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An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | Clinical Trials | Clareo Health

COMPLETEDPHASE2

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

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

NCT02760277•ReveraGen BioPharma, Inc.

View on ClinicalTrials.gov

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.

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 weeks.

Eligibility

Age

4 - 7 years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•1. Participant's parent or legal guardian has provided written informed consent/HIPAA authorization prior to any extension study-specific procedures;
•2. Participant has previously completed study VBP15-002 up to and including the Week 4 Follow-up assessments within 8 weeks prior to enrollment; and
•3. Participant and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion Criteria

•1. Participant had a serious or severe adverse event in study VBP15-002 that, in the opinion of the Investigator, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this study;
•2. Participant has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
•3. Participant has current or history of chronic systemic fungal or viral infections;
•4. Participant has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
•5. Participant has evidence of symptomatic cardiomyopathy. \[Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary\];
•6. Participant is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents. \[Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical corticosteroids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration\];
•7. Subject has used idebenone within 4 weeks prior to the first dose of study medication;
•8. Participant has an allergy or hypersensitivity to the study medication or to any of its constituents;
•9. Participant has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
•10. Participant has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator; or
+2 more criteria

Locations (12)

University of California Davis

Davis, California, United States

University of Florida

Gainesville, Florida, United States

Nemours Children's Hospital

Orlando, Florida, United States

Ann & Robert H. Lurie Children's Hospital

Chicago, Illinois, United States

Duke University

Durham, North Carolina, United States

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Royal Children's Hospital

Melbourne, Australia

Sydney Children's Hospital

Westmead, Australia

Alberta Children's Hospital

Calgary, Alberta, Canada

Schneider Children's Medical Center

Petah Tikva, Israel

Key Dates

Start Date

July 28, 2016

Primary Completion Date

April 26, 2018

Completion Date

April 26, 2018

Last Updated

July 23, 2019

Enrollment

ACTUAL participants

Conditions

Duchenne Muscular Dystrophy

Interventions

Vamorolone 0.25 mg/day/day

DRUG

Vamorolone 0.75 mg/day/day

DRUG

Vamorolone 2.0 mg/day/day

DRUG

Vamorolone 6.0 mg/day/day

DRUG

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

NCT07037862

Duchenne Muscular Dystrophy (DMD)

View study

RECRUITINGPHASE3

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT07160634

Duchenne Muscular Dystrophy

View study

RECRUITINGPHASE2

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

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 23, 2019Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

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

Fear of Falling in Muscular Dystrophy

Vasodilator and Exercise Study for DMD (VASO-REx)

DMD Voice: Qualitative Interviews With Patients and Caregivers