A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
- Conditions
- Duchenne Muscular DystrophyMuscular Disorder1002839610028302
- Registration Number
- NL-OMON50318
- Lead Sponsor
- ReveraGen BioPharma, Inc.
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 7
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-of-frame', 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. [Notes: Serum gamma glutamyl transferase (GGT),
creatinine, and total bilirubin all must be * upper limit of the normal range
at the Screening Visit. An abnormal vitamin D level that is considered
clinically significant will not exclude a subject from randomization.];
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 from blood collected during the Screening
Period, OR
- Documentation, provided at the Screening Visit, that the subject has had 2
doses of varicella vaccine, with or without serologic evidence of immunity; the
second of the 2 immunizations must have been given at least 14 days prior to
randomization.
9. Subject is able to swallow tablets, as confirmed by successful test
swallowing 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.
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-by case
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Tolerability:<br /><br>1. Premature discontinuations of study treatment due to adverse events.<br /><br><br /><br>Efficacy:<br /><br>1. Time to Stand Test (TTSTAND) velocity (rise/second): Comparison of the<br /><br>vamorolone 6.0 mg/kg/day dose level group versus the placebo group in change<br /><br>from baseline to the Week 24 assessment.</p><br>
- Secondary Outcome Measures
Name Time Method