A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
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ClinicalTrials.gov Identifier: NCT02760264 |
Recruitment Status :
Completed
First Posted : May 3, 2016
Results First Posted : January 2, 2019
Last Update Posted : January 2, 2019
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Study Type | Interventional |
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Study Design | Allocation: Non-Randomized; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Duchenne Muscular Dystrophy |
Interventions |
Drug: Vamorolone 0.25 mg/kg/day Drug: Vamorolone 0.75 mg/kg/day Drug: Vamorolone 2.0 mg/kg/day Drug: Vamorolone 6.0 mg/kg/day |
Enrollment | 48 |
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | Vamorolone 0.25 mg/kg/Day | Vamorolone 0.75 mg/kg/Day | Vamorolone 2.0 mg/kg/Day | Vamorolone 6.0 mg/kg/Day |
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Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Oral administration of 0.25 mg/kg/day daily for 14 days. |
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Oral administration of 0.75 mg/kg/day daily for 14 days. |
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Oral administration of 2.0 mg/kg/day daily for 14 days. |
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Oral administration of 6 mg/kg/day daily for 14 days. |
Period Title: 0.25 mg/kg/Day | ||||
Started | 12 | 0 | 0 | 0 |
Completed | 12 | 0 | 0 | 0 |
Not Completed | 0 | 0 | 0 | 0 |
Period Title: 0.75 mg/kg/Day | ||||
Started | 0 | 12 | 0 | 0 |
Completed | 0 | 12 | 0 | 0 |
Not Completed | 0 | 0 | 0 | 0 |
Period Title: 2.0 mg/kg/Day | ||||
Started | 0 | 0 | 12 | 0 |
Completed | 0 | 0 | 12 | 0 |
Not Completed | 0 | 0 | 0 | 0 |
Period Title: 6.0 mg/kg/Day | ||||
Started | 0 | 0 | 0 | 12 |
Completed | 0 | 0 | 0 | 12 |
Not Completed | 0 | 0 | 0 | 0 |
Arm/Group Title | Dose Level Group 1 | Dose Level Group 2 | Dose Level Group 3 | Dose Level Group 4 | Total | |
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Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days. |
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days. |
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days. |
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days. |
Total of all reporting groups | |
Overall Number of Baseline Participants | 12 | 12 | 12 | 12 | 48 | |
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[Not Specified]
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Age, Customized
Mean (Standard Deviation) Unit of measure: Years |
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Age | Number Analyzed | 12 participants | 12 participants | 12 participants | 12 participants | 48 participants |
5.2 (1.03) | 4.8 (0.83) | 4.7 (0.89) | 4.8 (0.75) | 4.9 (0.87) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 12 participants | 12 participants | 12 participants | 12 participants | 48 participants | |
Female |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Male |
12 100.0%
|
12 100.0%
|
12 100.0%
|
12 100.0%
|
48 100.0%
|
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
||||||
Number Analyzed | 12 participants | 12 participants | 12 participants | 12 participants | 48 participants | |
Hispanic or Latino |
0 0.0%
|
1 8.3%
|
0 0.0%
|
3 25.0%
|
4 8.3%
|
|
Not Hispanic or Latino |
12 100.0%
|
11 91.7%
|
12 100.0%
|
9 75.0%
|
44 91.7%
|
|
Unknown or Not Reported |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
||||||
Number Analyzed | 12 participants | 12 participants | 12 participants | 12 participants | 48 participants | |
American Indian or Alaska Native |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Asian |
0 0.0%
|
1 8.3%
|
0 0.0%
|
0 0.0%
|
1 2.1%
|
|
Native Hawaiian or Other Pacific Islander |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Black or African American |
1 8.3%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
1 2.1%
|
|
White |
11 91.7%
|
10 83.3%
|
12 100.0%
|
12 100.0%
|
45 93.8%
|
|
More than one race |
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
0 0.0%
|
|
Unknown or Not Reported |
0 0.0%
|
1 8.3%
|
0 0.0%
|
0 0.0%
|
1 2.1%
|
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Region of Enrollment
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 12 participants | 12 participants | 12 participants | 12 participants | 48 participants |
Canada |
3 25.0%
|
0 0.0%
|
2 16.7%
|
2 16.7%
|
7 14.6%
|
|
Sweden |
0 0.0%
|
4 33.3%
|
0 0.0%
|
0 0.0%
|
4 8.3%
|
|
United States |
9 75.0%
|
3 25.0%
|
2 16.7%
|
6 50.0%
|
20 41.7%
|
|
United Kingdom |
0 0.0%
|
0 0.0%
|
4 33.3%
|
2 16.7%
|
6 12.5%
|
|
Israel |
0 0.0%
|
3 25.0%
|
2 16.7%
|
0 0.0%
|
5 10.4%
|
|
Australia |
0 0.0%
|
2 16.7%
|
2 16.7%
|
2 16.7%
|
6 12.5%
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Name/Title: | Dr. Eric P. Hoffman |
Organization: | ReveraGen Biopharma Inc. |
Phone: | 240-672-0295 |
EMail: | ericphoffman@gmail.com |
Responsible Party: | ReveraGen BioPharma, Inc. |
ClinicalTrials.gov Identifier: | NCT02760264 |
Other Study ID Numbers: |
VBP15-002 1R44NS095423-01 ( U.S. NIH Grant/Contract ) 1U34AR068616-01 ( U.S. NIH Grant/Contract ) |
First Submitted: | April 28, 2016 |
First Posted: | May 3, 2016 |
Results First Submitted: | September 28, 2018 |
Results First Posted: | January 2, 2019 |
Last Update Posted: | January 2, 2019 |