A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy(DMD)
- Conditions
- Duchenne muscular dystrophy (DMD)MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2016-004262-26-GB
- Lead Sponsor
- ReveraGen BioPharma, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 48
1. Participant’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. Participant has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
a) Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture
consistent with typical DMD, OR
b) 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
c) 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;
Participant is = 4 years and < 7 years of age at time of enrollment in the study;
4. Participant is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening
or 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.Participant 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. Participant and caregiver are willing and able to comply with scheduled visits, study drug administration plan, and
study procedures.
Are the trial subjects under 18? yes
Number of subjects for this age range: 48
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Participant has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
2. Participant has current or history of chronic systemic fungal or viral infections;
3. Participant has had an acute illness within 4 weeks prior to the first dose of study medication;
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. Participant 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;
11.Participant is taking any other investigational drug currently or has taken any other investigational drug within 3
months prior to the start of study treatment; or
12.Participant has previously been enrolled in the study.
Note: Any parameter/test may be repeated at the Investigator's
discretion during Screening and/or Day 1 to determine sustainability
and reproducibility. In addition, subjects may be rescreened if ineligible
due to a transient condition which would prevent the subject from participating, such as an upper respiratory tract infection, or if ineligible
due to negative anti-varicella IgG antibody test result.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method