Treatments and trials
Although there are no FDA-approved therapies for Becker muscular dystrophy, physicians may suggest a number of treatments to help manage your symptoms and support muscle function. These may include heart medications, corticosteroids, physical therapy, respiratory therapy, and supportive braces. Please consult your Care Team specialists for more information and guidance.
On this page, you’ll learn more about
Out here, Elias isn’t thinking about Becker muscular dystrophy. He’s just waiting for his kids to come join him.
Understanding clinical trials
Before considering whether to participate in a clinical trial, it is important to first understand the basics. Clinical trials play a valuable role in identifying new treatments for specific diseases like Becker muscular dystrophy. They help researchers understand how potential treatments work and whether they are safe and effective. The protection of the rights, safety, and welfare of participants in clinical trials is regulated by the FDA in the US and similar agencies worldwide.
Observational studies follow people over time to see how a disease impacts their health and daily life. A specific type, called a natural history study, helps researchers understand how a condition changes and progresses. These insights are important for developing clinical trials and bringing new treatments to patients. Many recent natural history studies have helped shape what the community knows today, and that was made possible by those who participated.
Natural history studies do not involve testing drugs or treatments.
Interventional studies, also known as clinical trials, test new drugs, devices, or treatments to see how well they work for a specific disease or condition. These studies help researchers understand drug or treatment safety and effectiveness before they are approved for public use.
Clinical trial phases
Drug development involves different phases of clinical trials. Each phase has specific objectives.
Safety Profile
The treatment is first tested in a small group of people with a certain condition to learn about its safety and possible side effects.
Efficacy and Safety
Phase 2 trials involve a larger group of people to gather more safety information and test the effects of the treatment.
Confirm Efficacy and Safety
Phase 3 trials, or pivotal trials, involve a larger group of people than phase 2 trials. They are designed to confirm whether the treatment provides a benefit and to gather more detailed safety information. The treatment is usually compared to the current standard treatment or a placebo.
Study Side Effects and Effectiveness Over Time
Phase 4 trials, also called post-marketing surveillance trials, collect information on risks, benefits, and optimal use in a real-world setting after the treatment has been approved by the FDA.
Clinical trial participation – informed consent
Before joining a clinical trial, you’ll go through a process called informed consent. This helps you understand important information so you can decide if participation is right for you.
Informed Consent Process
Review Consent Form
Consent form review with clinical trial principal investigator:
- Purpose of trial
- Trial duration
- Participant responsibility
- Potential risks and benefits
Question & Answer Session
You’ll have the opportunity to take part in a Q&A session with the clinical trial principal investigator to help you fully understand the details of participation. Explore questions you may want to ask here.
Complete Informed Consent Form
By signing the consent form, you confirm that you understand the trial and choose to participate.
Before you join a clinical trial, clinical study investigators will explain the possible risks and benefits so you can make an informed decision. If you choose to participate, you’ll provide your consent in writing. This is a process called informed consent.
Clinical trial participation questions
Considering participating in a clinical trial?
Here are some helpful questions to support a productive discussion with your healthcare provider.
- Who can I contact if have questions during or after the trial?
- Does the clinical trial require hospitalization?
- Do I continue to see my Primary Care physician during the clinical trial?
- Who do I contact if I have a new symptom or side effect?
- What are the possible side effects and benefits of the treatment?
- Where do I receive treatment?
- How is the treatment administered?
- What is the frequency of treatment?
- How will participating in the clinical trial affect my daily routine?
- Will I be able to continue treatment after the clinical trial ends?
- Do I have to pay any medical or travel costs?
- Do I need health insurance to participate in the trial?
Clinical trial key terms
| Clinical Trial | A voluntary research study where participants are assigned to one or more groups (which may include a placebo or other control) so researchers can understand how these treatments affect health, the body, or behavior. |
|---|---|
| Informed Consent | Informed consent is a process where a member of the research team explains the risks and benefits of the clinical trial so the participant can make an informed decision about whether to participate. |
| Placebo | A placebo may be a pill or liquid that looks like the treatment being studied but does not contain any active ingredients or have any real treatment effect. |
| Randomization | Randomization is the process by which two or more alternative treatments are assigned to volunteers by chance rather than by choice. |
| Single- or Double-Blind Studies | Single- and double-blind studies are designed so that participants do not know which treatment they are receiving, helping reduce bias.
In a single-blind study, participants do not know which treatment they are given, but the research team does. In a double-blind study, neither the participants nor the research team knows which treatment is being given. If medically necessary, the research team can find out which treatment participants are receiving. |
