An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02760277
• Date of registration: 28/04/2016
• Prospective Registration: Yes
• Primary sponsor: ReveraGen BioPharma, Inc.
• Public title: An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
• Scientific title: A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
• Date of first enrolment: July 28, 2016
• Target sample size: 48
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02760277
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Canada, Israel, Sweden, United Kingdom, United States

Contacts

• Name: Paula R Clemens, MD
• Affiliation: University of Pittsburgh

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Participant's parent or legal guardian has provided written informed consent/HIPAA
authorization prior to any extension study-specific procedures;

2. Participant has previously completed study VBP15-002 up to and including the Week 4
Follow-up assessments within 8 weeks prior to enrollment; and

3. Participant and parent/guardian are willing and able to comply with scheduled visits,
study drug administration plan, and study procedures.

Exclusion Criteria:

1. Participant had a serious or severe adverse event in study VBP15-002 that, in the
opinion of the Investigator, was probably or definitely related to vamorolone use and
precludes safe use of vamorolone for the subject in this study;

2. Participant has current or history of major renal or hepatic impairment, diabetes
mellitus or immunosuppression;

3. Participant has current or history of chronic systemic fungal or viral infections;

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. Subject 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; or

11. Participant is currently taking any investigational drug, or has taken any
investigational drug other than vamorolone within 3 months prior to the start of study
treatment.

Note: Participants may be re-evaluated if ineligible due to a transient condition which
would prevent the subject from participating



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

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Vamorolone 2.0 mg/day/day

Drug: Vamorolone 0.25 mg/day/day

Drug: Vamorolone 0.75 mg/day/day

Drug: Vamorolone 6.0 mg/day/day

Primary Outcome(s)

Total Number of Adverse Events as Assessed by CTCAE Version 4.03 [Time Frame: 24 weeks]

Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 [Time Frame: 24 weeks]

BMI Z-score [Time Frame: 002 Baseline, 003 Week 12, Week 24]

Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity [Time Frame: 002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Secondary Outcome(s)

Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity [Time Frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA) [Time Frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH [Time Frame: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose [Time Frame: 002 Baseline, 003 Week 12, 003 Week 24]

Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin [Time Frame: 002 Baseline, 003 Week 12, 003 Week 24]

Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP [Time Frame: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c [Time Frame: 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29]

Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters [Time Frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Serum Pharmacodynamics Biomarkers Measured by Levels of CTX [Time Frame: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity [Time Frame: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin [Time Frame: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)]

Secondary ID(s)

VBP15-003

1R44NS095423-01

1U34AR068616-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cooperative International Neuromuscular Research Group

National Institute of Neurological Disorders and Stroke (NINDS)

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

University of Pittsburgh

Results

• Results available: Yes
• Date Posted: 23/07/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02760277