Learn About Duchenne Clinical Trials From Top to Bottom

Duchenne muscular dystrophy is a rare genetic disease. It is also referred to as DMD or just Duchenne. It mostly affects men, rarely women. DMD weakens and damages the muscles. Life expectancy is 26, although in some cases it can be 30 or 40. There is currently no cure for this degenerative disease—only treatment that alleviates the symptoms. This is one of the main reasons people consider joining a Duchenne clinical trial.

What Are Duchenne Muscular Dystrophy Clinical Trials?

Research centers conduct Duchenne clinical trials to try to find the cure but also to test new treatments and drugs that could treat symptoms more effectively.

There are various DMD clinical trials designed for different participants, study types, and clinical trial phases.


Who Can Participate in DMD Clinical Trials?

Three kinds of participants may be eligible for a Duchenne medical trial:

  1. Healthy volunteers—They can usually participate in Phase I DMD trials
  2. People who suffer from DMD
  3. Female carriers of DMD

What DMD Study Types Are There?

When you browse clinical trials, you could also consider different types:

  • Randomized—Participants are divided into groups and receive different treatments
  • Double-blind—Neither the medical professionals nor the volunteers know who is getting a placebo
  • Observational—They optimize testing procedures, examining better ways to detect the disease
  • Treatment—Medical professionals analyze and test new drugs, treatment, radiation therapy, and surgery procedures
  • Genetic—Gene changes that cause the disease are examined in this clinical trial

What Are Clinical Trial Phases?

After lab research and animal testing, the Food and Drug Administration (FDA) needs to approve a clinical trial with human participants. Once it sets off, a clinical trial can start going through phases, which the FDA also has to authorize.

Phase Description Number of Participants

Phase 0

  • Clinical trials don’t require this phase
  • Volunteers receive a small dose of the drug/treatment

10–15

Phase I

  • The treatment’s safety and dosage are crucial here
  • Volunteers that are healthy take part in this phase the most

15–30

Phase II

  • Medical professionals test the effectiveness of the treatment
  • Medical professionals also monitor for side effects

50–300

Phase III

  • The effectiveness and the side effects of the treatment are the primary focus
  • The new treatment is compared to the standard ones

More than 100

Phase IV

  • Long-term safety and effectiveness is the goal here
  • Long-term risks and benefits are examined

Hundreds to thousands

DoNotPay Can Help You Find and Sign Up for DMD Clinical Trials!

Googling “clinical trials near me,” comparing them, and deciding which one is right for you or your loved one can be challenging. DoNotPay can fast-track your search and find the clinical trials best suited for your preferences.

Open DoNotPay in any web browser and do the following:

  1. Select Clinical Trials
  2. Click on Get Started and apply filters
  3. Tap on the trial of your choice
  4. Hit Contact Now

That’s all you have to do! We will then email the research center on your behalf and start the enrollment process.

Other perks of using DoNotPay as your clinical study search tool include:

  • No fees
  • Regularly updated homepage
  • Extensive database of clinical trials
  • Wide array of university psychology studies and paid medical surveys
  • Limitless clinical study signups
  • Handy bookmarking feature to save and browse clinical trials with ease
  • Smart text alerts to let you know when a new clinical study starts recruiting
  • Continuous tracking of clinical trials you signed up for
  • Vast range of filters such as your age, location, medical condition, and compensation

Why Should I Join a Clinical Trial?

Clinical trials are essential for the betterment of science and medicine. Every existing medication has had to go through meticulous research and tests.

As a healthy volunteer, your motives may be altruistic or financial. As a person who suffers from or whose loved one suffers from Duchenne muscular dystrophy, all the more reason to join a clinical trial. If DMD patients could get even the slightest chance of better quality of life, reduced symptoms, or an efficient cure, participating in clinical trials is definitely worthwhile.

The Pros and Cons of Taking Part in Clinical Trials

With all its many benefits for patients and medicine in general, clinical studies do have some drawbacks. You can compare the two before making an informed decision about signing up for a medical trial:

Pros Cons
  • Earning money if it’s a paid study
  • Getting a chance for a better treatment
  • Monitoring by medical professionals
  • Contributing to the development of medicine
  • Receiving a placebo is a possibility
  • Facing risks of a clinical trial
  • Receiving a treatment that might not get approved by the FDA

What To Take Into Consideration Before Participating in a Clinical Study

Before you decide to sign up for a clinical trial, there are a few details to consider. Being a participant is a serious undertaking, and you should not take it lightly. Mull over these questions:

  1. Are clinical trials safe?
  2. Do I have time to dedicate myself fully to a clinical trial?
  3. Did I gather all the necessary information about the research center and the trial itself?
  4. Did I familiarize myself with the Informed Consent form?
  5. Do I qualify for a clinical trial?
  6. Did I talk to my doctor about whether I should join a trial?

How Else Can I Find Duchenne Clinical Trials?

If you’re interested in other ways you can search for DMD clinical studies, you should consider some of these websites:

Website Key Features Instructions

Duchenne

  • A thorough knowledge base about DMD
  • A large community of patients and caregivers
  • Excellent support for families
  • Helpful resources
  1. Go to Duchenne’s website
  2. Click on The World of Clinical Trials
  3. Scroll down and click on Find a Trial near You
  4. Tap on Visit Our Website
  5. Choose DMD under Conditions
  6. Select your location and hit Search

Parent Project Muscular Dystrophy

  • Detailed information about the causes, progression, and carriers
  • Separate sections for families, healthcare professionals, and educators
  • Current DMD research
  • Duchenne registry of patients and carriers for research centers
  1. Visit their website
  2. Scroll down to The Duchenne Registry
  3. Click on Join
  4. Tap on Join once again
  5. Fill in the registration form
  6. Read the instructions
  7. Click Join a Study

What To Expect From DMD Clinical Studies

When a research center representative reaches out to you after your application, they could ask you additional questions to determine your eligibility. Afterward, you will have to go through a screening process. This may include various check-ups and tests. All clinical trial participants must meet both the inclusion and exclusion criteria based on your age, background, medical condition, etc.

The next step in the process of enrollment is Informed Consent. Its purpose is to inform you about the ins and outs of the clinical trial and its possible risks.

How Long Does a DMD Clinical Trial Last?

There is no unified length of any clinical trial. This can depend on factors such as the trial type—e.g., Observational or Treatment—and the number of participants.

What Is Expected of a Participant?

You may have to travel if a research center isn’t near you, or you might stay there until the conclusion of the trial. Medical professionals could ask you to keep a log to report on your daily life during the clinical study.

You are allowed to change your mind at any time and leave the research center.

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DoNotPay can help you find other clinical trials as well. We have compiled a clinical trial database in states such as Ohio, Utah, Texas, Arizona, and North Carolina. Matching you or your loved one with a suitable clinical trial is not all we can do for you.

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