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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02760277




Registration number
NCT02760277
Ethics application status
Date submitted
28/04/2016
Date registered
3/05/2016
Date last updated
23/07/2019

Titles & IDs
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)
Secondary ID [1] 0 0
1R44NS095423-01
Secondary ID [2] 0 0
VBP15-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vamorolone 0.25 mg/day/day
Treatment: Drugs - Vamorolone 0.75 mg/day/day
Treatment: Drugs - Vamorolone 2.0 mg/day/day
Treatment: Drugs - Vamorolone 6.0 mg/day/day

Experimental: Dose Level Group 1 - Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.

Experimental: Dose Level Group 2 - Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.

Experimental: Dose Level Group 3 - Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.

Experimental: Dose Level Group 4 - Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.


Treatment: Drugs: Vamorolone 0.25 mg/day/day
Oral administration of 0.25 mg/kg/day daily for 24 weeks.

Treatment: Drugs: Vamorolone 0.75 mg/day/day
Oral administration of 0.75 mg/kg/day daily for 24 weeks.

Treatment: Drugs: Vamorolone 2.0 mg/day/day
Oral administration of 2.0 mg/kg/day daily for 24 weeks.

Treatment: Drugs: Vamorolone 6.0 mg/day/day
Oral administration of 6.0 mg/kg/day daily for 24 weeks.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
Total Number of Adverse Events as Assessed by CTCAE Version 4.03
Timepoint [2] 0 0
24 weeks
Primary outcome [3] 0 0
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity
Timepoint [3] 0 0
002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Primary outcome [4] 0 0
BMI Z-score
Timepoint [4] 0 0
002 Baseline, 003 Week 12, Week 24
Secondary outcome [1] 0 0
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
Timepoint [1] 0 0
002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Secondary outcome [2] 0 0
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH
Timepoint [2] 0 0
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 outcome [3] 0 0
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose
Timepoint [3] 0 0
002 Baseline, 003 Week 12, 003 Week 24
Secondary outcome [4] 0 0
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin
Timepoint [4] 0 0
002 Baseline, 003 Week 12, 003 Week 24
Secondary outcome [5] 0 0
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin
Timepoint [5] 0 0
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 outcome [6] 0 0
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP
Timepoint [6] 0 0
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 outcome [7] 0 0
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX
Timepoint [7] 0 0
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)
Secondary outcome [8] 0 0
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity
Timepoint [8] 0 0
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)
Secondary outcome [9] 0 0
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity
Timepoint [9] 0 0
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)
Secondary outcome [10] 0 0
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)
Timepoint [10] 0 0
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)
Secondary outcome [11] 0 0
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters
Timepoint [11] 0 0
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)

Eligibility
Key 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.
Minimum age
4 Years
Maximum age
7 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/07/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/04/2018
Sample size
Target
Accrual to date
Final
48
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [2] 0 0
Sydney Children's Hospital - Westmead
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
North Carolina
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
Canada
State/province [6] 0 0
Alberta
Country [7] 0 0
Israel
State/province [7] 0 0
Petah Tikwah
Country [8] 0 0
Sweden
State/province [8] 0 0
Gothenburg
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
ReveraGen BioPharma, Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
University of Pittsburgh
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Government body
Name [2] 0 0
National Institute of Neurological Disorders and Stroke (NINDS)
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Cooperative International Neuromuscular Research Group
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Government body
Name [4] 0 0
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Address [4] 0 0
Country [4] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Paula R Clemens, MD
Address 0 0
University of Pittsburgh
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02760277