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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:
Collaborators:
European Union
Cooperative International Neuromuscular Research Group
Newcastle University
University of Pittsburgh
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

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
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
Hide Arm/Group Description 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
Arm/Group Title Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6 Total
Hide Arm/Group Description 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
Hide Baseline Analysis Population Description
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)
1.Primary Outcome
Title Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline
Hide Description Vamorolone at 6.0mg/kg/day vs. placebo group in change from baseline to the Week 24 assessment
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Per the protocol, the primary clinical efficacy endpoint was "comparison of the vamorolone 6.0 mg/kg/day dose level group versus the placebo group in change from baseline to the Week 24 assessment."
Arm/Group Title Treatment Group 1 Treatment Group 2
Hide Arm/Group Description:
Patients enrolled in Treatment Group 1 (placebo comparator group) will receive placebo for 24 weeks.
Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks
Overall Number of Participants Analyzed 28 27
Mean (Standard Deviation)
Unit of Measure: Rises/Seconds
-.007  (0.0628) 0.054  (0.0666)
Time Frame Up to 48 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
Hide Arm/Group Description 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.
All-Cause Mortality
Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/28 (0.00%)   0/28 (0.00%)   0/15 (0.00%)   0/15 (0.00%)   0/14 (0.00%)   0/14 (0.00%) 
Hide Serious Adverse Events
Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/28 (3.57%)   2/28 (7.14%)   0/15 (0.00%)   0/15 (0.00%)   0/14 (0.00%)   0/14 (0.00%) 
Infections and infestations             
Gastroenteritis viral/viral gasteroenteritis  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Appendicitis  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Respiratory, thoracic and mediastinal disorders             
Asthma  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
1
Term from vocabulary, MedDRA
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Treatment Group 1 Treatment Group 2 Treatment Group 3 Treatment Group 4 Treatment Group 5 Treatment Group 6
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   26/28 (92.86%)   26/28 (92.86%)   15/15 (100.00%)   13/15 (86.67%)   12/14 (85.71%)   14/14 (100.00%) 
Blood and lymphatic system disorders             
Leukopenia  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Ear and labyrinth disorders             
Ear pain  1  0/28 (0.00%)  1/28 (3.57%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Tympanic membrane perforation  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Endocrine disorders             
Cushingoid  1  4/28 (14.29%)  9/28 (32.14%)  4/15 (26.67%)  4/15 (26.67%)  1/14 (7.14%)  1/14 (7.14%) 
Growth hormone deficiency  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Gastrointestinal disorders             
Abdominal Pain  1  3/28 (10.71%)  3/28 (10.71%)  2/15 (13.33%)  1/15 (6.67%)  2/14 (14.29%)  0/14 (0.00%) 
Abdominal Pain upper  1  1/28 (3.57%)  3/28 (10.71%)  3/15 (20.00%)  2/15 (13.33%)  1/14 (7.14%)  1/14 (7.14%) 
Constipation  1  3/28 (10.71%)  3/28 (10.71%)  0/15 (0.00%)  2/15 (13.33%)  1/14 (7.14%)  1/14 (7.14%) 
Diarrhoea  1  3/28 (10.71%)  5/28 (17.86%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  1/14 (7.14%) 
Vomiting  1  6/28 (21.43%)  6/28 (21.43%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  3/14 (21.43%) 
Mouth Ulceration  1  0/28 (0.00%)  2/28 (7.14%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Toothache  1  1/28 (3.57%)  1/28 (3.57%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Diarrhoea haemorrhagic  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Flatulance  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
General disorders             
Pyrexia  1  7/28 (25.00%)  3/28 (10.71%)  3/15 (20.00%)  1/15 (6.67%)  3/14 (21.43%)  3/14 (21.43%) 
Asthenia  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Fatigue  1  1/28 (3.57%)  0/28 (0.00%)  2/15 (13.33%)  2/15 (13.33%)  0/14 (0.00%)  0/14 (0.00%) 
Impaired healing  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Thirst  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Hepatobiliary disorders             
Hepatomegaly  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Infections and infestations             
Gasteroenteritis  1  0/28 (0.00%)  1/28 (3.57%)  2/15 (13.33%)  0/15 (0.00%)  1/14 (7.14%)  1/14 (7.14%) 
Nasopharyngitis  1  2/28 (7.14%)  4/28 (14.29%)  3/15 (20.00%)  2/15 (13.33%)  1/14 (7.14%)  2/14 (14.29%) 
Upper respiratory tract infection  1  10/28 (35.71%)  4/28 (14.29%)  2/15 (13.33%)  3/15 (20.00%)  3/14 (21.43%)  2/14 (14.29%) 
Rhinitis  1  2/28 (7.14%)  4/28 (14.29%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  2/14 (14.29%) 
Enterobiasis  1  1/28 (3.57%)  2/28 (7.14%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Conjunctivitis  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Ear Infection  1  1/28 (3.57%)  1/28 (3.57%)  0/15 (0.00%)  1/15 (6.67%)  1/14 (7.14%)  1/14 (7.14%) 
Gastroenteritis  1  0/28 (0.00%)  1/28 (3.57%)  2/15 (13.33%)  0/15 (0.00%)  1/14 (7.14%)  1/14 (7.14%) 
Tonsilitis  1  1/28 (3.57%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  2/14 (14.29%)  0/14 (0.00%) 
Viral Infection  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
COVID-19  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  1/14 (7.14%) 
Cystitis  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Fungal Infection  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Gastroenteritis viral/viral gasteroenteritis  1  3/28 (10.71%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Hand-foot-and-mouth disease  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Impetigo  1  2/28 (7.14%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Influenza  1  1/28 (3.57%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  2/14 (14.29%)  0/14 (0.00%) 
Moluscum contagiosum  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Otitis media bacterial  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Pharyngitis streptococcal  1  1/28 (3.57%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Pneumonia  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Respiratory track infection viral  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Steptococcal infection  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Tooth infection  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Viral upper respiratory tract infection  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Injury, poisoning and procedural complications             
Arthropod bite  1  1/28 (3.57%)  2/28 (7.14%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  1/14 (7.14%) 
Contusion  1  2/28 (7.14%)  1/28 (3.57%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Fall  1  2/28 (7.14%)  4/28 (14.29%)  2/15 (13.33%)  3/15 (20.00%)  0/14 (0.00%)  1/14 (7.14%) 
Ligament strain  1  1/28 (3.57%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Back injury  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Muscle strain  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Vaccination complication  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Investigations             
Blood uric acid increased  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  2/15 (13.33%)  0/14 (0.00%)  1/14 (7.14%) 
Protein Urine Present  1  1/28 (3.57%)  0/28 (0.00%)  1/15 (6.67%)  3/15 (20.00%)  0/14 (0.00%)  0/14 (0.00%) 
Weight increased  1  1/28 (3.57%)  3/28 (10.71%)  1/15 (6.67%)  1/15 (6.67%)  1/14 (7.14%)  1/14 (7.14%) 
Cortisol decreased  1  1/28 (3.57%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Bacterial test postive  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Blood pressure increased  1  1/28 (3.57%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Blood triglycerides increased  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Electrocardiogram abnormal  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Glycosylated haemoglobin increased  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Lipase decreased  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Urine analysis abnormal  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Weight decreased  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Metabolism and nutrition disorders             
Increased appetite  1  1/28 (3.57%)  2/28 (7.14%)  0/15 (0.00%)  1/15 (6.67%)  1/14 (7.14%)  0/14 (0.00%) 
Vitamin D Deficiency  1  2/28 (7.14%)  3/28 (10.71%)  1/15 (6.67%)  2/15 (13.33%)  0/14 (0.00%)  0/14 (0.00%) 
Decreased appetite  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Hypertriglyceridaemia  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Overweight  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Polydipsia  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Musculoskeletal and connective tissue disorders             
Pain in extremity  1  2/28 (7.14%)  3/28 (10.71%)  4/15 (26.67%)  4/15 (26.67%)  2/14 (14.29%)  0/14 (0.00%) 
Back pain  1  1/28 (3.57%)  2/28 (7.14%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  1/14 (7.14%) 
Athralgia  1  1/28 (3.57%)  1/28 (3.57%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Muscle spasms  1  0/28 (0.00%)  1/28 (3.57%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Myalgia  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Costrochondritis  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Joint contracture  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Joint swelling  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Muscle atrophy  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Muscular weakness  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Musculoskeletal pain  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Musculoskeletal stiffness  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Vertebral wedging  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Agitation  1  0/28 (0.00%)  1/28 (3.57%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Skin papilloma  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Nervous system disorders             
Headache  1  3/28 (10.71%)  2/28 (7.14%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  3/14 (21.43%) 
Psychomotor hyperactivity  1  2/28 (7.14%)  0/28 (0.00%)  1/15 (6.67%)  2/15 (13.33%)  0/14 (0.00%)  0/14 (0.00%) 
Dizziness  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Poor quality sleep  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Seizure  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Syncope  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Psychiatric disorders             
Abnormal behaviour  1  2/28 (7.14%)  1/28 (3.57%)  2/15 (13.33%)  0/15 (0.00%)  1/14 (7.14%)  1/14 (7.14%) 
Aggression  1  2/28 (7.14%)  1/28 (3.57%)  0/15 (0.00%)  2/15 (13.33%)  1/14 (7.14%)  0/14 (0.00%) 
Irritability  1  0/28 (0.00%)  3/28 (10.71%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Sleep disorder  1  0/28 (0.00%)  1/28 (3.57%)  1/15 (6.67%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Anger  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Dysphemia  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Emotional disorder  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Enuresis  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Initial insomnia  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Insomnia  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Mood swings  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  1/14 (7.14%)  0/14 (0.00%) 
Oppositional defiant disorder  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Personality change  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Trichotillomania  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Epistaxis  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  1/15 (6.67%)  1/14 (7.14%)  0/14 (0.00%) 
Night sweats  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Renal and urinary disorders             
Chromaturia  1  0/28 (0.00%)  1/28 (3.57%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Proteinuria  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  5/28 (17.86%)  3/28 (10.71%)  2/15 (13.33%)  4/15 (26.67%)  0/14 (0.00%)  2/14 (14.29%) 
Nasal congestion  1  0/28 (0.00%)  2/28 (7.14%)  1/15 (6.67%)  2/15 (13.33%)  0/14 (0.00%)  0/14 (0.00%) 
Rhinorrhoea  1  1/28 (3.57%)  1/28 (3.57%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  1/14 (7.14%) 
Oropharyngeal pain  1  1/28 (3.57%)  0/28 (0.00%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  1/14 (7.14%) 
Skin and subcutaneous tissue disorders             
Hypertrichosis  1  1/28 (3.57%)  3/28 (10.71%)  1/15 (6.67%)  1/15 (6.67%)  0/14 (0.00%)  1/14 (7.14%) 
Rash  1  2/28 (7.14%)  2/28 (7.14%)  2/15 (13.33%)  2/15 (13.33%)  1/14 (7.14%)  2/14 (14.29%) 
Eczema  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Perioral dermatitis  1  0/28 (0.00%)  1/28 (3.57%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Pruritis  1  0/28 (0.00%)  1/28 (3.57%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Dry skin  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Erythema  1  1/28 (3.57%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  1/14 (7.14%)  0/14 (0.00%) 
Rash macropapular  1  0/28 (0.00%)  0/28 (0.00%)  1/15 (6.67%)  0/15 (0.00%)  0/14 (0.00%)  0/14 (0.00%) 
Urticaria  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  0/15 (0.00%)  0/14 (0.00%)  1/14 (7.14%) 
Viral rash  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Vascular disorders             
Hot flush  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
Peripheral coldness  1  0/28 (0.00%)  0/28 (0.00%)  0/15 (0.00%)  1/15 (6.67%)  0/14 (0.00%)  0/14 (0.00%) 
1
Term from vocabulary, MedDRA
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
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.
Layout table for additonal information
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