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 maintain 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.
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Did you Know?
Clinical trials are underway to evaluate whether potential muscular dystrophy treatments may be safe and effective.
"I've discussed clinical trial participation with my Neurologist. It is definitely something that we're investigating and trying to pursue. I would love for the Becker community to have the benefits of a drug that potentially could slow the progression of the disease."
- Nevin, Living with Becker
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 focus on individuals and how the disease affects function and health over time. A type of observational study called a natural history study is designed to provide key information on specific diseases or conditions which supports the design of clinical trials required for approval and use by the general public.
Natural history studies do not study a drug treatment.
Interventional studies are commonly referred to as clinical trials. Interventional studies involve evaluating a potential drug, medical device or treatment for a specific purpose or disease. Usually multiple studies are needed to better understand how a drug or treatment work before approval and use by the general public.
Understanding Clinical Trials
Drug development involves different phases of clinical trials. Each phase has specific objectives.
Clinical Trial Phases
Focus: Safety Profile
Investigation treatment is tested in small group of participants to study safety and side effects.
Focus: Efficacy and Safety
Phase 2 trials are initiated with a larger group of people to collect additional safety information and test the effects of the investigational treatment on individuals who have a certain condition.
Focus: Confirm Efficacy and Safety
Phase 3 trials are designed to demonstrate whether an investigational treatment offers a treatment benefit and to collect more extensive safety data in a larger group of people than Phase 2 trials. Typically, the investigational treatment is compared against the current standard of treatment or placebo.
Focus: Study side effects and effectiveness over time
Phase 4 clinical trials may include thousands of people. Also called post-marketing surveillance trials, these trials are conducted after FDA approval to evaluate long term safety and effectiveness.
Clinical Trial Participation - Informed Consent
To help guide your decision on clinical trial participation, a process known as informed consent is designed to help you learn important information before agreeing to participate in a clinical trial.
Informed Consent Process
Consent Form Review with Clinical Trial Principal Investigator
Consent form review:
- Purpose of trial
- Trial duration
- Participant responsibility
- Potential risks and benefits
Question & Answer Session
Question & Answer session with clinical trial principal investigator to ensure you understand the details regarding clinical trial participation. Click here for questions you can ask.
Complete Informed Consent Form
Signing the consent form indicates that you understand the trial and that you wish to volunteer.
Clinical study investigators must ensure that you have been informed about risks and potential benefits of a clinical trial before you decide to participate and that you have provided your consent to participate in writing. This is called the informed consent process.
Clinical Trial Participation Questions
Considering participating in a clinical trial?
Here are some helpful questions to support a productive discussion with your health care 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 Terms14
| Clinical Trial | A research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes. |
|---|---|
| Informed Consent | Informed consent explains risks and potential benefits about a clinical trial before someone decides whether to participate. |
| Placebo | A placebo is a pill or liquid that looks like the new treatment but does not have any treatment value from active ingredients. |
| 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 Blinded Studies |
Single- or double-blind studies are studies in which the participants do not know which medicine is being used, so they can describe what happens without bias. In single-blind studies, you are not told what is being given, but the research team knows. In a double-blind study, neither you nor the research team are told what you are given. Members of the research team are not told which participants are receiving which treatment, in order to reduce bias. If medically necessary, however, it is always possible to find out which treatment you are receiving. |