Trial Outcomes & Findings for An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) (NCT NCT02760277)

Last Updated: 2019-07-23

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

48 participants

Primary outcome timeframe

24 weeks

Results posted on

2019-07-23

Participant Flow

Participant milestones

Participant milestones
Measure	Dose Level Group 1 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.	Dose Level Group 2 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.	Dose Level Group 3 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.	Dose Level Group 4 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.
Vamorolone 0.25 mg/kg/Day STARTED	12	0	0	0
Vamorolone 0.25 mg/kg/Day COMPLETED	12	0	0	0
Vamorolone 0.25 mg/kg/Day NOT COMPLETED	0	0	0	0
0.75 mg/kg/Day STARTED	0	12	0	0
0.75 mg/kg/Day COMPLETED	0	12	0	0
0.75 mg/kg/Day NOT COMPLETED	0	0	0	0
2.0 mg/kg/Day STARTED	0	0	12	0
2.0 mg/kg/Day COMPLETED	0	0	12	0
2.0 mg/kg/Day NOT COMPLETED	0	0	0	0
6.0 mg/kg/Day STARTED	0	0	0	12
6.0 mg/kg/Day COMPLETED	0	0	0	12
6.0 mg/kg/Day NOT COMPLETED	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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 n=48 Participants Total of all reporting groups
Age, Customized Age	5.2 years STANDARD_DEVIATION 1.03 • n=5 Participants	4.8 years STANDARD_DEVIATION 0.83 • n=7 Participants	4.7 years STANDARD_DEVIATION 0.89 • n=5 Participants	4.8 years STANDARD_DEVIATION 0.75 • n=4 Participants	4.9 years STANDARD_DEVIATION 0.87 • n=21 Participants
Sex/Gender, Customized Male	12 Participants n=5 Participants	12 Participants n=7 Participants	12 Participants n=5 Participants	12 Participants n=4 Participants	48 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	3 Participants n=4 Participants	4 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	12 Participants n=5 Participants	11 Participants n=7 Participants	12 Participants n=5 Participants	9 Participants n=4 Participants	44 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Race (NIH/OMB) White	11 Participants n=5 Participants	10 Participants n=7 Participants	12 Participants n=5 Participants	12 Participants n=4 Participants	45 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Region of Enrollment Canada	3 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants	2 participants n=4 Participants	7 participants n=21 Participants
Region of Enrollment Sweden	0 participants n=5 Participants	4 participants n=7 Participants	0 participants n=5 Participants	0 participants n=4 Participants	4 participants n=21 Participants
Region of Enrollment United States	9 participants n=5 Participants	3 participants n=7 Participants	2 participants n=5 Participants	6 participants n=4 Participants	20 participants n=21 Participants
Region of Enrollment United Kingdom	0 participants n=5 Participants	0 participants n=7 Participants	4 participants n=5 Participants	2 participants n=4 Participants	6 participants n=21 Participants
Region of Enrollment Israel	0 participants n=5 Participants	3 participants n=7 Participants	2 participants n=5 Participants	0 participants n=4 Participants	5 participants n=21 Participants
Region of Enrollment Australia	0 participants n=5 Participants	2 participants n=7 Participants	2 participants n=5 Participants	2 participants n=4 Participants	6 participants n=21 Participants

PRIMARY outcome

Timeframe: 24 weeks

Population: 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.

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 Subjects with Any CTCAE Grade 3 or Higher TEAE	0 participants	0 participants	0 participants	2 participants
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 Discontinuation of Study Drug due to TEAE	0 participants	0 participants	0 participants	0 participants
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 Subjects with Any Serious TEAE	0 participants	1 participants	0 participants	2 participants
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 Death	0 participants	0 participants	0 participants	0 participants
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 Subjects with Any TEAE	10 participants	10 participants	11 participants	11 participants
Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03 Subjects with Any Drug Related TEAE	1 participants	2 participants	4 participants	5 participants

PRIMARY outcome

Timeframe: 24 weeks

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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 Number of Adverse Events as Assessed by CTCAE Version 4.03 Total Number of AEs	48 Events	44 Events	54 Events	73 Events
Total Number of Adverse Events as Assessed by CTCAE Version 4.03 Total Number of TEAEs	48 Events	44 Events	54 Events	72 Events

PRIMARY outcome

Timeframe: 002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

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

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity 002 Baseline	0.18 Rises/Second Standard Deviation 0.065	0.24 Rises/Second Standard Deviation 0.090	0.22 Rises/Second Standard Deviation 0.082	0.19 Rises/Second Standard Deviation 0.056
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity 003 Baseline	0.15 Rises/Second Standard Deviation 0.045	0.22 Rises/Second Standard Deviation 0.077	0.24 Rises/Second Standard Deviation 0.078	0.22 Rises/Second Standard Deviation 0.070
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity 003 Week 12	0.18 Rises/Second Standard Deviation 0.072	0.23 Rises/Second Standard Deviation 0.102	0.24 Rises/Second Standard Deviation 0.089	0.22 Rises/Second Standard Deviation 0.075
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity 003 Week 12 Change from 002 Baseline	-0.01 Rises/Second Standard Deviation 0.061	0.00 Rises/Second Standard Deviation 0.054	0.02 Rises/Second Standard Deviation 0.066	0.02 Rises/Second Standard Deviation 0.034
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity 003 Week 24	0.18 Rises/Second Standard Deviation 0.081	0.24 Rises/Second Standard Deviation 0.114	0.26 Rises/Second Standard Deviation 0.108	0.24 Rises/Second Standard Deviation 0.086
Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity 003 Week 24 Change from 002 Baseline	-0.01 Rises/Second Standard Deviation 0.066	0.00 Rises/Second Standard Deviation 0.062	0.05 Rises/Second Standard Deviation 0.061	0.04 Rises/Second Standard Deviation 0.045

PRIMARY outcome

Timeframe: 002 Baseline, 003 Week 12, Week 24

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.

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
BMI Z-score 002 Baseline	1.165 z score Standard Deviation 0.6219	0.703 z score Standard Deviation 1.0738	1.200 z score Standard Deviation 0.5325	0.695 z score Standard Deviation 0.7189
BMI Z-score 003 Week 12	1.103 z score Standard Deviation 0.6457	0.494 z score Standard Deviation 1.0680	1.261 z score Standard Deviation 0.3981	1.011 z score Standard Deviation 0.7034
BMI Z-score 003 Week 12 Change from 002 Baseline	-0.062 z score Standard Deviation 0.2438	-0.209 z score Standard Deviation 0.4078	0.062 z score Standard Deviation 0.3886	0.174 z score Standard Deviation 0.5826
BMI Z-score 003 Week 24	1.004 z score Standard Deviation 0.6381	0.493 z score Standard Deviation 1.1696	1.242 z score Standard Deviation 0.4596	1.330 z score Standard Deviation 0.5857
BMI Z-score 003 Week 24 Change from 002 Baseline	-0.161 z score Standard Deviation 0.3234	-0.210 z score Standard Deviation 0.3629	0.043 z score Standard Deviation 0.3849	0.493 z score Standard Deviation 0.6363

SECONDARY outcome

Timeframe: 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29

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.

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 16	5.26 % of HbA1c Standard Deviation 0.239	5.35 % of HbA1c Standard Deviation 0.238	5.26 % of HbA1c Standard Deviation 0.156	5.31 % of HbA1c Standard Deviation 0.270
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 8	5.18 % of HbA1c Standard Deviation 0.226	5.33 % of HbA1c Standard Deviation 0.250	5.28 % of HbA1c Standard Deviation 0.204	5.25 % of HbA1c Standard Deviation 0.216
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 8 Change from 002 Baseline	0.00 % of HbA1c Standard Deviation 0.128	0.12 % of HbA1c Standard Deviation 0.153	0.09 % of HbA1c Standard Deviation 0.198	0.00 % of HbA1c Standard Deviation 0.100
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 16 Change from 002 Baseline	0.08 % of HbA1c Standard Deviation 0.204	0.14 % of HbA1c Standard Deviation 0.225	0.07 % of HbA1c Standard Deviation 0.130	0.05 % of HbA1c Standard Deviation 0.113
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 24	5.15 % of HbA1c Standard Deviation 0.302	5.22 % of HbA1c Standard Deviation 0.221	5.13 % of HbA1c Standard Deviation 0.160	5.24 % of HbA1c Standard Deviation 0.254
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 24 Change from 002 Baseline	-0.10 % of HbA1c Standard Deviation 0.220	0.00 % of HbA1c Standard Deviation 0.186	-0.07 % of HbA1c Standard Deviation 0.130	-0.02 % of HbA1c Standard Deviation 0.154
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 26-29	5.07 % of HbA1c Standard Deviation 0.208	—	—	—
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 26-29 Change from 002 Baseline	-0.07 % of HbA1c Standard Deviation 0.208	—	—	—
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 002 Baseline	5.18 % of HbA1c Standard Deviation 0.260	5.22 % of HbA1c Standard Deviation 0.244	5.19 % of HbA1c Standard Deviation 0.124	5.23 % of HbA1c Standard Deviation 0.231

SECONDARY outcome

Timeframe: 002 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 8 % Change from 002 Baseline	0.06 % change Standard Deviation 2.470	2.28 % change Standard Deviation 3.008	1.79 % change Standard Deviation 3.864	0.02 % change Standard Deviation 1.927
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 16 % Change from 002 Baseline	1.70 % change Standard Deviation 3.989	2.72 % change Standard Deviation 4.457	1.30 % change Standard Deviation 2.505	1.03 % change Standard Deviation 2.14
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 24 % Change from 002 Baseline	-1.89 % change Standard Deviation 4.801	0.08 % change Standard Deviation 3.629	-1.27 % change Standard Deviation 2.525	-0.33 % change Standard Deviation 2.895
Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c 003 Week 26-29 % Change from 002 Baseline	-1.25 % change Standard Deviation 3.942	—	—	—

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 26-29	9.5 pg/mL Standard Deviation 7.26	—	—	—
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 26-29 Change from 002 Baseline	-7.8 pg/mL Standard Deviation 4.19	—	—	—
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 002 Baseline	18.3 pg/mL Standard Deviation 2.96	18.0 pg/mL Standard Deviation 6.88	21.1 pg/mL Standard Deviation 6.13	19.3 pg/mL Standard Deviation 8.67
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Baseline	15.9 pg/mL Standard Deviation 4.52	18.6 pg/mL Standard Deviation 4.56	18.2 pg/mL Standard Deviation 5.29	18.4 pg/mL Standard Deviation 9.73
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 8	13.0 pg/mL Standard Deviation 6.25	7.1 pg/mL Standard Deviation 5.84	7.8 pg/mL Standard Deviation 4.42	6.5 pg/mL Standard Deviation 5.23
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 8 Change from 002 Baseline	-5.3 pg/mL Standard Deviation 6.92	-10.5 pg/mL Standard Deviation 9.32	-13.3 pg/mL Standard Deviation 7.33	-13.5 pg/mL Standard Deviation 7.84
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 16	12.2 pg/mL Standard Deviation 4.93	9.0 pg/mL Standard Deviation 5.86	9.0 pg/mL Standard Deviation 14.18	9.0 pg/mL Standard Deviation 4.49
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 16 Change from 002 Baseline	-6.2 pg/mL Standard Deviation 6.34	-9.1 pg/mL Standard Deviation 9.89	-12.0 pg/mL Standard Deviation 15.42	-12.2 pg/mL Standard Deviation 8.16
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 24	19.8 pg/mL Standard Deviation 6.32	14.0 pg/mL Standard Deviation 3.88	15.7 pg/mL Standard Deviation 9.63	11.3 pg/mL Standard Deviation 7.52
Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH 003 Week 24 Change from 002 Baseline	0.6 pg/mL Standard Deviation 6.63	-4.0 pg/mL Standard Deviation 5.48	-5.4 pg/mL Standard Deviation 11.09	-6.3 pg/mL Standard Deviation 7.78

SECONDARY outcome

Timeframe: 002 Baseline, 003 Week 12, 003 Week 24

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose 002 Baseline	87.5 mg/dL Standard Deviation 9.44	88.9 mg/dL Standard Deviation 18.71	89.3 mg/dL Standard Deviation 7.91	92.3 mg/dL Standard Deviation 8.19
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose 003 Week 12	81.5 mg/dL Standard Deviation 5.61	81.7 mg/dL Standard Deviation 4.35	84.3 mg/dL Standard Deviation 8.13	86.5 mg/dL Standard Deviation 5.57
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose 003 Week 12 Change from 002 Baseline	-6.8 mg/dL Standard Deviation 8.29	-7.6 mg/dL Standard Deviation 19.22	-5.1 mg/dL Standard Deviation 9.01	-5.2 mg/dL Standard Deviation 9.21
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose 003 Week 24	80.8 mg/dL Standard Deviation 6.56	80.8 mg/dL Standard Deviation 4.08	81.3 mg/dL Standard Deviation 7.94	84.6 mg/dL Standard Deviation 6.53
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose 003 Week 24 Change from 002 Baseline	-6.3 mg/dL Standard Deviation 11.97	-9.0 mg/dL Standard Deviation 20.87	-8.1 mg/dL Standard Deviation 10.28	-7.8 mg/dL Standard Deviation 9.44

SECONDARY outcome

Timeframe: 002 Baseline, 003 Week 12, 003 Week 24

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin 002 Baseline	5.54 uIU/mL Standard Deviation 3.651	3.09 uIU/mL Standard Deviation 2.033	3.40 uIU/mL Standard Deviation 1.548	3.96 uIU/mL Standard Deviation 2.027
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin 003 Week 12	4.17 uIU/mL Standard Deviation 3.167	2.97 uIU/mL Standard Deviation 1.669	3.89 uIU/mL Standard Deviation 2.189	6.97 uIU/mL Standard Deviation 3.526
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin 003 Week 12 Change from 002 Baseline	-1.13 uIU/mL Standard Deviation 3.822	-0.14 uIU/mL Standard Deviation 1.756	0.49 uIU/mL Standard Deviation 2.592	2.97 uIU/mL Standard Deviation 2.277
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin 003 Week 24	4.23 uIU/mL Standard Deviation 2.560	3.12 uIU/mL Standard Deviation 1.788	4.82 uIU/mL Standard Deviation 3.393	7.21 uIU/mL Standard Deviation 2.374
Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin 003 Week 24 Change from 002 Baseline	-1.67 uIU/mL Standard Deviation 4.478	0.34 uIU/mL Standard Deviation 2.898	1.36 uIU/mL Standard Deviation 3.262	3.26 uIU/mL Standard Deviation 2.862

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 002 Baseline	37.94 ng/mL Standard Deviation 11.622	35.66 ng/mL Standard Deviation 6.800	41.17 ng/mL Standard Deviation 5.617	44.36 ng/mL Standard Deviation 5.979
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Baseline	39.20 ng/mL Standard Deviation 14.136	41.84 ng/mL Standard Deviation 8.552	47.91 ng/mL Standard Deviation 6.648	42.81 ng/mL Standard Deviation 10.851
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 8	36.21 ng/mL Standard Deviation 10.374	41.78 ng/mL Standard Deviation 13.856	44.45 ng/mL Standard Deviation 7.439	41.55 ng/mL Standard Deviation 5.446
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 8 Change from 002 Baseline	-1.60 ng/mL Standard Deviation 8.849	6.13 ng/mL Standard Deviation 10.440	3.28 ng/mL Standard Deviation 8.325	-2.01 ng/mL Standard Deviation 8.898
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 16	39.01 ng/mL Standard Deviation 9.620	42.23 ng/mL Standard Deviation 9.393	44.60 ng/mL Standard Deviation 9.534	39.39 ng/mL Standard Deviation 6.972
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 16 Change from 002 Baseline	1.07 ng/mL Standard Deviation 9.916	6.57 ng/mL Standard Deviation 6.709	3.43 ng/mL Standard Deviation 10.718	-4.17 ng/mL Standard Deviation 8.494
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 24	38.80 ng/mL Standard Deviation 6.292	51.41 ng/mL Standard Deviation 11.265	51.98 ng/mL Standard Deviation 9.372	49.08 ng/mL Standard Deviation 7.771
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 24 Change from 002 Baseline	-1.34 ng/mL Standard Deviation 11.289	15.75 ng/mL Standard Deviation 10.211	10.81 ng/mL Standard Deviation 7.542	5.29 ng/mL Standard Deviation 7.858
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 26-29	40.10 ng/mL Standard Deviation 18.729	—	—	—
Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin 003 Week 26-29 Change from 002 Baseline	-1.23 ng/mL Standard Deviation 17.943	—	—	—

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 002 Baseline	555.8 ng/mL Standard Deviation 184.72	480.7 ng/mL Standard Deviation 118.20	508.2 ng/mL Standard Deviation 94.36	511.5 ng/mL Standard Deviation 106.50
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Baseline	573.9 ng/mL Standard Deviation 251.02	489.3 ng/mL Standard Deviation 121.66	492.0 ng/mL Standard Deviation 81.92	566.3 ng/mL Standard Deviation 149.32
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 8	511.6 ng/mL Standard Deviation 190.94	459.8 ng/mL Standard Deviation 101.93	485.2 ng/mL Standard Deviation 105.12	402.7 ng/mL Standard Deviation 70.46
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 8 Change from 002 Baseline	-20.3 ng/mL Standard Deviation 120.32	-22.9 ng/mL Standard Deviation 128.79	-23.0 ng/mL Standard Deviation 96.84	-105.6 ng/mL Standard Deviation 121.07
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 16	481.9 ng/mL Standard Deviation 159.93	431.8 ng/mL Standard Deviation 81.25	455.7 ng/mL Standard Deviation 99.50	488.5 ng/mL Standard Deviation 130.11
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 16 Change from 002 Baseline	-73.8 ng/mL Standard Deviation 109.31	-42.4 ng/mL Standard Deviation 109.07	-52.5 ng/mL Standard Deviation 104.05	-19.8 ng/mL Standard Deviation 130.12
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 24	457.1 ng/mL Standard Deviation 129.21	471.1 ng/mL Standard Deviation 121.10	565.5 ng/mL Standard Deviation 158.89	526.2 ng/mL Standard Deviation 130.18
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 24 Change from 002 Baseline	-30.8 ng/mL Standard Deviation 113.64	2.1 ng/mL Standard Deviation 165.16	57.3 ng/mL Standard Deviation 150.36	8.7 ng/mL Standard Deviation 88.95
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 26-29 Change from 002 Baseline	-152.0 ng/mL Standard Deviation 331.46	—	—	—
Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP 003 Week 26-29	619.0 ng/mL Standard Deviation 379.07	—	—	—

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 002 Baseline	871.0 pg/mL Standard Deviation 160.85	935.8 pg/mL Standard Deviation 286.50	936.8 pg/mL Standard Deviation 256.25	889.3 pg/mL Standard Deviation 186.68
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Baseline	915.9 pg/mL Standard Deviation 263.13	964.4 pg/mL Standard Deviation 319.26	949.8 pg/mL Standard Deviation 303.76	989.2 pg/mL Standard Deviation 216.29
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 8	897.1 pg/mL Standard Deviation 365.45	933.3 pg/mL Standard Deviation 330.20	928.3 pg/mL Standard Deviation 333.11	825.5 pg/mL Standard Deviation 164.36
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 8 Change from 002 Baseline	26.1 pg/mL Standard Deviation 368.76	-31.6 pg/mL Standard Deviation 236.34	-8.5 pg/mL Standard Deviation 248.82	-59.6 pg/mL Standard Deviation 259.08
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 16	885.4 pg/mL Standard Deviation 261.53	912.8 pg/mL Standard Deviation 305.08	939.8 pg/mL Standard Deviation 157.17	953.7 pg/mL Standard Deviation 199.53
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 16 Change from 002 Baseline	-2.6 pg/mL Standard Deviation 304.50	-46.3 pg/mL Standard Deviation 321.06	3.0 pg/mL Standard Deviation 244.71	102.3 pg/mL Standard Deviation 230.44
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 24	1109.3 pg/mL Standard Deviation 287.92	1235.6 pg/mL Standard Deviation 295.79	1248.7 pg/mL Standard Deviation 308.90	1237.0 pg/mL Standard Deviation 277.20
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 24 Change from 002 Baseline	212.3 pg/mL Standard Deviation 318.86	295.6 pg/mL Standard Deviation 357.93	346.5 pg/mL Standard Deviation 327.16	321.4 pg/mL Standard Deviation 264.65
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 26-29	1059.3 pg/mL Standard Deviation 536.26	—	—	—
Serum Pharmacodynamics Biomarkers Measured by Levels of CTX 003 Week 26-29 Change from 002 Baseline	569.5 pg/mL Standard Deviation 28.99	—	—	—

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity 002 Baseline	0.20 tasks/ second Standard Deviation 0.054	0.29 tasks/ second Standard Deviation 0.147	0.29 tasks/ second Standard Deviation 0.164	0.24 tasks/ second Standard Deviation 0.086
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity 003 Baseline	0.20 tasks/ second Standard Deviation 0.065	0.29 tasks/ second Standard Deviation 0.168	0.31 tasks/ second Standard Deviation 0.144	0.25 tasks/ second Standard Deviation 0.082
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity 003 Week 12	0.21 tasks/ second Standard Deviation 0.064	0.34 tasks/ second Standard Deviation 0.238	0.31 tasks/ second Standard Deviation 0.157	0.26 tasks/ second Standard Deviation 0.095
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity 003 Week 24 Change from 002 Baseline	0.00 tasks/ second Standard Deviation 0.076	0.01 tasks/ second Standard Deviation 0.066	0.04 tasks/ second Standard Deviation 0.090	0.05 tasks/ second Standard Deviation 0.061
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity 003 Week 12 Change from 002 Baseline	0.01 tasks/ second Standard Deviation 0.044	0.05 tasks/ second Standard Deviation 0.115	0.02 tasks/ second Standard Deviation 0.107	0.02 tasks/ second Standard Deviation 0.051
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity 003 Week 24	0.20 tasks/ second Standard Deviation 0.071	0.30 tasks/ second Standard Deviation 0.166	0.34 tasks/ second Standard Deviation 0.148	0.29 tasks/ second Standard Deviation 0.097

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity 002 Baseline	1.60 meters/ second Standard Deviation 0.312	1.77 meters/ second Standard Deviation 0.367	1.84 meters/ second Standard Deviation 0.347	1.64 meters/ second Standard Deviation 0.279
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity 003 Baseline	1.57 meters/ second Standard Deviation 0.371	1.78 meters/ second Standard Deviation 0.414	1.86 meters/ second Standard Deviation 0.418	1.72 meters/ second Standard Deviation 0.295
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity 003 Week 12	1.54 meters/ second Standard Deviation 0.306	1.77 meters/ second Standard Deviation 0.550	1.97 meters/ second Standard Deviation 0.503	1.88 meters/ second Standard Deviation 0.341
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity 003 Week 12 Change from 002 Baseline	-0.06 meters/ second Standard Deviation 0.261	0.00 meters/ second Standard Deviation 0.307	0.13 meters/ second Standard Deviation 0.316	0.26 meters/ second Standard Deviation 0.297
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity 003 Week 24	1.55 meters/ second Standard Deviation 0.384	1.84 meters/ second Standard Deviation 0.486	1.90 meters/ second Standard Deviation 0.321	1.89 meters/ second Standard Deviation 0.378
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity 003 Week 24 Change from 002 Baseline	-0.05 meters/ second Standard Deviation 0.311	0.06 meters/ second Standard Deviation 0.210	0.06 meters/ second Standard Deviation 0.210	0.27 meters/ second Standard Deviation 0.254

SECONDARY outcome

Timeframe: 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)

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.

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA) 002 Baseline	19.0 scores on a scale Standard Deviation 5.13	20.5 scores on a scale Standard Deviation 5.58	20.0 scores on a scale Standard Deviation 4.95	19.7 scores on a scale Standard Deviation 4.94
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA) 003 Baseline	20.1 scores on a scale Standard Deviation 7.30	20.8 scores on a scale Standard Deviation 5.66	21.7 scores on a scale Standard Deviation 3.87	20.4 scores on a scale Standard Deviation 4.01
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA) 003 Week 12	19.3 scores on a scale Standard Deviation 5.60	21.2 scores on a scale Standard Deviation 6.45	21.0 scores on a scale Standard Deviation 5.13	20.4 scores on a scale Standard Deviation 5.41
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA) 003 Week 12 Change from 002 Baseline	0.3 scores on a scale Standard Deviation 2.06	0.7 scores on a scale Standard Deviation 2.71	1.0 scores on a scale Standard Deviation 2.56	0.5 scores on a scale Standard Deviation 2.38
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA) 003 Week 24	19.8 scores on a scale Standard Deviation 7.09	21.6 scores on a scale Standard Deviation 7.23	22.3 scores on a scale Standard Deviation 3.80	22.3 scores on a scale Standard Deviation 5.76
Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA) 003 Week 24 Change from 002 Baseline	0.8 scores on a scale Standard Deviation 2.83	1.1 scores on a scale Standard Deviation 2.94	2.3 scores on a scale Standard Deviation 1.78	2.5 scores on a scale Standard Deviation 2.62

SECONDARY outcome

Timeframe: 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)

Outcome measures

Outcome measures
Measure	Dose Level Group 1 n=12 Participants 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.	Dose Level Group 2 n=12 Participants 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.	Dose Level Group 3 n=12 Participants 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.	Dose Level Group 4 n=12 Participants 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.
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters 002 Baseline	316.2 Meters Standard Deviation 59.47	331.5 Meters Standard Deviation 52.76	353.9 Meters Standard Deviation 65.40	336.8 Meters Standard Deviation 63.18
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters 003 Baseline	294.3 Meters Standard Deviation 60.62	332.2 Meters Standard Deviation 56.83	341.1 Meters Standard Deviation 49.42	335.1 Meters Standard Deviation 80.13
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters 003 Week 12	312.9 Meters Standard Deviation 60.93	358.7 Meters Standard Deviation 71.47	393.7 Meters Standard Deviation 59.72	369.9 Meters Standard Deviation 69.47
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters 003 Week 12 Change from 002 Baseline	6.0 Meters Standard Deviation 28.81	20.8 Meters Standard Deviation 38.09	39.8 Meters Standard Deviation 35.61	27.6 Meters Standard Deviation 42.00
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters 003 Week 24	306.2 Meters Standard Deviation 68.08	350.4 Meters Standard Deviation 64.23	383.1 Meters Standard Deviation 63.38	372.6 Meters Standard Deviation 69.12
Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters 003 Week 24 Change from 002 Baseline	-11.6 Meters Standard Deviation 29.45	18.9 Meters Standard Deviation 41.08	29.2 Meters Standard Deviation 35.91	43.9 Meters Standard Deviation 43.72

Adverse Events

Dose Level Group 1

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Dose Level Group 2

Serious events: 1 serious events

Other events: 10 other events

Deaths: 0 deaths

Dose Level Group 3

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Dose Level Group 4

Serious events: 2 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Dose Level Group 1 n=12 participants at risk 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.	Dose Level Group 2 n=12 participants at risk 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.	Dose Level Group 3 n=12 participants at risk 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.	Dose Level Group 4 n=12 participants at risk 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.
Infections and infestations Pneumonia	0.00% 0/12 • 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.	8.3% 1/12 • 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.	0.00% 0/12 • 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.	0.00% 0/12 • 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.
Metabolism and nutrition disorders Dehydration	0.00% 0/12 • 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.	0.00% 0/12 • 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.	0.00% 0/12 • 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.	8.3% 1/12 • 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.
Reproductive system and breast disorders Testicular Torsion	0.00% 0/12 • 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.	0.00% 0/12 • 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.	0.00% 0/12 • 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.	8.3% 1/12 • 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.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/12 • 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.	0.00% 0/12 • 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.	0.00% 0/12 • 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.	8.3% 1/12 • 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.

Other adverse events

Additional Information

Dr. Eric P. Hoffman

ReveraGen Biopharma Inc.

Phone: 240-672-0295

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place