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About
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.
Full 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.
Enrollment
Sex
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Volunteers
Inclusion criteria
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;
Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
Subject is ≥ 4 years and < 7 years of age at time of enrollment in the study;
Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
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);
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
Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
48 participants in 4 patient groups
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Data sourced from clinicaltrials.gov
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