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Trial record 17 of 1002 for:    BMD

An Extension Study to Assess 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: NCT02760277
Recruitment Status : Completed
First Posted : May 3, 2016
Results First Posted : July 23, 2019
Last Update Posted : July 23, 2019
Sponsor:
Collaborators:
University of Pittsburgh
National Institute of Neurological Disorders and Stroke (NINDS)
Cooperative International Neuromuscular Research Group
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
ReveraGen BioPharma, Inc.

Study Type Interventional
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/day/day
Drug: Vamorolone 0.75 mg/day/day
Drug: Vamorolone 2.0 mg/day/day
Drug: Vamorolone 6.0 mg/day/day
Enrollment 48
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Period Title: Vamorolone 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
Hide Arm/Group Description

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Total of all reporting groups
Overall Number of Baseline Participants 12 12 12 12 48
Hide Baseline Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Age, Customized  
Mean (Standard Deviation)
Unit of measure:  Years
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/Gender, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Male Number Analyzed 12 participants 12 participants 12 participants 12 participants 48 participants
12
 100.0%
12
 100.0%
12
 100.0%
12
 100.0%
48
 100.0%
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%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 12 participants 12 participants 12 participants 12 participants 48 participants
Canada 3 0 2 2 7
Sweden 0 4 0 0 4
United States 9 3 2 6 20
United Kingdom 0 0 4 2 6
Israel 0 3 2 0 5
Australia 0 2 2 2 6
1.Primary Outcome
Title Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03
Hide Description 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). Serious adverse events were recorded for up to 30 days after the final administration of study drug; 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- week Treatment Period, in boys ages 4-7 years with DMD.
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Measure Type: Number
Unit of Measure: participants
Subjects with Any TEAE 10 10 11 11
Subjects with Any Drug Related TEAE 1 2 4 5
Subjects with Any CTCAE Grade 3 or Higher TEAE 0 0 0 2
Discontinuation of Study Drug due to TEAE 0 0 0 0
Subjects with Any Serious TEAE 0 1 0 2
Death 0 0 0 0
2.Primary Outcome
Title Total Number of Adverse Events as Assessed by CTCAE Version 4.03
Hide Description 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). Serious adverse events were recorded for up to 30 days after the final administration of study drug; 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- week Treatment Period, in boys ages 4-7 years with DMD.
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Measure Type: Number
Unit of Measure: Events
Total Number of AEs 48 44 54 73
Total Number of TEAEs 48 44 54 72
3.Primary Outcome
Title Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity
Hide Description To compare the efficacy, as measured by the Time to Stand Test (TTSTAND), of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. untreated DMD historical controls in boys ages 4-7 years with DMD
Time Frame 002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: Rises/Second
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
0.18  (0.065) 0.24  (0.090) 0.22  (0.082) 0.19  (0.056)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
0.15  (0.045) 0.22  (0.077) 0.24  (0.078) 0.22  (0.070)
003 Week 12 Number Analyzed 11 participants 12 participants 12 participants 11 participants
0.18  (0.072) 0.23  (0.102) 0.24  (0.089) 0.22  (0.075)
003 Week 12 Change from 002 Baseline Number Analyzed 11 participants 12 participants 12 participants 11 participants
-0.01  (0.061) 0.00  (0.054) 0.02  (0.066) 0.02  (0.034)
003 Week 24 Number Analyzed 10 participants 12 participants 12 participants 11 participants
0.18  (0.081) 0.24  (0.114) 0.26  (0.108) 0.24  (0.086)
003 Week 24 Change from 002 Baseline Number Analyzed 10 participants 12 participants 12 participants 11 participants
-0.01  (0.066) 0.00  (0.062) 0.05  (0.061) 0.04  (0.045)
4.Primary Outcome
Title BMI Z-score
Hide Description

Summary of BMI Z-score of Safety Population.

Please note 0 is the mean. A negative result indicates a response that is many standard deviations below the mean, and a positive result indicates a response that is many standard deviations above the mean. In this case, the closer the group mean BMI Z-score is to 0 is more favorable.

Time Frame 002 Baseline, 003 Week 12, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: z score
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
1.165  (0.6219) 0.703  (1.0738) 1.200  (0.5325) 0.695  (0.7189)
003 Week 12 Number Analyzed 12 participants 12 participants 12 participants 11 participants
1.103  (0.6457) 0.494  (1.0680) 1.261  (0.3981) 1.011  (0.7034)
003 Week 12 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
-0.062  (0.2438) -0.209  (0.4078) 0.062  (0.3886) 0.174  (0.5826)
003 Week 24 Number Analyzed 12 participants 12 participants 12 participants 11 participants
1.004  (0.6381) 0.493  (1.1696) 1.242  (0.4596) 1.330  (0.5857)
003 Week 24 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
-0.161  (0.3234) -0.210  (0.3629) 0.043  (0.3849) 0.493  (0.6363)
5.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: % of HbA1c
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
5.18  (0.260) 5.22  (0.244) 5.19  (0.124) 5.23  (0.231)
003 Week 8 Number Analyzed 12 participants 12 participants 12 participants 11 participants
5.18  (0.226) 5.33  (0.250) 5.28  (0.204) 5.25  (0.216)
003 Week 8 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.00  (0.128) 0.12  (0.153) 0.09  (0.198) 0.00  (0.100)
003 Week 16 Number Analyzed 12 participants 11 participants 12 participants 11 participants
5.26  (0.239) 5.35  (0.238) 5.26  (0.156) 5.31  (0.270)
003 Week 16 Change from 002 Baseline Number Analyzed 12 participants 11 participants 12 participants 11 participants
0.08  (0.204) 0.14  (0.225) 0.07  (0.130) 0.05  (0.113)
003 Week 24 Number Analyzed 8 participants 12 participants 12 participants 11 participants
5.15  (0.302) 5.22  (0.221) 5.13  (0.160) 5.24  (0.254)
003 Week 24 Change from 002 Baseline Number Analyzed 8 participants 12 participants 12 participants 11 participants
-0.10  (0.220) 0.00  (0.186) -0.07  (0.130) -0.02  (0.154)
003 Week 26-29 Number Analyzed 3 participants 0 participants 0 participants 0 participants
5.07  (0.208)
003 Week 26-29 Change from 002 Baseline Number Analyzed 3 participants 0 participants 0 participants 0 participants
-0.07  (0.208)
6.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: % change
003 Week 8 % Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.06  (2.470) 2.28  (3.008) 1.79  (3.864) 0.02  (1.927)
003 Week 16 % Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
1.70  (3.989) 2.72  (4.457) 1.30  (2.505) 1.03  (2.14)
003 Week 24 % Change from 002 Baseline Number Analyzed 8 participants 12 participants 12 participants 11 participants
-1.89  (4.801) 0.08  (3.629) -1.27  (2.525) -0.33  (2.895)
003 Week 26-29 % Change from 002 Baseline Number Analyzed 3 participants 0 participants 0 participants 0 participants
-1.25  (3.942)
7.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: pg/mL
002 Baseline Number Analyzed 12 participants 11 participants 12 participants 12 participants
18.3  (2.96) 18.0  (6.88) 21.1  (6.13) 19.3  (8.67)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
15.9  (4.52) 18.6  (4.56) 18.2  (5.29) 18.4  (9.73)
003 Week 8 Number Analyzed 12 participants 12 participants 12 participants 11 participants
13.0  (6.25) 7.1  (5.84) 7.8  (4.42) 6.5  (5.23)
003 Week 8 Change from 002 Baseline Number Analyzed 12 participants 11 participants 12 participants 11 participants
-5.3  (6.92) -10.5  (9.32) -13.3  (7.33) -13.5  (7.84)
003 Week 16 Number Analyzed 11 participants 11 participants 12 participants 9 participants
12.2  (4.93) 9.0  (5.86) 9.0  (14.18) 9.0  (4.49)
003 Week 16 Change from 002 Baseline Number Analyzed 11 participants 10 participants 12 participants 9 participants
-6.2  (6.34) -9.1  (9.89) -12.0  (15.42) -12.2  (8.16)
003 Week 24 Number Analyzed 8 participants 12 participants 12 participants 10 participants
19.8  (6.32) 14.0  (3.88) 15.7  (9.63) 11.3  (7.52)
003 Week 24 Change from 002 Baseline Number Analyzed 8 participants 11 participants 12 participants 10 participants
0.6  (6.63) -4.0  (5.48) -5.4  (11.09) -6.3  (7.78)
003 Week 26-29 Number Analyzed 3 participants 0 participants 0 participants 0 participants
9.5  (7.26)
003 Week 26-29 Change from 002 Baseline Number Analyzed 3 participants 0 participants 0 participants 0 participants
-7.8  (4.19)
8.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 002 Baseline, 003 Week 12, 003 Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: mg/dL
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 10 participants
87.5  (9.44) 88.9  (18.71) 89.3  (7.91) 92.3  (8.19)
003 Week 12 Number Analyzed 11 participants 10 participants 12 participants 11 participants
81.5  (5.61) 81.7  (4.35) 84.3  (8.13) 86.5  (5.57)
003 Week 12 Change from 002 Baseline Number Analyzed 11 participants 10 participants 12 participants 10 participants
-6.8  (8.29) -7.6  (19.22) -5.1  (9.01) -5.2  (9.21)
003 Week 24 Number Analyzed 10 participants 10 participants 10 participants 11 participants
80.8  (6.56) 80.8  (4.08) 81.3  (7.94) 84.6  (6.53)
003 Week 24 Change from 002 Baseline Number Analyzed 10 participants 10 participants 10 participants 10 participants
-6.3  (11.97) -9.0  (20.87) -8.1  (10.28) -7.8  (9.44)
9.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 002 Baseline, 003 Week 12, 003 Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: uIU/mL
002 Baseline Number Analyzed 11 participants 8 participants 12 participants 12 participants
5.54  (3.651) 3.09  (2.033) 3.40  (1.548) 3.96  (2.027)
003 Week 12 Number Analyzed 12 participants 12 participants 12 participants 11 participants
4.17  (3.167) 2.97  (1.669) 3.89  (2.189) 6.97  (3.526)
003 Week 12 Change from 002 Baseline Number Analyzed 11 participants 8 participants 12 participants 11 participants
-1.13  (3.822) -0.14  (1.756) 0.49  (2.592) 2.97  (2.277)
003 Week 24 Number Analyzed 11 participants 9 participants 9 participants 7 participants
4.23  (2.560) 3.12  (1.788) 4.82  (3.393) 7.21  (2.374)
003 Week 24 Change from 002 Baseline Number Analyzed 10 participants 5 participants 9 participants 7 participants
-1.67  (4.478) 0.34  (2.898) 1.36  (3.262) 3.26  (2.862)
10.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: ng/mL
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
37.94  (11.622) 35.66  (6.800) 41.17  (5.617) 44.36  (5.979)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
39.20  (14.136) 41.84  (8.552) 47.91  (6.648) 42.81  (10.851)
003 Week 8 Number Analyzed 11 participants 12 participants 12 participants 11 participants
36.21  (10.374) 41.78  (13.856) 44.45  (7.439) 41.55  (5.446)
003 Week 8 Change from 002 Baseline Number Analyzed 11 participants 12 participants 12 participants 11 participants
-1.60  (8.849) 6.13  (10.440) 3.28  (8.325) -2.01  (8.898)
003 Week 16 Number Analyzed 12 participants 12 participants 12 participants 11 participants
39.01  (9.620) 42.23  (9.393) 44.60  (9.534) 39.39  (6.972)
003 Week 16 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
1.07  (9.916) 6.57  (6.709) 3.43  (10.718) -4.17  (8.494)
003 Week 24 Number Analyzed 7 participants 12 participants 12 participants 10 participants
38.80  (6.292) 51.41  (11.265) 51.98  (9.372) 49.08  (7.771)
003 Week 24 Change from 002 Baseline Number Analyzed 7 participants 12 participants 12 participants 10 participants
-1.34  (11.289) 15.75  (10.211) 10.81  (7.542) 5.29  (7.858)
003 Week 26-29 Number Analyzed 3 participants 0 participants 0 participants 0 participants
40.10  (18.729)
003 Week 26-29 Change from 002 Baseline Number Analyzed 3 participants 0 participants 0 participants 0 participants
-1.23  (17.943)
11.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: ng/mL
002 Baseline Number Analyzed 12 participants 11 participants 12 participants 12 participants
555.8  (184.72) 480.7  (118.20) 508.2  (94.36) 511.5  (106.50)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
573.9  (251.02) 489.3  (121.66) 492.0  (81.92) 566.3  (149.32)
003 Week 8 Number Analyzed 11 participants 12 participants 12 participants 11 participants
511.6  (190.94) 459.8  (101.93) 485.2  (105.12) 402.7  (70.46)
003 Week 8 Change from 002 Baseline Number Analyzed 11 participants 11 participants 12 participants 11 participants
-20.3  (120.32) -22.9  (128.79) -23.0  (96.84) -105.6  (121.07)
003 Week 16 Number Analyzed 12 participants 12 participants 12 participants 11 participants
481.9  (159.93) 431.8  (81.25) 455.7  (99.50) 488.5  (130.11)
003 Week 16 Change from 002 Baseline Number Analyzed 12 participants 11 participants 12 participants 11 participants
-73.8  (109.31) -42.4  (109.07) -52.5  (104.05) -19.8  (130.12)
003 Week 24 Number Analyzed 8 participants 12 participants 12 participants 10 participants
457.1  (129.21) 471.1  (121.10) 565.5  (158.89) 526.2  (130.18)
003 Week 24 Change from 002 Baseline Number Analyzed 8 participants 11 participants 12 participants 10 participants
-30.8  (113.64) 2.1  (165.16) 57.3  (150.36) 8.7  (88.95)
003 Week 26-29 Number Analyzed 3 participants 0 participants 0 participants 0 participants
619.0  (379.07)
003 Week 26-29 Change from 002 Baseline Number Analyzed 3 participants 0 participants 0 participants 0 participants
-152.0  (331.46)
12.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers Measured by Levels of CTX
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: pg/mL
002 Baseline Number Analyzed 11 participants 10 participants 12 participants 12 participants
871.0  (160.85) 935.8  (286.50) 936.8  (256.25) 889.3  (186.68)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
915.9  (263.13) 964.4  (319.26) 949.8  (303.76) 989.2  (216.29)
003 Week 8 Number Analyzed 11 participants 12 participants 12 participants 11 participants
897.1  (365.45) 933.3  (330.20) 928.3  (333.11) 825.5  (164.36)
003 Week 8 Change from 002 Baseline Number Analyzed 11 participants 10 participants 12 participants 11 participants
26.1  (368.76) -31.6  (236.34) -8.5  (248.82) -59.6  (259.08)
003 Week 16 Number Analyzed 12 participants 12 participants 12 participants 10 participants
885.4  (261.53) 912.8  (305.08) 939.8  (157.17) 953.7  (199.53)
003 Week 16 Change from 002 Baseline Number Analyzed 11 participants 10 participants 12 participants 10 participants
-2.6  (304.50) -46.3  (321.06) 3.0  (244.71) 102.3  (230.44)
003 Week 24 Number Analyzed 8 participants 12 participants 10 participants 10 participants
1109.3  (287.92) 1235.6  (295.79) 1248.7  (308.90) 1237.0  (277.20)
003 Week 24 Change from 002 Baseline Number Analyzed 8 participants 10 participants 10 participants 10 participants
212.3  (318.86) 295.6  (357.93) 346.5  (327.16) 321.4  (264.65)
003 Week 26-29 Number Analyzed 3 participants 0 participants 0 participants 0 participants
1059.3  (536.26)
003 Week 26-29 Change from 002 Baseline Number Analyzed 2 participants 0 participants 0 participants 0 participants
569.5  (28.99)
13.Secondary Outcome
Title Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Climb Test (TTCLIMB) in boys ages 4-7 years with DMD.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: tasks/ second
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
0.20  (0.054) 0.29  (0.147) 0.29  (0.164) 0.24  (0.086)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
0.20  (0.065) 0.29  (0.168) 0.31  (0.144) 0.25  (0.082)
003 Week 12 Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.21  (0.064) 0.34  (0.238) 0.31  (0.157) 0.26  (0.095)
003 Week 12 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.01  (0.044) 0.05  (0.115) 0.02  (0.107) 0.02  (0.051)
003 Week 24 Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.20  (0.071) 0.30  (0.166) 0.34  (0.148) 0.29  (0.097)
003 Week 24 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.00  (0.076) 0.01  (0.066) 0.04  (0.090) 0.05  (0.061)
14.Secondary Outcome
Title Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Run/Walk Test (TTRW) in boys ages 4-7 years with DMD.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: meters/ second
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
1.60  (0.312) 1.77  (0.367) 1.84  (0.347) 1.64  (0.279)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
1.57  (0.371) 1.78  (0.414) 1.86  (0.418) 1.72  (0.295)
003 Week 12 Number Analyzed 12 participants 12 participants 12 participants 11 participants
1.54  (0.306) 1.77  (0.550) 1.97  (0.503) 1.88  (0.341)
003 Week 12 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
-0.06  (0.261) 0.00  (0.307) 0.13  (0.316) 0.26  (0.297)
003 Week 24 Number Analyzed 12 participants 12 participants 12 participants 11 participants
1.55  (0.384) 1.84  (0.486) 1.90  (0.321) 1.89  (0.378)
003 Week 24 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
-0.05  (0.311) 0.06  (0.210) 0.06  (0.210) 0.27  (0.254)
15.Secondary Outcome
Title Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by North Star Ambulatory Assessment (NSAA) in boys ages 4-7 years with DMD. ***Total NSAA score is being reported. The score can range from 0 to 32. Higher scores (approaching 32) indicate a better outcome assessing functional mobility.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: scores on a scale
002 Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
19.0  (5.13) 20.5  (5.58) 20.0  (4.95) 19.7  (4.94)
003 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
20.1  (7.30) 20.8  (5.66) 21.7  (3.87) 20.4  (4.01)
003 Week 12 Number Analyzed 12 participants 12 participants 12 participants 11 participants
19.3  (5.60) 21.2  (6.45) 21.0  (5.13) 20.4  (5.41)
003 Week 12 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.3  (2.06) 0.7  (2.71) 1.0  (2.56) 0.5  (2.38)
003 Week 24 Number Analyzed 12 participants 12 participants 12 participants 11 participants
19.8  (7.09) 21.6  (7.23) 22.3  (3.80) 22.3  (5.76)
003 Week 24 Change from 002 Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
0.8  (2.83) 1.1  (2.94) 2.3  (1.78) 2.5  (2.62)
16.Secondary Outcome
Title Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters
Hide Description To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by 6-minute Walk Test (6MWT) in boys ages 4-7 years with DMD.
Time Frame 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)
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication in the extension study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description:

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: Meters
002 Baseline Number Analyzed 11 participants 12 participants 10 participants 11 participants
316.2  (59.47) 331.5  (52.76) 353.9  (65.40) 336.8  (63.18)
003 Baseline Number Analyzed 12 participants 12 participants 10 participants 11 participants
294.3  (60.62) 332.2  (56.83) 341.1  (49.42) 335.1  (80.13)
003 Week 12 Number Analyzed 11 participants 11 participants 10 participants 9 participants
312.9  (60.93) 358.7  (71.47) 393.7  (59.72) 369.9  (69.47)
003 Week 12 Change from 002 Baseline Number Analyzed 10 participants 11 participants 10 participants 9 participants
6.0  (28.81) 20.8  (38.09) 39.8  (35.61) 27.6  (42.00)
003 Week 24 Number Analyzed 10 participants 12 participants 10 participants 10 participants
306.2  (68.08) 350.4  (64.23) 383.1  (63.38) 372.6  (69.12)
003 Week 24 Change from 002 Baseline Number Analyzed 10 participants 12 participants 10 participants 9 participants
-11.6  (29.45) 18.9  (41.08) 29.2  (35.91) 43.9  (43.72)
Time Frame Adverse events, including Serious Adverse Events (SAEs), and concomitant medications will be assessed at each study visit and recorded throughout the 24 week treatment period.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Hide Arm/Group Description

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

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

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

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

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

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

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

Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily for 24 weeks.

All-Cause Mortality
Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/12 (0.00%)   0/12 (0.00%)   0/12 (0.00%)   0/12 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/12 (0.00%)   1/12 (8.33%)   0/12 (0.00%)   2/12 (16.67%) 
Infections and infestations         
Pneumonia  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Metabolism and nutrition disorders         
Dehydration  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Reproductive system and breast disorders         
Testicular Torsion  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Respiratory, thoracic and mediastinal disorders         
Hypoxia  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
1
Term from vocabulary, MedDRA
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Dose Level Group 1 Dose Level Group 2 Dose Level Group 3 Dose Level Group 4
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   10/12 (83.33%)   10/12 (83.33%)   11/12 (91.67%)   11/12 (91.67%) 
Cardiac disorders         
Left ventricular dysfunction  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Ear and labyrinth disorders         
Ear pain  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  1/12 (8.33%) 
Endocrine disorders         
Cushingoid  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Eye disorders         
Excessive eye blinking  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Gastrointestinal disorders         
Abdominal distension  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Abdominal Pain  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Abdominal pain upper  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  2/12 (16.67%) 
Constipation  1  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%)  2/12 (16.67%) 
Diarrhoea  1  2/12 (16.67%)  0/12 (0.00%)  2/12 (16.67%)  1/12 (8.33%) 
Faeces discoloured  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Lip swelling  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Gastrointestinal disorder  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Nausea  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Vomiting  1  1/12 (8.33%)  1/12 (8.33%)  3/12 (25.00%)  2/12 (16.67%) 
General disorders         
Gait disturbance  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Pyrexia  1  2/12 (16.67%)  7/12 (58.33%)  5/12 (41.67%)  3/12 (25.00%) 
Thirst  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Immune system disorders         
Seasonal allergy  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Infections and infestations         
Bronchitis  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Conjunctivitis  1  1/12 (8.33%)  0/12 (0.00%)  2/12 (16.67%)  1/12 (8.33%) 
Ear infection  1  1/12 (8.33%)  1/12 (8.33%)  1/12 (8.33%)  1/12 (8.33%) 
Enterobiasis  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Eye infection  1  2/12 (16.67%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Gastroenteritis  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Gastroenteritis viral  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Gastrointestinal infection  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Hordeolum  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Influenza  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  3/12 (25.00%) 
Otitis Media  1  1/12 (8.33%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Pharyngitis  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Pharyngitis streptococcal  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Pneumonia  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Sinusitis  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Sinusitis bacterial  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Staphylococcal skin infection  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Tooth infection  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Upper respiratory tract infection  1  1/12 (8.33%)  1/12 (8.33%)  1/12 (8.33%)  0/12 (0.00%) 
Viral infection  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Viral upper respiratory tract infection  1  4/12 (33.33%)  4/12 (33.33%)  7/12 (58.33%)  5/12 (41.67%) 
Injury, poisoning and procedural complications         
Arthropod Sting  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Foot fracture  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Human Bite  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Laceration  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Muscle strain  1  1/12 (8.33%)  2/12 (16.67%)  0/12 (0.00%)  1/12 (8.33%) 
Tendon injury  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Upper limb fracture  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Investigations         
Blood cortisol abnormal  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Urine output increased  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Weight increased  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Metabolism and nutrition disorders         
Dehydration  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Hyperlipidaemia  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%) 
Hypoglycaemia  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Vitamin D deficiency  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Back pain  1  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Myalgia  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%) 
Neck pain  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Pain in extremity  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  2/12 (16.67%) 
Pain in jaw  1  2/12 (16.67%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Tendon pain  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Nervous system disorders         
Headache  1  0/12 (0.00%)  1/12 (8.33%)  2/12 (16.67%)  1/12 (8.33%) 
Presyncope  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Psychomotor hyperactivity  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Psychiatric disorders         
Abnormal behavior  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Anxiety  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Emotional disorder  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%) 
Insomnia  1  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Personality change  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Stereotypy  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Tic  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Reproductive system and breast disorders         
Testicular Torsion  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Respiratory, thoracic and mediastinal disorders         
Asthma  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Cough  1  1/12 (8.33%)  4/12 (33.33%)  1/12 (8.33%)  3/12 (25.00%) 
Epistaxis  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Hypoxia  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Nasal Congestion  1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%) 
Oropharyngeal pain  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Productive cough  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Rhinorrhoea  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Upper-airway cough syndrome  1  0/12 (0.00%)  2/12 (16.67%)  0/12 (0.00%)  0/12 (0.00%) 
Skin and subcutaneous tissue disorders         
Dermatitis  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Dermatitis contact  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Eczema  1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Hypertrichosis  1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  2/12 (16.67%) 
Rash  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Rash pruritic  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Swelling face  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Uriticaria  1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
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 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 P. Hoffman
Organization: ReveraGen Biopharma Inc.
Phone: 240-672-0295
EMail: ericphoffman@gmail.com
Layout table for additonal information
Responsible Party: ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier: NCT02760277     History of Changes
Other Study ID Numbers: VBP15-003
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: February 25, 2019
Results First Posted: July 23, 2019
Last Update Posted: July 23, 2019