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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03038399




Registration number
NCT03038399
Ethics application status
Date submitted
30/01/2017
Date registered
31/01/2017
Date last updated
20/05/2021

Titles & IDs
Public title
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Scientific title
A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Secondary ID [1] 0 0
VBP15-LTE
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 months.

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

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

Treatment: Drugs: Vamorolone 6.0 mg/day/day
Oral administration of 6.0 mg/kg/day daily for 24 months.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03 - To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);
Timepoint [1] 0 0
24 months
Primary outcome [2] 0 0
Total Number of Adverse Events as Assessed by CTCAE Version 4.03 - To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).
Timepoint [2] 0 0
24 Months

Eligibility
Key inclusion criteria
1. Subject's parent or legal guardian has provided written informed consent and HIPAA
authorization (if applicable) prior to any VBP15-LTE long-term extension
study-specific procedures;

2. Subject has previously completed study VBP15-003 up to and including the Week 24 Final
assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the
VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is
enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering]; and

3. Subject 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
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subject had a serious or severe adverse event in study VBP15-003 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 long-term extension study;

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

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

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 evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac
abnormality on investigation would not be exclusionary];

6. 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 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
glucocorticoids 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. Subject has an allergy or hypersensitivity to the study medication or to any of its
constituents;

9. Subject has severe behavioral or cognitive problems that preclude participation in the
study, in the opinion of the Investigator;

10. 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

11. Subject 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: Subjects 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
Parallel
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
Other
Name [2] 0 0
Cooperative International Neuromuscular Research Group
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
This long-term extension study is an open-label, multiple-dose study to evaluate the
long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by
liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who
participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.
Trial website
https://clinicaltrials.gov/show/NCT03038399
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

Summary results
Other publications