NCT02760264

A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (actual)
• Sponsor: ReveraGen BioPharma, Inc. · Industry
• Sex: Male
• Age: 4 Years – 6 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and \< 7 years old.

Detailed description

This study will evaluate the safety and tolerability of a new steroid-like medication called vamorolone in boys with DMD ages ≥ 4 years and \< 7 years. Enrolled participants will take the study medication for 14 days followed by a 14 day follow-up period. The potential effectiveness of vamorolone in treating DMD will also be explored.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Vamorolone 0.25 mg/kg/day	Oral administration of 0.25 mg/kg/day daily for 14 days.
DRUG	Vamorolone 0.75 mg/kg/day	Oral administration of 0.75 mg/kg/day daily for 14 days.
DRUG	Vamorolone 2.0 mg/kg/day	Oral administration of 2.0 mg/kg/day daily for 14 days.
DRUG	Vamorolone 6.0 mg/kg/day	Oral administration of 6 mg/kg/day daily for 14 days.

Timeline

• Start date: 2016-06-01
• Primary completion: 2018-05-01
• Completion: 2018-05-01
• First posted: 2016-05-03
• Last updated: 2019-01-02
• Results posted: 2019-01-02

Locations

12 sites across 6 countries: United States, Australia, Canada, Israel, Sweden, United Kingdom