Vamorolone for Becker Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+1 other location

Age: 18 - 65

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: ReveraGen BioPharma, Inc.

Prior Safety Data

Trial Summary

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.

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. ^{1 2 3 4 5}

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. ^{1 2 3 5 6}

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. ^{1 2 3 5 7}

Research Team

Paula Clemens, M.D.

Principal Investigator

University of Pittsburgh

Eligibility Criteria

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.

I am between 18 and 65 years old.

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.

Treatment Details

Interventions

• Placebo (Other)
• Vamorolone (Corticosteroid)

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).

Participant Groups

Group I: Vamorolone 500mg/day [250mg if <50kg body weight] Experimental Treatment 1 Intervention

Subjects will be randomized to one of two treatment groups in a 1:2 ratio (placebo:vamorolone).

Group II: Placebo Placebo Group 1 Intervention

Subjects will be randomized to one of two treatment groups in a 1:2 ratio (placebo:vamorolone).

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

University of Pittsburgh Pittsburgh, PA

Who Is Running the Clinical Trial?

ReveraGen BioPharma, Inc.

Lead Sponsor

Trials

9

Patients Recruited

440+

Santhera Pharmaceuticals

Industry Sponsor

Trials

32

Patients Recruited

2,800+

Findings from Research

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice. McCormack, NM., Nguyen, NY., Tully, CB., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy. Li, X., Conklin, LS., van den Anker, J., et al.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. Smith, EC., Conklin, LS., Hoffman, EP., et al.[2020]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Hoffman, EP., Schwartz, BD., Mengle-Gaw, LJ., et al.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Heier, CR., Yu, Q., Fiorillo, AA., et al.[2020]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of vamorolone in treatment of Duchene muscle dystrophy. A meta-analysis. Elhalag, RH., Motawea, KR., Talat, NE., et al.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice. [2023]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. [2020]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Efficacy of vamorolone in treatment of Duchene muscle dystrophy. A meta-analysis. [2023]