NL-OMON50318CompletedPhase 2
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) - VBP15-004 - VISION DMD
Conditions
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- ReveraGen BioPharma, Inc.
- Enrollment
- 7
Study Overview
Brief Summary
Trial is onging in other countries
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 2 to 11 (—)
Inclusion Criteria
- •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
- •- 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.
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 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
- •17. Subject has previously been enrolled in the study.
- +2 more not shown
Investigators
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