A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Summary

Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to \<7 years with DMD.

Conditions

• Duchenne Muscular Dystrophy

Interventions

DRUG

Vamorolone

Oral administration of 2.0 mg/kg/day for the duration of the study.

DRUG

Prednisone

Oral administration of 0.75 mg/kg/day for 24 weeks.

OTHER

Placebo

Oral administration of placebo daily for 24 weeks.

DRUG

Vamorolone

Oral administration of 6.0 mg/kg/day for the duration of the study.

DRUG

Prednisone

Oral administration of 0.75 mg/kg/day for 24 weeks.

OTHER

Placebo

Oral administration of placebo daily for 24 weeks.

DRUG

Vamorolone

Oral administration of 2.0 mg/kg/day for 20 weeks.

DRUG

Vamorolone

Oral administration of 6.0 mg/kg/day for 20 weeks.

Sponsors & Collaborators

• European Union

  collaborator OTHER

• Cooperative International Neuromuscular Research Group

  collaborator NETWORK

• Newcastle University

  collaborator OTHER

• University of Pittsburgh

  collaborator OTHER

• ReveraGen BioPharma, Inc.

  lead INDUSTRY

Principal Investigators

• Michela Guglieri, M.D. · John Walton Muscular Dystrophy Research Centre

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

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

4 Years

Max Age

7 Years

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2018-06-29

Primary Completion

2021-02-23

Completion

2021-08-19

FDA Drug

Yes

Countries

• United States
• Australia
• Belgium
• Canada
• Czechia
• Greece
• Israel
• Netherlands
• Spain
• Sweden
• United Kingdom

Study Locations