A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

• Register: ClinicalTrials.gov
• Last refreshed on: 20 March 2023
• Main ID: NCT03439670
• Date of registration: 09/01/2018
• Prospective Registration: Yes
• Primary sponsor: ReveraGen BioPharma, Inc.
• Public title: A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
• Scientific title: 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)
• Date of first enrolment: June 29, 2018
• Target sample size: 121
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03439670
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Canada, Czechia, Germany, Greece, Israel, Italy, Netherlands, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Michela Guglieri, M.D.
• Affiliation: John Walton Muscular Dystrophy Research Centre

• Name: Paula Clemens, M.D.
• Affiliation: University of Pittsburgh

Key inclusion & exclusion criteria

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 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.

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 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
intolerance. Inhaled and/or topical glucocorticoids are permitted if last use is at
least 4 weeks prior to first dose of study medication or if 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 behavioral 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, 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
anti-varicella IgG antibody test result.



Age minimum: 4 Years
Age maximum: 7 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Prednisone

Drug: Vamorolone

Other: Placebo

Primary Outcome(s)

Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline [Time Frame: 24 weeks]

Secondary ID(s)

VBP15-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

European Union

University of Pittsburgh

Cooperative International Neuromuscular Research Group

Newcastle University

Results

• Results available: Yes
• Date Posted: 13/07/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03439670