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A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)

NCT05166109 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 46

Last updated 2025-09-18

No results posted yet for this study

Summary

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

Conditions

  • Becker Muscular Dystrophy

Interventions

DRUG

Vamorolone

Vamorolone 4.0% wt/wt oral suspension will be administered for the duration of the study.

DRUG

Placebo

Placebo to Vamorolone 4.0% wt/wt oral suspension will be administered for the duration of the study.

Sponsors & Collaborators

  • Santhera Pharmaceuticals

    collaborator INDUSTRY

  • ReveraGen BioPharma, Inc.

    lead INDUSTRY

Principal Investigators

  • Paula Clemens, M.D. · University of Pittsburgh

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
64 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-07
Primary Completion
2025-08-18
Completion
2025-08-18
FDA Drug
Yes

Countries

  • United States
  • Italy

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05166109 on ClinicalTrials.gov