in clinical trials and most private insurances have followed suit systems so that hopefully will become less of an issue is who move forward when will research findings be disclosed to you so when will the information that comes out from the clinical trial be made public of the approximate number of people participating in the study how many other people like you are there how many people are we trying to get into this study is it 20 is it 300 is it 4000 what is the goal and the fact that you can still decide to withdraw from the study at any time so we want to reinforce this twice because it's very important for people to understand you can quit at any time if you don't feel comfortable and that should be stated it may explore to you before you purchase next slide so that is informed consent so the next thing we wanted to talk to you about is some basic methods in trial design so when you start to get out there and look at different types of protocol titles so we printed on your informed consent forms or if you get on clinical trials that go for CCFA of clinical trial website and you see these clips these trials for protocols you're going to see words like randomized double-blind placebo and you're like what does this mean to me I don't know what randomize means well we're going to tell you so randomized usually means that there are two or more treatments available in the trial so basically somebody the pharmaceutical company is looking at one new drug versus a placebo or a pre-existing drug to see which one has a better effect in the patient population and that these treatment assignments are assigned by chance most often they're assigned by a computer-generated program so that it's as random as flipping a coin whether or not you will receive one treatment versus another sometimes they'll do what's called cohort stratification which means every other person gets put into a treatment group which is a little bit different but still it's very random in that the sequence in which you sign up is random another type of trial you're going to hear about is a double-blind and the double-blind means that neither you nor your doctor know which treatment you are receiving sometimes they'll go as far as a triple blind which means neither you your doctor nor the pharmacist who is prescribing the medication knows which one you're receiving and the reason for that is because they're trying to counter against something that's called the placebo effect and the placebo effect is something that scientists first started to notice a couple decades ago actually that people who knew that they were involved in a clinical trial and thought that they were receiving investigational agent would say that they felt better just because they knew they were in a trial and they're like hey I should feel better so they started to report that they felt better because generally when you become more conscious of your health and you take a more interested role in what's going on you'll start to feel a little bit better about what you do on a day to day life and so in order to counter against that and also to counter against judgment on behalf of the doctor saying I think I know which one is on and I think that this is what's going on they decided to go ahead and create blinding so blinding helps keep everybody in the darkness we don't know what we're on so any positive outcomes that you see or feel really should be tied to the treatment and now just a perception of how you think you should be responding a placebo is an inactive product that usually resembles the study drug or study device sometimes in a device study they call it a sham which means that it's just a dummy device in the drug it means that it's pretty much the look and feel of whatever the active treatment is but it doesn't have the key molecule that I was talking about in the beginning that's the active agent that actually changes how the body metabolizes that drug and like I said they do it so that you don't know what you're on so those are some basic underlying methods of how we do clinical trials most clinical trials that you participate in are going to be randomized they are going to be double-blind and they are going to be placebo controlled those are the most commonly encountered types of clinical trials in the market today next so with that we wanted to talk about the different phases so you saw me with my infographic before that we go from ten thousand different types of compounds down to one eventually approved drugs and along the way there's a couple of different steps and the first step in that process is what we call the preclinical phase so this is what we which is usually experiments done in vitro which means in a cell or a test-tube so we're checking how a different molecule interacts with different genetic material or in vivo which means that is done in animals and the reason why we do that is to see how its metabolized by different types of organs to see if there is any type of effect on the con the biochemistry of the animal that we can that might be a danger if we put it into humans usually these are wide-ranging doses of active drugs and the reason why is because they want to see what's the maximum toxicity or the biggest load of the dose that they can give before we start to see negative effects so they know where the ceiling is and then they want to see which is the most where the bottom is meaning how much drugs do they have to give at a minimum to see any type of effect in the chemistry and processing of the material they're also looking to see how the drug moves through the badi most drugs are metabolized either by the liver or the kidneys and figuring out which organ that is going to affect by this particular molecule and they're also designed to determine if potential drugs have a scientific merit meaning that they are worthy of further development into an I&D which is an actual term that is short for investigational new drug application which is something that the FDA requires every pharmaceutical company to file before they move into clinical trial testing in humans and that means that from that point on they're going to go into the different phases of clinical trial and test instruments and they have to report back to the FDA at every point along those phases to let them know what they're seeing and the FDA will determine whether or not they can move forward and ultimately if their drugs will be approved for market next slide so from preclinical we move into what we call phase one phase one is usually the first time a drug order or device is tested in humans it's tested in a very small amount of healthy volunteers usually between twenty to a hundred healthy volunteers and the reason why they start and healthy populations if possible is because we don't want to make somebody who's sick sicker unnecessarily Phase one clinical trials in cancer patients usually are given to people who have cancer already and those are usually ended end-of-life types of situations so basically they're looking for it what is a safe dose so we know from our place clinical testing with the maximum doses and what the minimum dose is and before we move into Phase one they've usually narrowed it down to three doses that are in the relatively safe range and they want to figure out what it has the most benefit to the body with the least amount of risk and so that's why they tested in healthy population review from there they go to phase 2 phase 2 studies are really looking at safety and interested and effectiveness are there any short-term side effects so anything that happens immediately after dosing with a drug so are is there any rash that accompanies the drug do you have headaches do you feel dizzy does your heart beat faster these are things that we want to know about in short-term side effects we're also narrowing down career for selection which means that we're trying to figure out which population with the disease is going to benefit the most from this drug so is somebody with diabetes who's well controlled or do we need to look at a population that's a little bit higher on the glucose level that is going to see a bigger effect with this drug and it will be a more benefit to them phase two is usually tested on a larger group of people they're looking at a group of 100 to 300 unlike phase one trials phase two trials usually involve patients who have the disease or condition that's being studied as I just mentioned and the goal is really to study how effective and safe the treatment is in a larger group over time and to figure out do patients improve do we see a marketed difference in how they of how their body reacts to the disease after half taking a treatment Phase three that's the next step phase three is looking at whether not it's better than standard treatment so we already know what the safe dose of this new drug is but if we compare it to something that's already available or on the market is it more effective the FDA will never allow us to look at something to prove that it's less effective they want us to either be as good or better than what's currently available and that's common practice in common sense right we want to make sure that any new treatments we are looking at are actually going to benefit and make things better we never want to come in and say oh you were right that other drug you had on the market is way better than this one that is not Knut's standard course of testing they're looking to evaluate again risks and benefits hopefully more benefit less risks and these studies are actually the most expensive and difficult studies to roll because they involve the largest patient population base three studies usually look at a group on between 1,000 to 3,000 patients and they last a number of years and this is actually if you were in a pharmaceutical company the phase that they call the valley of death so this is the place where most drugs don't make it out into base for trial a lot of drugs get discontinued in Phase three because they turned out not to be as effective as they thought patients are not as compliant because the drug does it make them feel good and they are no longer willing to take the drug so pharmaceutical companies will decide that it's no longer worth pursuing this application so this is where you go from your I hear one the number of drugs that are really going to make it out to the market pays for clinical trial are usually conducted after the government has approved a treatment for market so they're looking at long term safety effects of a drug after it's been approved so it's RT showmen is effective and it's safe enough to be available to the population through their doctor but now they want to see if there's any long-term side effects that we didn't discover in the first 10 to 15 years of testing if after 20 years we start to see something a little bit different that we need to reconsider how Prosser approved they're going to look at observation of serious side effects a serious side effect is something that results in a hospitalization or a medical intervention or ultimately in death or a congenital birth defect so they're looking to see if there's anything that pops up that really changes how people's lives are affected when they take this drug by tiny unit into face works animal trials there should not be any unexpected serious adverse events most of that information comes out in the early phases of testing and that's why they start on small populations so they can catch it before the number of people who are exposed to the drug get larger they're also going to test in groups in special populations so seeing if there's any differentiation between men and women or people with a worse condition or who are worse off on the spectrum than others so if you were looking at somebody with diabetes again you might look at somebody who's more controlled this is somebody who's more or less controlled um and that is that on Facebook all right next one so the tape is a clinical trial there's five and these faces are fairly common no matter what type of clinical trial you're looking at and there are several types of clinical trials available most trials that you guys are going to come across in your daily dives or treatment trials meaning that we're looking to find a new treatment that's going to affect how people live their lives and how they handle their disease so we really want to look more at drugs and devices that are implanted we save a child another type of trial is something that's called the prevention trial which can sometimes happen when you're looking at a healthy population they're looking to see if there's a preventive measure that can decrease the risk of developing a condition later on in life some common types of trials that walk around this are heart disease whether or not aspirin is a preventative measure and effective preventative measure against the development of stroke or heart attacks is one example of a type of a preventive trial another type of trial is a diagnostic trial and this type of trial is usually looking at a lab or an imaging type of test determine if one type of test is better at identifying a disease early on or more sensitive to identifying the disease meaning that it takes less time or less genetic material to pick up a viral count or pick up a disease present in the bloodstream and then there are the quality of life trials quality of life trials are really looking at whether or not a change in medicine habits or a change in alternative therapy can improve the quality of life as an individual living with the disease so do you sleep better do you have less pain if you take this drug does that make does this cancer drug cause less pain than this other cancer drug in your bones and so you're therefore you're able to sleep better you feel more awake you're able to accomplish more during your day and ultimately your quality of life is better with this drug than it was with a different drug compassionate use compassionate use trials are where we have determined that there is a drug available that is in an investigational stage that is shown benefit in a chronic condition or in a condition that is life-threatening and the FDA has authorized certain extensions of that drug to the population outside of a clinical trial so it's usually a one-person trial where they're trying to get an access to invest a gel agent because it has the potential to save or drastically also their life and again I worked in cancer for a long time so a lot of my examples will come from cancer it's very common in cancer trials to see compassion ease case scenario next slide that's a little bit about the different types of trials so what about the people who conduct these trials who are they and what are they called your clinical research team should be comprised principal investigator and this is usually a physician sometimes the nurse practitioner and this is clinician is responsible for leading the trial at that site in that office making sure that all the patients enrolled in this study are safe and that so information is collected according to plan and that the information gets to where it needs to be so that we can get what we need in order to journal and not a drug or treatment Estate's a clinical research coordinator is somebody that's going to interface with you every time you come into the clinic this person is the first thing you can talk to you about your consent they're going to tell you in your next visit is supposed to be they're going to help get you in touch with whatever other clinicians you need to be in touch with if you need to go somewhere for x-ray if you need to be somewhere for blood draw they are they're going to be your lifeline in a clinical trial and they are the people who you probably will become most familiar with because you see them so often so it's very important that you feel that you trust your clinical coordinator and a coordinator can be anybody from a ma which is a medical assistant all the way up to a nurse practitioner it's just somebody who's been designated by the investigator to oversee your care plan next slide so clinical trials for inflammatory bowel disease the we wanted to give you some ideas of the type of trials that are out there for you trials are appropriate for many different types of people including those with IBD um specific requirements vary by trial so it depends on which trial we're looking at whether or not your criteria would qualify and you can talk more about that with your individual doctor the more people who take part in clinics 12:05 the faster we will find a better way to treat and potentially cure IBD so the more people who participate the shorter the time Lynch discovery and the more hope we can offer others out there who living with the same disease next slide common features of IBD SETI's are that they are looking to identify an endpoint so an endpoint is usually the goal of the clinical trial and it employs around IBD typically revolve around system symptom improvement do you pal left side effects when you participate and rug or follow a certain treatment plan do you have an improved quality of life are there less Laura is your day the routine less interrupted are you able to go longer without having to take interventive medication small bowel or colon healing do you see more restoration tissue after taking a certain drug or therapy reduction in need for other medications examples corticosteroids do you have to rely on corticosteroids as heavily as you did at the beginning of the trial if they alter your treatment plan and improving in blood or stool markers of inflammation so ultimately it's do you see less of those is what you would be looking at next slide so special considerations for IBD study age pediatric clinical trials have different requirements for consent and depending on which state you're in it'll change whether or not you need the mother or the father to purchase it to sign off on a new year consent form some states only require one parent some states require both so it's important to talk about that when you're looking at a treatment course for a child and a lot of Trump pediatric trials if your child is above the age of 12 require something that's called ascent meaning that your child has to opt in as well and that is really for the respective persons and that your child at that point is old enough to kind of understand what's happening and tell you if I'm not they're comfortable with what you and the physician are discussing financial considerations clinical trials often cover medication under investigation but sometimes they do not cover other standard therapy so it's important to talk to your clinical coordinator about what is considered to enter the therapy and what is research meaning what's going to go to your insurance and what's going to be paid for by the study and that should be outlined very clearly for you before you begin participation in the sample trial some studies provide continuing education for patients who respond after specified trial endpoint meaning that if you are on a study that is looking at an active drug versus a placebo if a study has shown significant effect on the active treatment sometimes the sponsor and the FDA will allow them to offer the active treatment to anybody who had received placebo in the crossover study and that's usually designed so that they can get the drug to as many people as they can because it is shown so much benefit next slide so where can you go to find more information you can look at clinical trials on CCFA org at the website there you can go to research match research match has a tool that's called trial finder which can help direct you through some of the information that's on clinical trials backed up we scrape clinical on a daily basis to pull down relevant clinical trial information so that to make it easier to find and easy to read to check that out you can always go to clinical trials that gov itself it has all the information you could ever hope for it more on clinical trials being invested in the US as well as international trial you can go to see TFA partners org which is CFA's partnership with several academic centers designed to help get patients into a registry of patient reported outcomes so that they can help you get connected to trials that will match your criteria simply saw in an online survey and reseed researched updates and information about IBD through CCFA and then ultimately a great place to ask is your doctor he made he or she may not know the answer but they'll help get you connected um all you have to do is ask and that is that next slide and the end it's important to emphasize that we all love the same thing whether you're somebody like myself who helps conduct clinical trials or somebody like Leslie who's participated in clinical child or somebody like you who's just out there thinking about simple trials we all want a better life for ourselves and a less chance of future generations having to deal with the same disease so working together begin finances faster we want to go beyond the limits of financial contributions and allow for a bigger impact through research you don't have to give money to two causes to help them find a cure you can actually be to cure yourself and come check us out at research match org backslash partners backslash T CFA and I believe that it's last I and this concludes the presentation great Thank You Katherine lovely for that presentation now we are going to enter the question and answer part of the program so today's questions will be taken from our web participants for everyone's benefit please keep your questions without many personal details so that we can provide an answer that's general in nature and please note that today's presenters are not medical doctors or gastroenterologist they are experts in the area of clinical research therefore in the interest of time I also ask that you keep your questions related to the topic of clinical trials and to ask a question previews the Q&A box on the toolbar on the right side of your screen so the first question for whoever would like to answer it is a two-parter so I have been on many types of medications to manage my Crohn's disease and none seem to be helping control my symptoms and keep flares under control number one is a clinical trial really a good option for me if I'm looking for another treatment number two what if I get a placebo okay well I think that is an interesting question I think that if you have been through a number of medications and feel that you have come to the end of your road that's available to you through regular physicians clinical trials can be an option I think it will be necessary for you to have a bright conversation with your physician about what you're looking for and to go through some of the trial opportunities out there to see what might be a good fit for you the trial finder tool that I mentioned before on research match actually has some filtering built around that exact option that you've run out of treatment options and are looking for a different alternative and that can help direct you to some trials that may be a good starting points of that discussion and as far as whether or not you get a placebo that is a risk in most clinical trials and unfortunately it's a necessary evil like I said to make sure that we keep our data as clean as we can and that patients are reporting and doctors are reporting with actually happening and not what they're perceived to be happening however as you mentioned on the last slide there even if you do receive a placebo there is the chance that you could be in one of those trials that will extend over the after treatment once it's shown benefit so just because you're on placebo doesn't mean that has to be the last thing that you received there I think also is lovely about there is some benefit for having that extra relationship with your physician they get to know you in a different way let them if you want to comment on that a little bit I think that's really key that you do share your concerns with your physician and that they can look back at your dosing and other factors that may be playing a part in the increasing severity of your symptoms so like Katherine said I do believe the clinical trial should still be one of the options that you have in your back pocket but certainly talking more with your physician about your concerns is key great thank you the next question if my ulcerative colitis gets better while I'm in the clinical trial what will happen when the trial is closed well the medication be stopped even if I'm feeling better so again if you're also implying improved while you're on an experimental treatment you're under the auspice of a clinical trial that is great that is definitely something they want to see if the improvement is enough they will open that arm up to extended care or the crossover because they want to maintain that status of life it's not always 100% that will happen now so sometimes there is a chance that you will be taken off of that medication most trials that I've ever conducted where there's been a significant increase in patients quality of life have rolled into another phase of study and that is just anecdotal evidence on my half I don't know if I have a better answer than that it really is it's a good question I wish I had a better answer but to say that there's a chance you will be taken off and there's a chance you won't and it's hard to say without knowing the type of trial going into it whether or not that will be the case great thank you next question what happens if a person seems to be getting worse during a clinical trial if you're getting worse during a clinical trial you need to have a frank discussion with your doctor and your doctor it is closely observing you during this clinical trial and we'll be watching that and nobody wants you to stay on the treatment if it's not working and we definitely don't want to do a on a treatment if it's making you feel worse so your doctor will take you off a study if you are not showing improvement and then factor deteriorating that is they will no no no good doctor will keep you on a study just to get data off of you at the point of suffering so that should never happen and if it does feel free to remove yourself from the console trial as well don't don't don't deal with that great thank you next question what did the investigators do with information on side effects during a trial how can they determine whether a side effect is due to the study or to something else it's a great question so side effects and the whole what we call in my terminology is safety reporting are reported a number of different times so you are reporting on a daily basis basically any side effects that you're experiencing or you're seeing in your patients to your sponsor so to Pfizer I tell them hey I had mrs. Jones come in today and she's reporting that she set aside or she had headaches that are lasting more than several hours and are causing her to take over-the-counter medication that gets reported out to your sponsor and his sponsors takes all that information and collaborates it and we start to see if there is a pattern they actually know which drug you're receiving if you're receiving active or passive oh so they can break it into groups and see if headaches were more frequent in the groups that have active treatment um different side effects if they are serious in nature so some of those ones that I talked about before that require hospitalization or medical intervention are reported to the FDA within 24 hours so that the FDA is aware of the safety information and then the sponsor has to disseminate that information to all the other doctors who are participating in the clinical trials as well so we're constantly getting updates on the side effects that are being seen in the population of both at our site and then in also other states that are participating so that we are all well informed of what's being seen and in terms of assessing whether or not something is related to a drug your doctor can make a determinate in his or her best judgment of whether or not it's related to the drug sometimes it has to do with the timing of when the event occurs so are you getting headaches within ten minutes of taking the drug or are you getting headaches three days later system the likelihood of that headaches is related to the drug that you took three days ago it's a little bit less than it is if it happened 10 minutes after taking a drug so some of that comes into play also the sponsor can help determine a relationship like I said by looking at the different frequency and then ultimately the FDA will help determine if there's any relationship and major events as well because they're looking at all of that data across the United States and if it's an international trial at any global size of law and if you can imagine doing that visit with your physician when you're in a trial they will go through a checklist on Katherine mentioned like she gave the example of headaches but they will go through a list of things that may be side effects of the drug and they will question you if there are symptoms that are not listed and you should definitely also share that with your physician who will share it with the sponsor right and to play off of that so the different phases of trials that I talked about anything that's found out in phase one is told to investigators going into phase two told the Messier is going into Phase three so during those whole courses of those phases were identifying those expected risk up front and there's expected side effects so we know to ask you are you feeling dizzy do you have headaches because we've seen that before and then if you have something that says hey I don't I'm not busy or I don't have headaches I can't I feel shortness of breath that's a new side effect and we write that down and then we because there's a whole other process of assessing whether or not that's related um which is what I was leading to before great thank you next question how common are crossover trials and do patients participating in current server trials continue to receive compensation during treatment and usually if you are enrolled in a crossover trial where they're providing the drug to you for free at that point they are not compensated because they're providing the the care around the drug in the drug itself and the commonality across trials depends on the type of trial they're looking at for the different diseases I believe that they're a little bit more common in IVD than they are and others but I can't give you an exact percentage because it's not really my area of expertise on that one lovely any pop them up no it depends I think CCFA would probably be able to give you a little bit more information about that if you wrote them and oh great thank you next question can I continue to work with my gastroenterologist and primary care doctor during the study yes um anything that happens to you during the trial if the doctor who's conducting the trial is not your primary care doctor he or she should be communicating the type of information that comes across because it's all part of your clinical care just because it's part of a trial does it mean it's not part of your medical care so the information that becomes available during those testing should be shared with your physician so feel free to work with them feel free to tell them what you're doing the more people who know about it the better care you're going to receive and just um just piggyback on that when I was done participating in the clinical trial my doctor was very engaged because fortunately for me I thought it was a good thing I started losing weight and so to explain that to her I had to let her know that I was in a clinical trial and that it was one of the great side effects for me right and another example that I have from actually conducting clinical trials a lot of times when we do bloodwork will find high triglycerides which means a high cholesterol or your blood tip tip or your blood pressure is higher than average and we will communicate that to your primary care physician because that's something that needs to be treated outside of the child just because it's something that should be treated so I know that I personally have shared information with primary care physicians to help round out the care of a patient great thank you one last question when would I find out about the study results are participants given the data on the trial findings that is something that we are constantly working on um in terms getting that more more accessible some sponsors will send communications to participants and say hey we found out great news here's what we've seen some don't sometimes that information comes about in publications so that some days gotten together and written an article and research match is working on providing a way to match up studies alpha clinical trials backed up as results and put those results out publicly on our site so that they're publicly available any findings that are found on cities registered a clinical trial site does are expected to be reported there as well sometimes your doctor may know because they've read the literature so they'll tell you there really is not a standardized way of how results are communicated unfortunately it's something that we are constantly pushing for on our end in terms of patient advocacy to get more information about that and make it more common so that people can know how their participation has changed the world around them I think it's important that patients know how they how they've contributed to the research and like Katherine said we are working here at research match to create a venue for dissemination what we want patients to know from the studies that are match from clinical trials and also and research match is what is the clinically meaningful information they can glean from these studies so when you read something about vitamin e's great for you do you know if that's the vitamin E that you buy at your local Walgreens or there's something more potent that could have a better benefit to your health so that's what we're trying to create if this platform for you to understand what's clinically minutes meaningful and that you can share with your doctor or your doctor can share with you great thank you once again Katherine and Leslie for your presentation for answering the participant questions we greatly appreciate it before we conclude the program I just wanted to provide some impel information for for some of the listeners so if you have any additional questions about clinical trials you can reach out to see CFA's IBD Help Center monday through friday 9 a.m. to 5 p.m. Eastern Standard time at eight eight eight six nine four eight eight seven two by email at info at CCFA org or you can even chat with us online with an information specialist directly via our answer chat and you can find more information on CCA's website which is wwf/e org if you would like to watch other educational webcasts on IBD please visit the website on the screen to explore other topics on Crohn's disease and ulcerative colitis you can also connect with other IBD patients and engage in discussions juicy CFA's community website our support groups as well as our power of two peer to peer mentor program and another resource that we have is GI buddy which is a tracking tool and a mobile app that has everything you'll need to stay on top of manager your inflammatory bowel disease and for more information on GI buddy you can go to CCFA org backslash GI buddy and another way you can play a role in research is by becoming a CTS a partner CCFA Partners is an internet-based study that can help researchers better understand IBD while providing you with the tools and resources that you need to help manage your health and when becoming a CCFA partner you will join over 14,000 members that are already a part of this patient-powered research networks you can also participate in other education events by connecting with your local CTF a chapter which you can find by visiting CCSA's website and if you're looking for a way to give back our take steps walk program offers a wonderful way for family and friends and the community to raise a mission critical funds to help us find a cure and our walk events are filled with live music and food kids entertainment and educational materials and you can learn more by visiting see see take steps orgy you can also join our team challenge events with the TAF marathon full marathon triathlon Ironman and cycling training programs you'll train for a rewarding and exciting endurance event at one of our great destination races and you can find more information by visiting WWE team challenge org and also your feedback is very important to us as it helps us improve and develop future programs so in the chat box within the webinar toolbar on the right you will find a link to our evaluation so please be sure to complete this brief evaluation before you depart and on behalf of the Crohn's and Colitis foundation of America and research match thank you so much for joining us goodbye


  1. iam volunteer.i have particapate iintrest to clinical trails in abroad. So please tell me details sir.. do u accepted to Indian People.

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