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

COMPLETEDPHASE2

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)

NCT02760264•ReveraGen BioPharma, Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

4 - 6 years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•1. Subject's parent or legal guardian has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
•2. Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
•1. Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
•2. Identifiable mutation within the DMD gene (deletion/duplication of one or more exons) where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical DMD, OR
•3. Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e. nonsense mutation, deletion/duplication leading to a downstream stop codon), with a typical clinical picture of DMD;
•3. Subject is ≥ 4 years and \< 7 years of age at time of enrollment in the study;
•4. Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
•5. Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator. (Note: Serum gamma glutamyl transferase \[GGT\], creatinine, and total bilirubin all must be ≤ upper limit of the normal range at the Screening Visit);
•6. Subject has evidence of chicken pox immunity as determined by presence of IgG antibodies to varicella, as documented by a positive test result from the testing laboratory at the Screening Visit; and
•7. Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion Criteria

•1. Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
•2. Subject has current or history of chronic systemic fungal or viral infections;
•3. Subject has had an acute illness within 4 weeks prior to the first dose of study medication;
•4. Subject 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. Subject has evidence of symptomatic cardiomyopathy. \[Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary\];
•6. Subject 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. Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
•9. Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
•10. Subject 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;
+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 Tikvah, Israel

Key Dates

Start Date

June 1, 2016

Primary Completion Date

May 1, 2018

Completion Date

May 1, 2018

Last Updated

January 2, 2019

Enrollment

ACTUAL participants

Conditions

Duchenne Muscular Dystrophy

Interventions

Vamorolone 0.25 mg/kg/day

DRUG

Vamorolone 0.75 mg/kg/day

DRUG

Vamorolone 2.0 mg/kg/day

DRUG

Vamorolone 6.0 mg/kg/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

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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: January 2, 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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A 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