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

NCT03038399 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 46

Last updated 2021-05-20

Study results available
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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.

Conditions

Interventions

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.

Sponsors & Collaborators

  • University of Pittsburgh

    collaborator OTHER

  • Cooperative International Neuromuscular Research Group

    collaborator NETWORK

  • ReveraGen BioPharma, Inc.

    lead INDUSTRY

Principal Investigators

  • Paula R Clemens, MD · University of Pittsburgh

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2017-02-02
Primary Completion
2020-04-30
Completion
2020-04-30
FDA Drug
Yes

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 NCT03038399 on ClinicalTrials.gov