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A 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: NCT02760264
Recruitment Status : Completed
First Posted : May 3, 2016
Results First Posted : January 2, 2019
Last Update Posted : January 2, 2019
Sponsor:
Collaborators:
University of Pittsburgh
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
National Institute of Neurological Disorders and Stroke (NINDS)
Cooperative International Neuromuscular Research Group
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/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
Hide Arm/Group Description

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
Hide Arm/Group Description

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
Hide Baseline Analysis Population Description
[Not Specified]
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: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
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%
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: 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%
1.Primary Outcome
Title Overall Summary 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.

Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group
Time Frame Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in safety population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Measure Type: Number
Unit of Measure: Number of Events
Total Number of Adverse Events 16 18 13 11
Total Number of Treatment Emergent Adverse Events 13 13 11 9
Any Treatment Emergent Adverse Events 7 6 8 7
Any Drug Related Treatment Emergent Adverse Events 1 2 2 3
Any CTCAE Grade 3 or Higher TEAE 0 0 0 0
Discontinuation of Study Drug due to TEAE 0 0 0 0
Any Serious Treatment Emergent Adverse Events 0 0 0 0
Death 0 0 0 0
2.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subject who receive at least dose of vamorolone study medication will be included in the safety population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: mg/ dL
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)
Week 2 Number Analyzed 12 participants 12 participants 12 participants 12 participants
85.3  (9.29) 83.1  (6.69) 89.5  (5.20) 89.2  (11.12)
Week 2 Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 10 participants
-2.2  (10.46) -5.8  (18.92) 0.2  (8.79) -1.3  (9.41)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dose Level Group 1, Dose Level Group 2, Dose Level Group 3, Dose Level Group 4
Comments [Not Specified]
Type of Statistical Test Other
Comments Treatment levels were not compared.
Other Statistical Analysis

Safety Population was used for this analysis. Safety Population includes all subjects who receive at least one dose of vamorolone study medication.

Treatment levels were not compared.
3.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Insulin Resistance) -Fasting Glucose
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subject who receive at least dose of vamorolone study medication will be included in the safety population.
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/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.
Overall Number of Participants Analyzed 12 12 12 10
Mean (Standard Deviation)
Unit of Measure: Week 2 % change from Baseline
-1.8  (13.07) -4.2  (13.98) 0.8  (9.76) -1.2  (9.80)
4.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline , Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: µIU/mL
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)
Week 2 Number Analyzed 11 participants 11 participants 12 participants 12 participants
5.29  (2.671) 3.22  (1.924) 3.87  (2.118) 6.73  (4.599)
Week 2 Change from Baseline Number Analyzed 10 participants 8 participants 12 participants 12 participants
-0.65  (2.913) 0.34  (1.289) 0.47  (2.777) 2.78  (4.651)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dose Level Group 1, Dose Level Group 2, Dose Level Group 3, Dose Level Group 4
Comments [Not Specified]
Type of Statistical Test Other
Comments Treatment levels were not compared.
Other Statistical Analysis

Safety Population was used for this analysis. Safety Population includes all subjects who receive at least one dose of vamorolone study medication.

Treatment levels were not compared.
5.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 10 8 12 12
Mean (Standard Deviation)
Unit of Measure: Week 2 % change from Baseline
-5.54  (32.622) 26.07  (76.483) 42.85  (107.337) 83.55  (117.064)
6.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Adrenal Axis Suppression)- First in Morning Cortisol
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Week 2 (pre-dose)
Hide Outcome Measure Data
Hide Analysis Population 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/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.
Overall Number of Participants Analyzed 11 11 11 10
Mean (Standard Deviation)
Unit of Measure: mcg/dL
10.425  (1.7358) 9.755  (2.7614) 7.321  (3.0322) 3.010  (1.0141)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dose Level Group 1, Dose Level Group 2, Dose Level Group 3, Dose Level Group 4
Comments [Not Specified]
Type of Statistical Test Other
Comments Treatment levels were not compared.
Other Statistical Analysis

Safety Population was used for this analysis. Safety Population includes all subjects who receive at least one dose of vamorolone study medication.

Treatment levels were not compared.
7.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Day 1, Week 2, Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: ng/mL
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)
Day 1 Number Analyzed 12 participants 12 participants 12 participants 11 participants
39.37  (14.189) 35.89  (7.526) 34.93  (6.881) 33.52  (9.135)
Day 1 Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
1.43  (8.197) 0.23  (5.304) -6.23  (10.930) -11.37  (9.137)
Week 2 Number Analyzed 12 participants 12 participants 12 participants 12 participants
38.53  (12.025) 35.10  (8.238) 37.51  (8.624) 29.04  (4.853)
Week 2 Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
0.58  (5.986) -0.56  (7.934) -3.66  (9.818) -15.32  (6.450)
Week 4 Number Analyzed 12 participants 12 participants 12 participants 12 participants
39.20  (14.136) 42.24  (8.426) 47.91  (6.648) 42.81  (10.851)
Week 4 Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
1.26  (5.650) 6.58  (9.341) 6.74  (9.747) -1.55  (11.176)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dose Level Group 1, Dose Level Group 2, Dose Level Group 3, Dose Level Group 4
Comments [Not Specified]
Type of Statistical Test Other
Comments Treatment levels were not compared.
Other Statistical Analysis

Safety Population was used for this analysis. Safety Population includes all subjects who receive at least one dose of vamorolone study medication.

Treatment levels were not compared.
8.Secondary Outcome
Title Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Day 1, Week 2, Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: % change from Baseline
Day 1 Percent Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 11 participants
3.95  (22.015) 1.41  (15.449) -12.63  (25.020) -25.05  (20.228)
Week 2 Percent Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
2.48  (16.756) -0.07  (22.408) -7.81  (23.664) -33.87  (12.548)
Week 4 Percent Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
2.72  (14.063) 20.91  (28.644) 18.74  (25.095) -2.43  (25.932)
9.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline Day 1 Week 2 Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: ng/mL
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)
Day 1 Number Analyzed 12 participants 12 participants 12 participants 11 participants
474.0  (116.45) 443.7  (112.38) 417.1  (87.35) 475.2  (147.10)
Day 1 Change from Baseline Number Analyzed 12 participants 11 participants 12 participants 11 participants
-81.8  (124.24) -34.5  (101.97) -91.1  (64.64) -36.5  (144.04)
Week 2 Number Analyzed 12 participants 12 participants 12 participants 12 participants
443.8  (93.86) 407.8  (96.54) 346.6  (68.59) 303.7  (56.38)
Week 2 Change from Baseline Number Analyzed 12 participants 11 participants 12 participants 12 participants
-112.0  (125.08) -70.6  (123.69) -161.6  (73.52) -207.8  (78.16)
Week 4 Number Analyzed 12 participants 12 participants 12 participants 12 participants
573.8  (251.02) 496.7  (117.57) 492.0  (81.92) 566.3  (149.32)
Week 4 Change from Baseline Number Analyzed 12 participants 11 participants 12 participants 12 participants
18.1  (153.10) 21.3  (122.87) -16.2  (79.64) 54.8  (118.09)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dose Level Group 1, Dose Level Group 2, Dose Level Group 3, Dose Level Group 4
Comments [Not Specified]
Type of Statistical Test Other
Comments Treatment levels were not compared.
Other Statistical Analysis

Safety Population was used for this analysis. Safety Population includes all subjects who receive at least one dose of vamorolone study medication.

Treatment levels were not compared.
10.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Day 1, Week 2, Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: % change from Baseline
Day 1 Percent Change from Baseline Number Analyzed 12 participants 11 participants 12 participants 11 participants
-12.2  (17.07) -5.4  (24.58) -17.4  (12.59) -5.7  (30.76)
Week 2 Percent Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
-17.5  (14.65) -11.2  (29.19) -30.9  (11.80) -39.9  (9.22)
Week 4 Percent Change from Baseline Number Analyzed 12 participants 12 participants 12 participants 12 participants
2.8  (25.57) 7.8  (30.86) -1.4  (17.47) 11.8  (28.24)
11.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Day 1, Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: pg/mL
Baseline Number Analyzed 11 participants 12 participants 12 participants 12 participants
871.0  (160.85) 935.8  (286.50) 936.8  (256.25) 889.3  (186.68)
Day 1 Number Analyzed 12 participants 12 participants 12 participants 11 participants
974.8  (252.77) 940.8  (227.60) 838.3  (233.30) 786.8  (331.68)
Day 1 Change from Baseline Number Analyzed 11 participants 10 participants 12 participants 11 participants
72.4  (241.87) -12.9  (233.44) -98.5  (237.69) -115.7  (421.04)
Week 2 Number Analyzed 12 participants 12 participants 12 participants 12 participants
963.7  (157.68) 903.3  (251.01) 710.4  (180.03) 625.7  (203.19)
Week 2 Change from Baseline Number Analyzed 11 participants 10 participants 12 participants 12 participants
85.0  (185.90) -19.9  (266.99) -226.4  (185.99) -263.7  (229.69)
Week 4 Number Analyzed 12 participants 12 participants 12 participants 12 participants
915.9  (263.13) 983.5  (298.47) 949.8  (303.76) 989.2  (216.29)
Week 4 Change from Baseline Number Analyzed 11 participants 10 participants 12 participants 12 participants
17.4  (267.45) 34.8  (271.50) 12.9  (181.45) 99.8  (211.90)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Dose Level Group 1, Dose Level Group 2, Dose Level Group 3, Dose Level Group 4
Comments [Not Specified]
Type of Statistical Test Other
Comments Treatment levels were not compared.
Other Statistical Analysis

Safety Population was used for this analysis. Safety Population includes all subjects who receive at least one dose of vamorolone study medication.

Treatment levels were not compared.
12.Secondary Outcome
Title Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Hide Description Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.
Time Frame Baseline, Day 1, Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
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/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.
Overall Number of Participants Analyzed 12 12 12 12
Mean (Standard Deviation)
Unit of Measure: % change from Baseline
Day 1 Percent Change from Baseline Number Analyzed 11 participants 10 participants 12 participants 11 participants
9.9  (25.38) 2.7  (23.76) -8.0  (23.93) -6.7  (53.72)
Week 2 Percent Change from Baseline Number Analyzed 11 participants 10 participants 12 participants 12 participants
11.9  (20.52) 2.2  (26.44) -22.5  (15.27) -27.7  (26.50)
Week 4 Percent Change from Baseline Number Analyzed 11 participants 10 participants 12 participants 12 participants
3.6  (28.96) 6.8  (24.06) 2.7  (19.03) 14.5  (32.98)
13.Secondary Outcome
Title Pharmacokinetic (PK) Assessments (Tmax)
Hide Description Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. tmax= time when plasma concentration is at maximum.
Time Frame Day 1, Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication and have sufficient quantifiable plasma concentration data for PK analysis will be included in the PK population. [Note that the PK population will be determined by the study pharmacokineticist upon review of the concentration information for each subject in each cohort.
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/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.
Overall Number of Participants Analyzed 11 12 12 12
Mean (Standard Deviation)
Unit of Measure: hour
Day 1 3.6  (1.2) 4.6  (2.1) 2.5  (1.3) 2.7  (1.3)
Week 2 3.8  (1.80) 3.8  (2.2) 2.8  (1) 2.3  (0.86)
14.Secondary Outcome
Title Pharmacokinetic (PK) Assessments (AUC Inf)
Hide Description Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. AUC inf= Area under the concentration vs. time curve to time infinity.
Time Frame Day 1, Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication and have sufficient quantifiable plasma concentration data for PK analysis will be included in the PK population. [Note that the PK population will be determined by the study pharmacokineticist upon review of the concentration information for each subject in each cohort.
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/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.
Overall Number of Participants Analyzed 11 12 12 12
Mean (Standard Deviation)
Unit of Measure: [(hr)(ng)/mL]
Day 1 118  (48) 379  (117) 761  (352) 3279  (1693)
Week 2 164  (61) 544  (155) 1138  (467) 3606  (897)
15.Secondary Outcome
Title Pharmacokinetic (PK) Assessments CL (ml/hr/kg)
Hide Description Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay
Time Frame Day 1, Week 2
Hide Outcome Measure Data
Hide Analysis Population 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/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.
Overall Number of Participants Analyzed 11 12 12 12
Mean (Standard Deviation)
Unit of Measure: ml/hr/kg
Day 1 2459  (897) 2285  (1103) 2697  (1285) 2320  (1375)
Week 2 1828  (919) 1509  (482) 2047  (771) 1777  (476)
16.Secondary Outcome
Title Pharmacokinetic (PK) Assessments t(1/2)
Hide Description Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. t1/2= elimination half life.
Time Frame Day 1, Week 2
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who receive at least one dose of vamorolone study medication and have sufficient quantifiable plasma concentration data for PK analysis will be included in the PK population. [Note that the PK population will be determined by the study pharmacokineticist upon review of the concentration information for each subject in each cohort.
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/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.
Overall Number of Participants Analyzed 11 12 12 12
Mean (Standard Deviation)
Unit of Measure: hour
Day 1 2.1  (0.85) 1.8  (0.43) 1.9  (0.79) 1.9  (0.95)
Week 2 1.9  (0.96) 2.1  (0.8) 1.9  (1.02) 1.4  (0.35)
17.Secondary Outcome
Title Pharmacokinetic (PK) Assessments (Cmax)
Hide Description Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay
Time Frame Day 1, Week 2
Hide Outcome Measure Data
Hide Analysis Population 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/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.
Overall Number of Participants Analyzed 11 12 12 12
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 1 22.9  (13.4) 75.9  (25.9) 199  (111) 855.6  (471)
Week 2 32.2  (15.2) 124.7  (42.5) 252.2  (96) 970  (270)
18.Secondary Outcome
Title Metabolites in Safety Testing (MIST) Assessment
Hide Description A portion of each blood sample of the Week 2 (Day 14) pharmacokinetic assessment time points for the subjects receiving vamorolone 2 mg/kg/day was used for analysis of vamorolone metabolites.
Time Frame Week 2 (Day 14)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Dose Level Group 3
Hide Arm/Group Description:

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.
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: % of total drug related exposure
M1 34.42  (2.79)
M2 1.16  (0.06)
M3 1.21  (0.07)
M4 37.84  (3.78)
M5 2.73  (0.17)
Vamorolone 22.64  (0.69)
Time Frame Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
Adverse Event Reporting Description The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
 
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/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.
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%) 
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%)   0/12 (0.00%)   0/12 (0.00%)   0/12 (0.00%) 
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   7/12 (58.33%)   6/12 (50.00%)   8/12 (66.67%)   7/12 (58.33%) 
Gastrointestinal disorders         
Abdominal Pain † 1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Abdominal Pain Upper † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Constipation † 1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Diarrhoea † 1  1/12 (8.33%)  0/12 (0.00%)  1/12 (8.33%)  2/12 (16.67%) 
Faeces Discoloured † 1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Lip Swelling † 1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Vomiting † 1  2/12 (16.67%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
General disorders         
Pyrexia † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Infections and infestations         
Enterobiasis † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Gastroenteritis viral † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Lice Infestation † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Viral Upper Respiratory Tract Infection † 1  3/12 (25.00%)  2/12 (16.67%)  1/12 (8.33%)  0/12 (0.00%) 
Injury, poisoning and procedural complications         
Fall † 1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Musculoskeletal and connective tissue disorders         
Muscle Spasms † 1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Myalgia † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Pain in Extremity † 1  0/12 (0.00%)  3/12 (25.00%)  0/12 (0.00%)  0/12 (0.00%) 
Nervous system disorders         
Headache † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%) 
Lethargy † 1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Psychiatric disorders         
Agitation † 1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Emotional Disorder † 1  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%)  1/12 (8.33%) 
Insomnia † 1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Irritability † 1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
Restlessness † 1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Renal and urinary disorders         
Chromaturia † 1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Cough † 1  0/12 (0.00%)  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%) 
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         
Eczema † 1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Rash Macular † 1  1/12 (8.33%)  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%) 
Vascular disorders         
Flushing † 1  0/12 (0.00%)  0/12 (0.00%)  0/12 (0.00%)  1/12 (8.33%) 
1
Term from vocabulary, MedDRA 18.1
†
Indicates events were collected by systematic assessment
[Not Specified]
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
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
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