Understanding the Clinical Trial Process for Huntington's Disease Families
The clinical trial process is an essential part of the research journey, a critical step in bringing new treatments and therapies to our HD community. This article provides a small glimpse into a very complicated, complex process. As patients and families, we can leave all the crazy details to the professionals. Here are some facts that may be important for our community to know.
1. The Importance of Clinical Trials
Clinical trials are controlled studies on human participants designed to evaluate drug and medical device efficacy, safety, and tolerability. For diseases like HD, clinical trials can offer hope. I often hear families use the words hopeless, powerless. From what I found personally, participating in clinical research gives hope and, in a sense, the power to make a change in an often powerless journey.
2. Starting with Preclinical Studies
Before a clinical trial for HD can begin, there are usually extensive laboratory and animal studies. These preclinical studies are designed to identify potential treatments, understand their effects, and ensure they are safe to be tested in humans.
3. Phases of Clinical Trials
Once potential treatment shows promise in preclinical studies, it advances to human testing, which is done in phases:
Phase I: A small group of participants to evaluate the safety, determine a safe dosage range, and identify possible side effects.
Phase II: A larger group of HD patients to evaluate the efficacy of the drug and further assess its safety.
Phase III: This phase involves a larger number of participants, sometimes spanning multiple countries, to confirm the drug's effectiveness and monitor side effects.
Phase IV: Post-marketing studies that provide additional information, including the drug's risks, benefits, and optimal use. We saw something like this recently with Austedo.
4. Patient Enrollment and Eligibility
HD patients interested in participating in clinical trials must first undergo an eligibility assessment. We all have heard the words inclusion/exclusion criteria. This process is sometimes stressful for the patients and caregivers. I have heard from many people that when they go through this process, it is a pass or fail and entirely out of their control. They are disappointed if they aren't accepted into the trial. This is something families need to discuss prior. Talk about how it would make you feel if you were ineligible to participate in the trial and how you want your family to support you if you do not meet the eligibility criteria.
You will be asked a lot of questions during this process, but here are some questions you may want to consider asking before committing to enroll in the clinical trial:
What is the purpose of this clinical trial?
How long will the trial last?
How much time out of my life will it take to participate in the clinical trial?
Does this trial involve a placebo?
What are the chances of me getting a placebo?
Will there be reimbursement for expenses?
What are the exclusions to be in the trial?
What will happen if I qualify to be in the trial?
What are all the expectations and requirements for my participation?
Do I have to be tested positive for HD to participate?
What type of intervention/hospitalization will happen during the trial?
Do I need a care partner to participate in the trial?
Is there a drug extension offered after the trial?
What precautions are put in place in case I have a reaction during this trial?
Will you have to follow me after the trial is over?
Does being in the trial exclude me from other trials?
Is there a contact for me during the trial for any questions or concerns that come up?
5. Informed Consent
Before joining a clinical trial, HD patients, or their caregivers, must give informed consent. This means they understand the trial's purpose, potential risks and benefits, and their rights. Importantly, participation is always voluntary, and patients can withdraw at any time.
COERCION: The practice of persuading someone to do something by using force or threats
Please remember that trials are voluntary and up to the participant. Coercing someone to participate in a clinical trial is illegal.
6. During the Trial
Participants will either receive the treatment or a placebo in most cases. During the trial, participants will have regular check-ups, which may include physical exams, blood tests, and neurological assessments. Any side effects or unusual symptoms should be immediately reported to the research team.
Here are some tips and things you may want to consider bringing to your clinical trial day:
Leave early for the trial time. If you are stressed about arriving late, both you and your loved one will have a much more challenging day. This is especially important if it is your first time visiting the clinic for this study.
Eat a full breakfast (unless asked to fast).
If you are not bringing your medications, print out a list of all medications, including vitamins and over-the-counter medications and supplements.
Both caregivers/patients should wear comfortable clothing and shoes, as it will be a long day.
Favorite drinks (thickener & straw if needed)
Entertainment (i.e. DVD player or cards)
Change of clothes (keep the weather in mind)
D.M.E. (walker, wheelchair, etc.)
7. After the Trial
Once the trial concludes, researchers will analyze the data collected to determine the treatment's effectiveness and safety. Participants will usually return to their regular medical care but might also receive follow-ups from the trial team. Ultimately, if a drug proves effective and safe through Phases I-III, it may be approved by regulatory bodies, like the U.S. Food and Drug Administration (FDA), making it accessible to the broader HD community. There is also an open-label extension which will make it so the participant can stay on the drug while it is undergoing the regulatory approval process.
8. Access to Trial Results
Clinical trial results are often published in medical journals or presented at conferences, like a Help 4 HD HIPE event. HD patients who participated might also receive updates directly from the trial organizers. This has been a very sensitive subject within our community. We have had a couple of sponsors announce clinical trial results in a way that the community has felt betrayed. They felt like the sponsor had their investors and anything that had to do with money at the forefront of the conversations and left the trial participants in the dark to find out the news on Business Weekly or even Facebook. This is something. Help 4 HD International and others are trying to talk with sponsors about it. Many of these companies are public and obligated to keep their shareholders "in the know." But we try to talk with sponsors about how the patients and families deserve to be "in the know." Seeing as how they put their time and bodies on the line to help us advance HD research. They dont want to hear all their time and effort ended in a trial that didn't make its clinical endpoints on the business wire or social media.
Clinical trials are a beacon of hope for the HD community. They bridge scientific discovery and future treatments that can better our lives. For patients considering participation, discussing with healthcare professionals, understanding the process, and weighing the potential risks and benefits is crucial. Also, talk to your family and find your support system. It is an emotional process. You will have ups and downs. Find your people and talk openly with them about how you want to be supported through your journey participating in clinical research. Each participant's contribution brings us one step closer to a world with new effective treatments – and perhaps one day, our greatest hope, a cure.