A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03439670 |
Recruitment Status :
Completed
First Posted : February 20, 2018
Results First Posted : July 13, 2022
Last Update Posted : March 9, 2023
|
Sponsor:
ReveraGen BioPharma, Inc.
Collaborators:
European Union
Cooperative International Neuromuscular Research Group
Newcastle University
University of Pittsburgh
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.
- Study Details
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
Study Type | Interventional |
---|---|
Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
Condition |
Duchenne Muscular Dystrophy |
Interventions |
Drug: Vamorolone Drug: Prednisone Other: Placebo |
Enrollment | 121 |
Participant Flow
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | Treatment Group 1 | Treatment Group 2 | Treatment Group 3 | Treatment Group 4 | Treatment Group 5 | Treatment Group 6 |
---|---|---|---|---|---|---|
![]() |
Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks. |
Period Title: Overall Study | ||||||
Started | 30 | 30 | 15 | 16 | 15 | 15 |
Completed | 28 | 26 | 15 | 15 | 14 | 14 |
Not Completed | 2 | 4 | 0 | 1 | 1 | 1 |
Baseline Characteristics
Arm/Group Title | Treatment Group 1 | Treatment Group 2 | Treatment Group 3 | Treatment Group 4 | Treatment Group 5 | Treatment Group 6 | Total | |
---|---|---|---|---|---|---|---|---|
![]() |
Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks. | Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks. | Total of all reporting groups | |
Overall Number of Baseline Participants | 28 | 28 | 15 | 15 | 14 | 14 | 114 | |
![]() |
The baseline analysis population refers to the baseline visit per protocol while the Participant Flow refers to all randomized participants.
|
|||||||
Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
||||||||
Number Analyzed | 28 participants | 28 participants | 15 participants | 15 participants | 14 participants | 14 participants | 114 participants | |
<=18 years |
28 100.0%
|
28 100.0%
|
15 100.0%
|
15 100.0%
|
14 100.0%
|
14 100.0%
|
114 100.0%
|
|
Between 18 and 65 years |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
>=65 years |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
||||||||
Number Analyzed | 28 participants | 28 participants | 15 participants | 15 participants | 14 participants | 14 participants | 114 participants | |
Female |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Male |
28 100.0%
|
28 100.0%
|
15 100.0%
|
15 100.0%
|
14 100.0%
|
14 100.0%
|
114 100.0%
|
|
Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
||||||||
Number Analyzed | 28 participants | 28 participants | 15 participants | 15 participants | 14 participants | 14 participants | 114 participants | |
American Indian or Alaska Native |
0 0.0%
|
0 0.0%
|
0 0.0%
|
1 6.7%
|
0 0.0%
|
0 0.0%
|
1 0.9%
|
|
Asian |
4 14.3%
|
3 10.7%
|
2 13.3%
|
1 6.7%
|
1 7.1%
|
1 7.1%
|
12 10.5%
|
|
Native Hawaiian or Other Pacific Islander |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Black or African American |
1 3.6%
|
1 3.6%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
2 1.8%
|
|
White |
23 82.1%
|
23 82.1%
|
12 80.0%
|
12 80.0%
|
11 78.6%
|
13 92.9%
|
94 82.5%
|
|
More than one race |
0 0.0%
|
1 3.6%
|
1 6.7%
|
1 6.7%
|
1 7.1%
|
0 0.0%
|
4 3.5%
|
|
Unknown or Not Reported |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
1 7.1%
|
0 0.0%
|
1 0.9%
|
|
TTSTAND (seconds)
Mean (Standard Deviation) Unit of measure: Seconds |
||||||||
Number Analyzed | 28 participants | 28 participants | 15 participants | 15 participants | 14 participants | 14 participants | 114 participants | |
6.01 (2.406) | 5.97 (1.991) | 5.36 (1.948) | 4.53 (0.808) | 5.51 (1.578) | 5.58 (2.288) | 5.560 (1.9498) |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Results Point of Contact
Name/Title: | Dr. Eric Hoffman |
Organization: | ReveraGen BioPharma Inc. |
Phone: | 301-762-7980 |
EMail: | ericphoffman@gmail.com |
Other Publications:
Investigator's Brochure, Version 4, Vamorolone (17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione) 4% Oral Suspension, ReveraGen BioPharma, Inc., January 10, 2017.
FDA Draft Guidance for Industry: Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment. June 2015.
Responsible Party: | ReveraGen BioPharma, Inc. |
ClinicalTrials.gov Identifier: | NCT03439670 |
Other Study ID Numbers: |
VBP15-004 |
First Submitted: | January 9, 2018 |
First Posted: | February 20, 2018 |
Results First Submitted: | February 28, 2022 |
Results First Posted: | July 13, 2022 |
Last Update Posted: | March 9, 2023 |