NCT03038399

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: ReveraGen BioPharma, Inc. · Industry
• Sex: Male
• Age: 4 Years – 7 Years
• Healthy volunteers: Not accepted

Summary

This long-term extension study is an open-label, multiple-dose study to evaluate the long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.

Detailed description

This study will evaluate if it is safe to use a new medication called vamorolone for more than two weeks in children with DMD, if boys with DMD who take the study medication have improved muscle function compared to boys with DMD in other studies who did not take any type of steroid, and to see if boys with DMD who take the study medication gain less weight compared to boys with DMD in a prior study who took another type of steroid called prednisone. Enrolled participants will take the study medication for 24 months.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Vamorolone 0.25 mg/day/day	Oral administration of 0.25 mg/kg/day daily for 24 months.
DRUG	Vamorolone 0.75 mg/day/day	Oral administration of 0.75 mg/kg/day daily for 24 months.
DRUG	Vamorolone 2.0 mg/day/day	Oral administration of 2.0 mg/kg/day daily for 24 months.
DRUG	Vamorolone 6.0 mg/day/day	Oral administration of 6.0 mg/kg/day daily for 24 months.

Timeline

• Start date: 2017-02-02
• Primary completion: 2020-04-30
• Completion: 2020-04-30
• First posted: 2017-01-31
• Last updated: 2021-05-20
• Results posted: 2021-05-20

Locations

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

Regulatory

• FDA-regulated drug study