A Study to Assess the efficacy and the safety of Vamorolone in Boys withDuchenne 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
- EUCTR2017-002704-27-GR
- Lead Sponsor
- ReveraGen BioPharma, Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 120
1. Subject's parent(s) or legal guardian(s) has (have) provided written
informed consent and Health Insurance Portability and Accountability
Act (HIPAA) authorization, where applicable, prior to any study-related
procedures; participants will be asked to give written or verbal assent
according to local requirements
2. Subject has a centrally confirmed (by TRiNDS central genetic
counselor[s]) diagnosis of DMD as defined as:
• Dystrophin immunofluorescence and/or immunoblot showing
complete dystrophin deficiency, and clinical picture consistent with
typical DMD, OR
• Identifiable mutation within the DMD gene (deletion/duplication of
one or more exons), where reading frame can be predicted as 'out-offrame',
and clinical picture consistent with typical DMD, OR
• 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 clinical picture consistent
with typical DMD;
3. Subject is =4 years and <7 years of age at time of enrollment in the
study;
4. Subject weighs >13.0 kg and =39.9 kg at the Screening Visit;
5. Subject is able to walk independently without assistive devices;
6. Subject is able to complete the Time to Stand Test (TTSTAND) without
assistance in <10 seconds, as assessed at the Screening Visit;
7. 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];
8. 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 local laboratory at the Screening Visit;
9. Subject is able to swallow tablets, as confirmed by successful test
aswallowing of placebo tablets during the Screening Period; and
10. Subject and parent(s)/guardian(s) 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: 120
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. 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 a history of primary hyperaldosteronism;
6. Subject has evidence of symptomatic cardiomyopathy [Note:
Asymptomatic cardiac abnormality on investigation would not be
exclusionary];
7. 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 indication other than DMD for no longer than 1 month cumulative, with last use at least 3 months prior to first
dose of study medication, will be considered for eligibility on a case-bycase
basis, unless discontinued for intolerence. Inhaled and/or topical
glucocorticoids prescribed for an indication other than DMD are
permitted if last use is at least 4 weeks prior to first dose of study
medication or are administered at stable dose beginning at least 4 weeks
prior to first dose of study medication and anticipated to be used at the
stable dose regimen for the duration of the study];
8. Subject has an allergy or hypersensitivity to the study medication or
to any of its constituents;
9. Subject has used idebenone within 4 weeks prior to the first dose of
study medication;
10. Subject has severe behavioural or cognitive problems that preclude
participation in the study, in the opinion of the Investigator;
11. 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;
12. Subject is taking (or has taken within 4 weeks prior to the first dose
of study medication) herbal remedies and supplements which can impact
muscle strength and function (e.g. Co-enzyme Q10, Creatine,
Proglandine etc);
13. Subject is taking (or has taken within 3 months prior to the first dose
of study medication) any medication indicated for DMD, including
Exondys51 and Translarna;
14. Subject has been administered a live attenuated vaccine within 14
days prior to the first dose of study medication;
15. Subject is currently taking any other investigational drug or has
taken any other investigational drug within 3 months prior to the first
dose of study medication;
16.Subject has a sibling who is currently enrolled in any vamorolone
study or Expanded Access Program, or who intends to enroll in any
vamorolone study or Expanded Access Program during the subject's
participation in the VBP15-004 study; or
17. Subject has previously been enrolled in the study.
Note: Any parameter/test may be repeated at the Investigator's
discretion during Screening to determine 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 injury, or if ineligible due to negative an
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