A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT03439670

Recruitment Status : Completed

First Posted : February 20, 2018

Results First Posted : July 13, 2022

Last Update Posted : March 9, 2023

View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborators:

European Union

Cooperative International Neuromuscular Research Group

Newcastle University

University of Pittsburgh

Information provided by (Responsible Party):

ReveraGen BioPharma, Inc.

Study Type	Interventional
Study Design	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition	Duchenne Muscular Dystrophy
Interventions	Drug: Vamorolone Drug: Prednisone Other: Placebo
Enrollment	121

Participant Flow

Recruitment Details
Pre-assignment Details


Arm/Group Title	Treatment Group 1	Treatment Group 2	Treatment Group 3	Treatment Group 4	Treatment Group 5	Treatment Group 6
Arm/Group Description	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...
Arm/Group Description	Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.
Period Title: Overall Study
Started	30	30	15	16	15	15
Completed	28	26	15	15	14	14
Not Completed	2	4	0	1	1	1

Baseline Characteristics

Arm/Group Title		Treatment Group 1	Treatment Group 2	Treatment Group 3	Treatment Group 4	Treatment Group 5	Treatment Group 6	Total
Arm/Group Description		Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Total of all reporting groups
Arm/Group Description		Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.	Total of all reporting groups
Overall Number of Baseline Participants		28	28	15	15	14	14	114
Baseline Analysis Population Description		...
Baseline Analysis Population Description		The baseline analysis population refers to the baseline visit per protocol while the Participant Flow refers to all randomized participants.
Age, Categorical Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	28 participants	28 participants	15 participants	15 participants	14 participants	14 participants	114 participants
	<=18 years	28 100.0%	28 100.0%	15 100.0%	15 100.0%	14 100.0%	14 100.0%	114 100.0%
	Between 18 and 65 years	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	>=65 years	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	28 participants	28 participants	15 participants	15 participants	14 participants	14 participants	114 participants
	Female	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Male	28 100.0%	28 100.0%	15 100.0%	15 100.0%	14 100.0%	14 100.0%	114 100.0%
Race (NIH/OMB) Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	28 participants	28 participants	15 participants	15 participants	14 participants	14 participants	114 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%	1 6.7%	0 0.0%	0 0.0%	1 0.9%
	Asian	4 14.3%	3 10.7%	2 13.3%	1 6.7%	1 7.1%	1 7.1%	12 10.5%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Black or African American	1 3.6%	1 3.6%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	2 1.8%
	White	23 82.1%	23 82.1%	12 80.0%	12 80.0%	11 78.6%	13 92.9%	94 82.5%
	More than one race	0 0.0%	1 3.6%	1 6.7%	1 6.7%	1 7.1%	0 0.0%	4 3.5%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%	0 0.0%	1 7.1%	0 0.0%	1 0.9%
TTSTAND (seconds) Mean (Standard Deviation) Unit of measure: Seconds
	Number Analyzed	28 participants	28 participants	15 participants	15 participants	14 participants	14 participants	114 participants
		6.01 (2.406)	5.97 (1.991)	5.36 (1.948)	4.53 (0.808)	5.51 (1.578)	5.58 (2.288)	5.560 (1.9498)

Outcome Measures

1.Primary Outcome


Title	Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline
Description	Vamorolone at 6.0mg/kg/day vs. placebo group in change from baseline t...
Description	Vamorolone at 6.0mg/kg/day vs. placebo group in change from baseline to the Week 24 assessment
Time Frame	24 weeks

Outcome Measure Data

Analysis Population Description

Per the protocol, the primary clinical efficacy endpoint was "comparison of the vamorolone 6.0 mg/kg/day dose level group versus the placebo group in change from baseline to the Week 24 assessment."


Arm/Group Title	Treatment Group 1	Treatment Group 2
Arm/Group Description:	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...
Arm/Group Description:	Patients enrolled in Treatment Group 1 (placebo comparator group) will receive placebo for 24 weeks.	Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks
Overall Number of Participants Analyzed	28	27
Mean (Standard Deviation) Unit of Measure: Rises/Seconds
	-.007 (0.0628)	0.054 (0.0666)

Adverse Events

Time Frame	Up to 48 weeks
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Treatment Group 1	Treatment Group 2	Treatment Group 3	Treatment Group 4	Treatment Group 5	Treatment Group 6
Arm/Group Description	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...	Patients enrolled in Treatment Grou...
Arm/Group Description	Patients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 2 (experimental group) will receive vamorolone 6.0 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 2.0mg/kg/day for 20 weeks	Patients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by vamorolone 6.0mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 5 will receive placebo for 24 weeks followed by vamorolone 2mg/kg/day for 20 weeks.	Patients enrolled in Treatment Group 6 will receive placebo for 24 weeks followed by vamorolone 6mg/kg/day for 20 weeks.
All-Cause Mortality
	Treatment Group 1	Treatment Group 2	Treatment Group 3	Treatment Group 4	Treatment Group 5	Treatment Group 6
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Serious Adverse Events Serious Adverse Events
	Treatment Group 1	Treatment Group 2	Treatment Group 3	Treatment Group 4	Treatment Group 5	Treatment Group 6
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	1/28 (3.57%)	2/28 (7.14%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Infections and infestations
Gastroenteritis viral/viral gasteroenteritis ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Appendicitis ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Respiratory, thoracic and mediastinal disorders
Asthma ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
1 Term from vocabulary, MedDRA † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Treatment Group 1	Treatment Group 2	Treatment Group 3	Treatment Group 4	Treatment Group 5	Treatment Group 6
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	26/28 (92.86%)	26/28 (92.86%)	15/15 (100.00%)	13/15 (86.67%)	12/14 (85.71%)	14/14 (100.00%)
Blood and lymphatic system disorders
Leukopenia ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Ear and labyrinth disorders
Ear pain ^† 1	0/28 (0.00%)	1/28 (3.57%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Tympanic membrane perforation ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Endocrine disorders
Cushingoid ^† 1	4/28 (14.29%)	9/28 (32.14%)	4/15 (26.67%)	4/15 (26.67%)	1/14 (7.14%)	1/14 (7.14%)
Growth hormone deficiency ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Gastrointestinal disorders
Abdominal Pain ^† 1	3/28 (10.71%)	3/28 (10.71%)	2/15 (13.33%)	1/15 (6.67%)	2/14 (14.29%)	0/14 (0.00%)
Abdominal Pain upper ^† 1	1/28 (3.57%)	3/28 (10.71%)	3/15 (20.00%)	2/15 (13.33%)	1/14 (7.14%)	1/14 (7.14%)
Constipation ^† 1	3/28 (10.71%)	3/28 (10.71%)	0/15 (0.00%)	2/15 (13.33%)	1/14 (7.14%)	1/14 (7.14%)
Diarrhoea ^† 1	3/28 (10.71%)	5/28 (17.86%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	1/14 (7.14%)
Vomiting ^† 1	6/28 (21.43%)	6/28 (21.43%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	3/14 (21.43%)
Mouth Ulceration ^† 1	0/28 (0.00%)	2/28 (7.14%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Toothache ^† 1	1/28 (3.57%)	1/28 (3.57%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Diarrhoea haemorrhagic ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Flatulance ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
General disorders
Pyrexia ^† 1	7/28 (25.00%)	3/28 (10.71%)	3/15 (20.00%)	1/15 (6.67%)	3/14 (21.43%)	3/14 (21.43%)
Asthenia ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Fatigue ^† 1	1/28 (3.57%)	0/28 (0.00%)	2/15 (13.33%)	2/15 (13.33%)	0/14 (0.00%)	0/14 (0.00%)
Impaired healing ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Thirst ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Hepatobiliary disorders
Hepatomegaly ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Infections and infestations
Gasteroenteritis ^† 1	0/28 (0.00%)	1/28 (3.57%)	2/15 (13.33%)	0/15 (0.00%)	1/14 (7.14%)	1/14 (7.14%)
Nasopharyngitis ^† 1	2/28 (7.14%)	4/28 (14.29%)	3/15 (20.00%)	2/15 (13.33%)	1/14 (7.14%)	2/14 (14.29%)
Upper respiratory tract infection ^† 1	10/28 (35.71%)	4/28 (14.29%)	2/15 (13.33%)	3/15 (20.00%)	3/14 (21.43%)	2/14 (14.29%)
Rhinitis ^† 1	2/28 (7.14%)	4/28 (14.29%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	2/14 (14.29%)
Enterobiasis ^† 1	1/28 (3.57%)	2/28 (7.14%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Conjunctivitis ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Ear Infection ^† 1	1/28 (3.57%)	1/28 (3.57%)	0/15 (0.00%)	1/15 (6.67%)	1/14 (7.14%)	1/14 (7.14%)
Gastroenteritis ^† 1	0/28 (0.00%)	1/28 (3.57%)	2/15 (13.33%)	0/15 (0.00%)	1/14 (7.14%)	1/14 (7.14%)
Tonsilitis ^† 1	1/28 (3.57%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	2/14 (14.29%)	0/14 (0.00%)
Viral Infection ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
COVID-19 ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	1/14 (7.14%)
Cystitis ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Fungal Infection ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Gastroenteritis viral/viral gasteroenteritis ^† 1	3/28 (10.71%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Hand-foot-and-mouth disease ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Impetigo ^† 1	2/28 (7.14%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Influenza ^† 1	1/28 (3.57%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	2/14 (14.29%)	0/14 (0.00%)
Moluscum contagiosum ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Otitis media bacterial ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Pharyngitis streptococcal ^† 1	1/28 (3.57%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Pneumonia ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Respiratory track infection viral ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Steptococcal infection ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Tooth infection ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Viral upper respiratory tract infection ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Injury, poisoning and procedural complications
Arthropod bite ^† 1	1/28 (3.57%)	2/28 (7.14%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	1/14 (7.14%)
Contusion ^† 1	2/28 (7.14%)	1/28 (3.57%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Fall ^† 1	2/28 (7.14%)	4/28 (14.29%)	2/15 (13.33%)	3/15 (20.00%)	0/14 (0.00%)	1/14 (7.14%)
Ligament strain ^† 1	1/28 (3.57%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Back injury ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Muscle strain ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Vaccination complication ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Investigations
Blood uric acid increased ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	2/15 (13.33%)	0/14 (0.00%)	1/14 (7.14%)
Protein Urine Present ^† 1	1/28 (3.57%)	0/28 (0.00%)	1/15 (6.67%)	3/15 (20.00%)	0/14 (0.00%)	0/14 (0.00%)
Weight increased ^† 1	1/28 (3.57%)	3/28 (10.71%)	1/15 (6.67%)	1/15 (6.67%)	1/14 (7.14%)	1/14 (7.14%)
Cortisol decreased ^† 1	1/28 (3.57%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Bacterial test postive ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Blood pressure increased ^† 1	1/28 (3.57%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Blood triglycerides increased ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Electrocardiogram abnormal ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Glycosylated haemoglobin increased ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Lipase decreased ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Urine analysis abnormal ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Weight decreased ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Metabolism and nutrition disorders
Increased appetite ^† 1	1/28 (3.57%)	2/28 (7.14%)	0/15 (0.00%)	1/15 (6.67%)	1/14 (7.14%)	0/14 (0.00%)
Vitamin D Deficiency ^† 1	2/28 (7.14%)	3/28 (10.71%)	1/15 (6.67%)	2/15 (13.33%)	0/14 (0.00%)	0/14 (0.00%)
Decreased appetite ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Hypertriglyceridaemia ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Overweight ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Polydipsia ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Musculoskeletal and connective tissue disorders
Pain in extremity ^† 1	2/28 (7.14%)	3/28 (10.71%)	4/15 (26.67%)	4/15 (26.67%)	2/14 (14.29%)	0/14 (0.00%)
Back pain ^† 1	1/28 (3.57%)	2/28 (7.14%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	1/14 (7.14%)
Athralgia ^† 1	1/28 (3.57%)	1/28 (3.57%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Muscle spasms ^† 1	0/28 (0.00%)	1/28 (3.57%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Myalgia ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Costrochondritis ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Joint contracture ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Joint swelling ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Muscle atrophy ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Muscular weakness ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Musculoskeletal pain ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Musculoskeletal stiffness ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Vertebral wedging ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Agitation ^† 1	0/28 (0.00%)	1/28 (3.57%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Nervous system disorders
Headache ^† 1	3/28 (10.71%)	2/28 (7.14%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	3/14 (21.43%)
Psychomotor hyperactivity ^† 1	2/28 (7.14%)	0/28 (0.00%)	1/15 (6.67%)	2/15 (13.33%)	0/14 (0.00%)	0/14 (0.00%)
Dizziness ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Poor quality sleep ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Seizure ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Syncope ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Psychiatric disorders
Abnormal behaviour ^† 1	2/28 (7.14%)	1/28 (3.57%)	2/15 (13.33%)	0/15 (0.00%)	1/14 (7.14%)	1/14 (7.14%)
Aggression ^† 1	2/28 (7.14%)	1/28 (3.57%)	0/15 (0.00%)	2/15 (13.33%)	1/14 (7.14%)	0/14 (0.00%)
Irritability ^† 1	0/28 (0.00%)	3/28 (10.71%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Sleep disorder ^† 1	0/28 (0.00%)	1/28 (3.57%)	1/15 (6.67%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Anger ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Dysphemia ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Emotional disorder ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Enuresis ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Initial insomnia ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Insomnia ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Mood swings ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	1/14 (7.14%)	0/14 (0.00%)
Oppositional defiant disorder ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Personality change ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Trichotillomania ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Epistaxis ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	1/15 (6.67%)	1/14 (7.14%)	0/14 (0.00%)
Night sweats ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Renal and urinary disorders
Chromaturia ^† 1	0/28 (0.00%)	1/28 (3.57%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Proteinuria ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Respiratory, thoracic and mediastinal disorders
Cough ^† 1	5/28 (17.86%)	3/28 (10.71%)	2/15 (13.33%)	4/15 (26.67%)	0/14 (0.00%)	2/14 (14.29%)
Nasal congestion ^† 1	0/28 (0.00%)	2/28 (7.14%)	1/15 (6.67%)	2/15 (13.33%)	0/14 (0.00%)	0/14 (0.00%)
Rhinorrhoea ^† 1	1/28 (3.57%)	1/28 (3.57%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	1/14 (7.14%)
Oropharyngeal pain ^† 1	1/28 (3.57%)	0/28 (0.00%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	1/14 (7.14%)
Skin and subcutaneous tissue disorders
Hypertrichosis ^† 1	1/28 (3.57%)	3/28 (10.71%)	1/15 (6.67%)	1/15 (6.67%)	0/14 (0.00%)	1/14 (7.14%)
Rash ^† 1	2/28 (7.14%)	2/28 (7.14%)	2/15 (13.33%)	2/15 (13.33%)	1/14 (7.14%)	2/14 (14.29%)
Eczema ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Perioral dermatitis ^† 1	0/28 (0.00%)	1/28 (3.57%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Pruritis ^† 1	0/28 (0.00%)	1/28 (3.57%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Dry skin ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Erythema ^† 1	1/28 (3.57%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	1/14 (7.14%)	0/14 (0.00%)
Rash macropapular ^† 1	0/28 (0.00%)	0/28 (0.00%)	1/15 (6.67%)	0/15 (0.00%)	0/14 (0.00%)	0/14 (0.00%)
Urticaria ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	0/15 (0.00%)	0/14 (0.00%)	1/14 (7.14%)
Viral rash ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Vascular disorders
Hot flush ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
Peripheral coldness ^† 1	0/28 (0.00%)	0/28 (0.00%)	0/15 (0.00%)	1/15 (6.67%)	0/14 (0.00%)	0/14 (0.00%)
1 Term from vocabulary, MedDRA † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

Layout table for Results Point of Contact information

Name/Title:	Dr. Eric Hoffman
Organization:	ReveraGen BioPharma Inc.
Phone:	301-762-7980
EMail:	ericphoffman@gmail.com

Publications of Results:

Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans N, Haberlova J, Straub V, Mengle-Gaw LJ, Schwartz BD, Harper AD, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster R, McMillan HJ, Kuntz NL, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez-Padilla JJ, Nascimento-Osorio A, Niks EH, de Groot IJM, Katsalouli M, James MK, van den Anker J, Damsker JM, Ahmet A, Ward LM, Jaros M, Shale P, Dang UJ, Hoffman EP. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Neurol. 2022 Oct 1;79(10):1005-1014. doi: 10.1001/jamaneurol.2022.2480.

Other Publications:

Mah JK, Korngut L, Dykeman J, Day L, Pringsheim T, Jette N. A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 2014 Jun;24(6):482-91. doi: 10.1016/j.nmd.2014.03.008. Epub 2014 Mar 22.

Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 Dec 24;51(6):919-28. doi: 10.1016/0092-8674(87)90579-4.

Evans NP, Misyak SA, Robertson JL, Bassaganya-Riera J, Grange RW. Immune-mediated mechanisms potentially regulate the disease time-course of duchenne muscular dystrophy and provide targets for therapeutic intervention. PM R. 2009 Aug;1(8):755-68. doi: 10.1016/j.pmrj.2009.04.010.

Kumar A, Boriek AM. Mechanical stress activates the nuclear factor-kappaB pathway in skeletal muscle fibers: a possible role in Duchenne muscular dystrophy. FASEB J. 2003 Mar;17(3):386-96. doi: 10.1096/fj.02-0542com.

Acharyya S, Villalta SA, Bakkar N, Bupha-Intr T, Janssen PM, Carathers M, Li ZW, Beg AA, Ghosh S, Sahenk Z, Weinstein M, Gardner KL, Rafael-Fortney JA, Karin M, Tidball JG, Baldwin AS, Guttridge DC. Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy. J Clin Invest. 2007 Apr;117(4):889-901. doi: 10.1172/JCI30556. Epub 2007 Mar 22.

Dogra C, Changotra H, Wergedal JE, Kumar A. Regulation of phosphatidylinositol 3-kinase (PI3K)/Akt and nuclear factor-kappa B signaling pathways in dystrophin-deficient skeletal muscle in response to mechanical stretch. J Cell Physiol. 2006 Sep;208(3):575-85. doi: 10.1002/jcp.20696.

Pahl HL. Activators and target genes of Rel/NF-kappaB transcription factors. Oncogene. 1999 Nov 22;18(49):6853-66. doi: 10.1038/sj.onc.1203239.

Spencer MJ, Montecino-Rodriguez E, Dorshkind K, Tidball JG. Helper (CD4(+)) and cytotoxic (CD8(+)) T cells promote the pathology of dystrophin-deficient muscle. Clin Immunol. 2001 Feb;98(2):235-43. doi: 10.1006/clim.2000.4966.

Spencer MJ, Tidball JG. Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord. 2001 Sep;11(6-7):556-64. doi: 10.1016/s0960-8966(01)00198-5.

Malik V, Rodino-Klapac LR, Mendell JR. Emerging drugs for Duchenne muscular dystrophy. Expert Opin Emerg Drugs. 2012 Jun;17(2):261-77. doi: 10.1517/14728214.2012.691965.

Reeves EKM, Hoffman EP, Nagaraju K, Damsker JM, McCall JM. VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem. 2013 Apr 15;21(8):2241-2249. doi: 10.1016/j.bmc.2013.02.009. Epub 2013 Feb 18. Erratum In: Bioorg Med Chem. 2015 Apr 1;23(7):1664.

Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27.

Pane M, Mazzone ES, Sivo S, Sormani MP, Messina S, D'Amico A, Carlesi A, Vita G, Fanelli L, Berardinelli A, Torrente Y, Lanzillotta V, Viggiano E, D Ambrosio P, Cavallaro F, Frosini S, Barp A, Bonfiglio S, Scalise R, De Sanctis R, Rolle E, Graziano A, Magri F, Palermo C, Rossi F, Donati MA, Sacchini M, Arnoldi MT, Baranello G, Mongini T, Pini A, Battini R, Pegoraro E, Previtali S, Bruno C, Politano L, Comi GP, Bertini E, Mercuri E. Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes. PLoS One. 2014 Oct 1;9(10):e108205. doi: 10.1371/journal.pone.0108205. eCollection 2014. Erratum In: PLoS One. 2015;10(3):e0121882. PLoS One. 2015;10(12):e0144079.

Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008 Jan 23;(1):CD003725. doi: 10.1002/14651858.CD003725.pub3.

Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L, Quinn J, Tatem K, Jordan S, Dadgar S, Rodriguez OC, Albanese C, Calhoun M, Gordish-Dressman H, Jaiswal JK, Connor EM, McCall JM, Hoffman EP, Reeves EK, Nagaraju K. VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med. 2013 Oct;5(10):1569-85. doi: 10.1002/emmm.201302621. Epub 2013 Sep 9.

Damsker JM, Dillingham BC, Rose MC, Balsley MA, Heier CR, Watson AM, Stemmy EJ, Jurjus RA, Huynh T, Tatem K, Uaesoontrachoon K, Berry DM, Benton AS, Freishtat RJ, Hoffman EP, McCall JM, Gordish-Dressman H, Constant SL, Reeves EK, Nagaraju K. VBP15, a glucocorticoid analogue, is effective at reducing allergic lung inflammation in mice. PLoS One. 2013 May 7;8(5):e63871. doi: 10.1371/journal.pone.0063871. Print 2013.

Dillingham BC, Knoblach SM, Many GM, Harmon BT, Mullen AM, Heier CR, Bello L, McCall JM, Hoffman EP, Connor EM, Nagaraju K, Reeves EKM, Damsker JM. VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis. Cell Mol Neurobiol. 2015 Apr;35(3):377-387. doi: 10.1007/s10571-014-0133-y. Epub 2014 Nov 13.

Dadgar S, Wang Z, Johnston H, Kesari A, Nagaraju K, Chen YW, Hill DA, Partridge TA, Giri M, Freishtat RJ, Nazarian J, Xuan J, Wang Y, Hoffman EP. Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. J Cell Biol. 2014 Oct 13;207(1):139-58. doi: 10.1083/jcb.201402079.

Raman SV, Hor KN, Mazur W, Halnon NJ, Kissel JT, He X, Tran T, Smart S, McCarthy B, Taylor MD, Jefferies JL, Rafael-Fortney JA, Lowe J, Roble SL, Cripe LH. Eplerenone for early cardiomyopathy in Duchenne muscular dystrophy: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2015 Feb;14(2):153-61. doi: 10.1016/S1474-4422(14)70318-7. Epub 2014 Dec 30. Erratum In: Lancet Neurol. 2015 Feb;14(2):135.

Avioli LV. Glucocorticoid effects on statural growth. Br J Rheumatol. 1993 May;32 Suppl 2:27-30. doi: 10.1093/rheumatology/32.suppl_2.27.

Wolthers OD, Pedersen S. Short term linear growth in asthmatic children during treatment with prednisolone. BMJ. 1990 Jul 21;301(6744):145-8. doi: 10.1136/bmj.301.6744.145.

Bircan Z, Soran M, Yildirim I, Dogan M, Sahin A, Bilici A, Danaci M. The effect of alternate-day low dose prednisolone on bone age in children with steroid dependent nephrotic syndrome. Int Urol Nephrol. 1997;29(3):357-61. doi: 10.1007/BF02550936.

Manolagas SC, Weinstein RS. New developments in the pathogenesis and treatment of steroid-induced osteoporosis. J Bone Miner Res. 1999 Jul;14(7):1061-6. doi: 10.1359/jbmr.1999.14.7.1061. No abstract available.

Sali A, Guerron AD, Gordish-Dressman H, Spurney CF, Iantorno M, Hoffman EP, Nagaraju K. Glucocorticoid-treated mice are an inappropriate positive control for long-term preclinical studies in the mdx mouse. PLoS One. 2012;7(4):e34204. doi: 10.1371/journal.pone.0034204. Epub 2012 Apr 3.

Kauh E, Mixson L, Malice MP, Mesens S, Ramael S, Burke J, Reynders T, Van Dyck K, Beals C, Rosenberg E, Ruddy M. Prednisone affects inflammation, glucose tolerance, and bone turnover within hours of treatment in healthy individuals. Eur J Endocrinol. 2012 Mar;166(3):459-67. doi: 10.1530/EJE-11-0751. Epub 2011 Dec 17.

Wehling-Henricks M, Oltmann M, Rinaldi C, Myung KH, Tidball JG. Loss of positive allosteric interactions between neuronal nitric oxide synthase and phosphofructokinase contributes to defects in glycolysis and increased fatigability in muscular dystrophy. Hum Mol Genet. 2009 Sep 15;18(18):3439-51. doi: 10.1093/hmg/ddp288. Epub 2009 Jun 19.

Younes M, Neffati F, Touzi M, Hassen-Zrour S, Fendri Y, Bejia I, Ben Amor A, Bergaoui N, Najjar MF. Systemic effects of epidural and intra-articular glucocorticoid injections in diabetic and non-diabetic patients. Joint Bone Spine. 2007 Oct;74(5):472-6. doi: 10.1016/j.jbspin.2006.10.009. Epub 2007 Jul 6.

Moon HJ, Choi KH, Lee SI, Lee OJ, Shin JW, Kim TW. Changes in blood glucose and cortisol levels after epidural or shoulder intra-articular glucocorticoid injections in diabetic or nondiabetic patients. Am J Phys Med Rehabil. 2014 May;93(5):372-8. doi: 10.1097/PHM.0000000000000001.

Investigator's Brochure, Version 4, Vamorolone (17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione) 4% Oral Suspension, ReveraGen BioPharma, Inc., January 10, 2017.

Fleishaker DL, Mukherjee A, Whaley FS, Daniel S, Zeiher BG. Safety and pharmacodynamic dose response of short-term prednisone in healthy adult subjects: a dose ranging, randomized, placebo-controlled, crossover study. BMC Musculoskelet Disord. 2016 Jul 16;17:293. doi: 10.1186/s12891-016-1135-3.

Hathout Y, Conklin LS, Seol H, Gordish-Dressman H, Brown KJ, Morgenroth LP, Nagaraju K, Heier CR, Damsker JM, van den Anker JN, Henricson E, Clemens PR, Mah JK, McDonald C, Hoffman EP. Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children. Sci Rep. 2016 Aug 17;6:31727. doi: 10.1038/srep31727.

Agwu JC, Spoudeas H, Hindmarsh PC, Pringle PJ, Brook CG. Tests of adrenal insufficiency. Arch Dis Child. 1999 Apr;80(4):330-3. doi: 10.1136/adc.80.4.330.

van Staa TP, Leufkens HG, Abenhaim L, Zhang B, Cooper C. Oral corticosteroids and fracture risk: relationship to daily and cumulative doses. Rheumatology (Oxford). 2000 Dec;39(12):1383-9. doi: 10.1093/rheumatology/39.12.1383.

FDA Draft Guidance for Industry: Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment. June 2015.

Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve. 1981 May-Jun;4(3):186-97. doi: 10.1002/mus.880040304. No abstract available.

Mazzone ES, Messina S, Vasco G, Main M, Eagle M, D'Amico A, Doglio L, Politano L, Cavallaro F, Frosini S, Bello L, Magri F, Corlatti A, Zucchini E, Brancalion B, Rossi F, Ferretti M, Motta MG, Cecio MR, Berardinelli A, Alfieri P, Mongini T, Pini A, Astrea G, Battini R, Comi G, Pegoraro E, Morandi L, Pane M, Angelini C, Bruno C, Villanova M, Vita G, Donati MA, Bertini E, Mercuri E. Reliability of the North Star Ambulatory Assessment in a multicentric setting. Neuromuscul Disord. 2009 Jul;19(7):458-61. doi: 10.1016/j.nmd.2009.06.368. Epub 2009 Jun 23.

McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, Atkinson L, Reha A, Hirawat S, Miller LL. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve. 2010 Apr;41(4):500-10. doi: 10.1002/mus.21544.

Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, Duong T, Henricson EK, Pegoraro E, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group Investigators. Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study. Ann Neurol. 2015 Apr;77(4):684-96. doi: 10.1002/ana.24370. Epub 2015 Mar 13.

Spurney C, Shimizu R, Morgenroth LP, Kolski H, Gordish-Dressman H, Clemens PR; CINRG Investigators. Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy. Muscle Nerve. 2014 Aug;50(2):250-6. doi: 10.1002/mus.24163. Epub 2014 May 14.

McDonald CM, Henricson EK, Abresch RT, Han JJ, Escolar DM, Florence JM, Duong T, Arrieta A, Clemens PR, Hoffman EP, Cnaan A; Cinrg Investigators. The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used. Muscle Nerve. 2013 Jul;48(1):32-54. doi: 10.1002/mus.23807. Epub 2013 May 16.

Henricson EK, Abresch RT, Cnaan A, Hu F, Duong T, Arrieta A, Han J, Escolar DM, Florence JM, Clemens PR, Hoffman EP, McDonald CM; CINRG Investigators. The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures. Muscle Nerve. 2013 Jul;48(1):55-67. doi: 10.1002/mus.23808. Epub 2013 May 6.

Escolar DM, Hache LP, Clemens PR, Cnaan A, McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y, Lotze T, Pestronk A, Ryan MM, Monasterio E, Day JW, Zimmerman A, Arrieta A, Henricson E, Mayhew J, Florence J, Hu F, Connolly AM. Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. Neurology. 2011 Aug 2;77(5):444-52. doi: 10.1212/WNL.0b013e318227b164. Epub 2011 Jul 13.

Layout table for additonal information

Responsible Party:	ReveraGen BioPharma, Inc.
ClinicalTrials.gov Identifier:	NCT03439670
Other Study ID Numbers:	VBP15-004
First Submitted:	January 9, 2018
First Posted:	February 20, 2018
Results First Submitted:	February 28, 2022
Results First Posted:	July 13, 2022
Last Update Posted:	March 9, 2023

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