39 Participants Needed

Vamorolone for Becker Muscular Dystrophy

Recruiting at 1 trial location
EP
Overseen ByEric P Hoffman, Ph.D.
Prior Safety DataThis treatment has passed at least one previous human trial

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?

Yes, Vamorolone is a promising drug for Becker Muscular Dystrophy. It has shown potential in improving muscle strength and increasing dystrophin protein, which is important for muscle health. It also appears to be safer than other similar drugs, making it a strong candidate for further study.12346

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?

PC

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

Subject agrees to use barrier contraception methods during participation in the study and for 30 days after the tapering dose is completed
My NSAA score is 32 or lower.
Clinical laboratory test results are within the normal range at the Screening Visit or not clinically significant in the opinion of the Investigator
See 6 more

Exclusion Criteria

I have not taken any experimental drugs in the last 3 months.
I have not received a live vaccine in the last 14 days.
I have heart muscle disease with symptoms.
See 9 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 5 weeks

Baseline

Baseline assessments are conducted prior to the first administration of study medication

1 day

Treatment

Participants receive vamorolone or placebo for 24 weeks with assessments at Week 4, Week 12, and Week 24

24 weeks
Visits at Day 1, Week 4, Week 12, and Week 24

Dose-tapering

Participants not continuing with further vamorolone treatment undergo a 4-week dose-tapering period

4 weeks
Contact at Week 26

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks
Visit at Week 28

What Are the Treatments Tested in This Trial?

Interventions

  • Placebo
  • Vamorolone
Trial Overview The study tests Vamorolone, a potential treatment for BMD, against a placebo over six months. It's double-blind, meaning neither researchers nor participants know who gets the real drug versus placebo. The goal is to assess safety, how well it works (efficacy), and its effects on the body (pharmacodynamics).
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Vamorolone 500mg/day [250mg if <50kg body weight]Experimental Treatment1 Intervention
Subjects will be randomized to one of two treatment groups in a 1:2 ratio (placebo:vamorolone).
Group II: PlaceboPlacebo Group1 Intervention
Subjects will be randomized to one of two treatment groups in a 1:2 ratio (placebo:vamorolone).

Find a Clinic Near You

Who Is Running the Clinical Trial?

ReveraGen BioPharma, Inc.

Lead Sponsor

Trials
9
Recruited
440+

Santhera Pharmaceuticals

Industry Sponsor

Trials
32
Recruited
2,800+

Published Research Related to This Trial

Vamorolone, a novel corticosteroid, shows promise in treating Becker muscular dystrophy (BMD) by improving muscle strength and reducing inflammation in a mouse model, indicating its potential efficacy for this genetic condition.
Compared to prednisolone, vamorolone has a better safety profile, causing fewer side effects related to behavior and growth, while also increasing dystrophin protein levels in both heart and skeletal muscle.
Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice.McCormack, NM., Nguyen, NY., Tully, CB., et al.[2023]
Vamorolone, a new anti-inflammatory drug, was found to be safe and well-tolerated in a 2-week study involving 48 boys aged 4 to 7 with Duchenne muscular dystrophy, even at the highest dose of 6.0 mg/kg/day.
The study showed that vamorolone has a better safety profile compared to traditional glucocorticoids, with reduced insulin resistance and adrenal suppression, while still demonstrating anti-inflammatory effects and improving muscle membrane stability.
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.Conklin, LS., Damsker, JM., Hoffman, EP., et al.[2022]
In a clinical trial involving boys with Duchenne muscular dystrophy, vamorolone demonstrated significant improvements in clinical outcomes after 24 weeks of daily dosing, particularly in the time to stand from supine, which was the most sensitive measure of efficacy.
The study found that typical exposure to vamorolone at a daily dose of 2 mg/kg led to substantial decreases in proinflammatory biomarkers within just 2 weeks, indicating its potential anti-inflammatory effects without significant impact on heart rhythm as measured by QTcF intervals.
Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy.Li, X., Conklin, LS., van den Anker, J., et al.[2021]

Citations

Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice. [2023]
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. [2022]
Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy. [2021]
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. [2020]
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. [2021]
Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. [2020]
Efficacy of vamorolone in treatment of Duchene muscle dystrophy. A meta-analysis. [2023]
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