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

NCT02760277 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 48

Last updated 2019-07-23

Study results available
· View outcomes & findings →

Summary

The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD, and to see how any potential side effects compare to those seen in boys using steroids.

Conditions

Interventions

DRUG

Vamorolone 0.25 mg/day/day

Oral administration of 0.25 mg/kg/day daily for 24 weeks.

DRUG

Vamorolone 0.75 mg/day/day

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

DRUG

Vamorolone 2.0 mg/day/day

Oral administration of 2.0 mg/kg/day daily for 24 weeks.

DRUG

Vamorolone 6.0 mg/day/day

Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Sponsors & Collaborators

  • University of Pittsburgh

    collaborator OTHER

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • Cooperative International Neuromuscular Research Group

    collaborator NETWORK

  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

    collaborator NIH

  • ReveraGen BioPharma, Inc.

    lead INDUSTRY

Principal Investigators

  • Paula R Clemens, MD · University of Pittsburgh

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
4 Years
Max Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-07-28
Primary Completion
2018-04-26
Completion
2018-04-26

Countries

  • United States
  • Australia
  • Canada
  • Israel
  • Sweden
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

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 NCT02760277 on ClinicalTrials.gov