Vamorolone for Becker Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight \<50 kg) daily administered orally compared to placebo over a treatment period of 24 weeks in males with BMD.Funding Source - FDA OOPD
Do I need to stop my current medications to join the trial?
Yes, you need to stop taking oral glucocorticoids or other oral immunosuppressive agents at least 3 months before starting the study medication. Inhaled or topical glucocorticoids are allowed if used at a stable dose for at least 4 weeks before starting the study. You also need to stop taking certain herbal remedies and supplements that affect muscle strength and function at least 4 weeks before starting the study.
Will I have to stop taking my current medications?
The trial requires that you have not taken oral glucocorticoids or other oral immunosuppressive agents for at least 3 months before starting the study medication. Additionally, you should not have used certain mineralocorticoid receptor agents or herbal remedies that affect muscle strength within 4 weeks before starting the study.
What safety data is available for Vamorolone in treating Becker Muscular Dystrophy?
Vamorolone has been shown to have improved safety compared to prednisolone, avoiding or reducing key side effects related to behavior and growth. It has been tested in the bmx mouse model of Becker Muscular Dystrophy, where it demonstrated efficacy and safety. Additionally, in clinical trials for Duchenne Muscular Dystrophy, vamorolone showed no significant relationship between QTcF interval changes and maximum plasma concentration, indicating a favorable safety profile. Vamorolone targets dual nuclear receptors to treat inflammation and cardiomyopathy with improved safety over traditional corticosteroids like prednisone.12345
Is Vamorolone safe for humans?
Vamorolone has been shown to have improved safety compared to other similar drugs like prednisolone, with fewer side effects related to behavior and growth in studies involving muscular dystrophy. It is designed to reduce inflammation with fewer negative effects, making it a potentially safer option for treating conditions like Duchenne and Becker muscular dystrophy.12345
Is the drug Vamorolone a promising treatment for Becker Muscular Dystrophy?
How is the drug vamorolone unique for treating Becker muscular dystrophy?
Vamorolone is unique because it is a first-in-class dissociative steroidal anti-inflammatory drug that not only reduces inflammation but also increases dystrophin protein levels, which is crucial for muscle function. Unlike other treatments, it shows improved safety by reducing side effects commonly associated with traditional steroids.12346
What data supports the idea that Vamorolone for Becker Muscular Dystrophy is an effective drug?
The available research shows that Vamorolone improves muscle strength and endurance in a mouse model of Becker Muscular Dystrophy. It increases the amount of dystrophin protein, which is important for muscle function, and has fewer side effects compared to another drug, prednisolone. This suggests that Vamorolone could be an effective treatment for Becker Muscular Dystrophy.12347
What data supports the effectiveness of the drug Vamorolone for Becker Muscular Dystrophy?
Research shows that Vamorolone, a drug initially developed for Duchenne muscular dystrophy, improved muscle strength and increased dystrophin protein in a mouse model of Becker muscular dystrophy. This suggests it may help treat Becker muscular dystrophy by reducing inflammation and increasing dystrophin, a protein important for muscle health.12347
Who Is on the Research Team?
Paula Clemens, M.D.
Principal Investigator
University of Pittsburgh
Are You a Good Fit for This Trial?
This trial is for males with Becker Muscular Dystrophy who can walk 10 meters in ≤ 30 seconds, even with a cane or walker. They should have an NSAA score ≤ 32 and not be on oral steroids or immunosuppressants for the past 3 months. Participants must agree to use barrier contraception during the study and be between 18-65 years old.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline
Baseline assessments are conducted prior to the first administration of study medication
Treatment
Participants receive vamorolone or placebo for 24 weeks with assessments at Week 4, Week 12, and Week 24
Dose-tapering
Participants not continuing with further vamorolone treatment undergo a 4-week dose-tapering period
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Placebo
- Vamorolone
Find a Clinic Near You
Who Is Running the Clinical Trial?
ReveraGen BioPharma, Inc.
Lead Sponsor
Santhera Pharmaceuticals
Industry Sponsor