A Study to Assess Vamorolone in Boys With Duchenne Muscul... | NCT02760264

ID	Type	Description	Link
1R44NS095423-01	U.S. NIH Grant/Contract		View source
1U34AR068616-01	U.S. NIH Grant/Contract		View source

Status Module

Name	Class
University of Pittsburgh	OTHER
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)	NIH
National Institute of Neurological Disorders and Stroke (NINDS)	NIH

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Dose Level Group 1	Experimental	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.	Drug: Vamorolone 0.25 mg/kg/day
Dose Level Group 2	Experimental	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.	Drug: Vamorolone 0.75 mg/kg/day
Dose Level Group 3	Experimental	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.	Drug: Vamorolone 2.0 mg/kg/day
Dose Level Group 4	Experimental	Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.	Drug: Vamorolone 6.0 mg/kg/day

Name	Type	Description	Arm Group Labels	Other Names
Vamorolone 0.25 mg/kg/day	Drug	Oral administration of 0.25 mg/kg/day daily for 14 days.	Dose Level Group 1

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03	Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug. Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group	Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.

Secondary Outcomes

Measure	Description	Time Frame
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose	Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.	Baseline, Week 2
Serum Pharmacodynamic Biomarkers (Insulin Resistance) -Fasting Glucose

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Subject's parent or legal guardian has provided written informed consent/Health Insurance Portability and Accountability Act (HIPAA) authorization prior to any study-related procedures;
Subject has a confirmed (by Central Genetic Counselor) diagnosis of DMD as defined as:
1. Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
2. Identifiable mutation within the DMD gene (deletion/duplication of one or more exons) where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical DMD, OR
3. Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e. nonsense mutation, deletion/duplication leading to a downstream stop codon), with a typical clinical picture of DMD;
Subject is ≥ 4 years and < 7 years of age at time of enrollment in the study;
Subject is able to complete the Time to Stand Test (TTSTAND) without assistance, as assessed at the Screening and Baseline Visits;
Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator. (Note: Serum gamma glutamyl transferase [GGT], creatinine, and total bilirubin all must be ≤ upper limit of the normal range at the Screening Visit);
Subject has evidence of chicken pox immunity as determined by presence of IgG antibodies to varicella, as documented by a positive test result from the testing laboratory at the Screening Visit; and
Subject and parent/guardian are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Exclusion Criteria:

Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
Subject has current or history of chronic systemic fungal or viral infections;
Subject has had an acute illness within 4 weeks prior to the first dose of study medication;
Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
Subject is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents. [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 3 months cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis. Inhaled and/or topical corticosteroids prescribed for an indication other than DMD are permitted but must be administered at stable dose for at least 3 months prior to study drug administration];
Subject has used idebenone within 4 weeks prior to the first dose of study medication;
Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
Subject is taking any other investigational drug currently or has taken any other investigational drug within 3 months prior to the start of study treatment; or
Subject has previously been enrolled in the study.

Name	Affiliation	Role
Paula R Clemens, MD	University of Pittsburgh	Study Chair

Facility	Status	City	State	ZIP	Country	Contacts
University of California Davis		Davis	California	95616	United States
University of Florida

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
38521535	Derived	Hoffman E, Gaglianone S, Ketema R, Tu W, Peay H, Clemens P, Dang U, Conklin L. Return of participant-level clinical trial results to participants: pilot of a simplified centralised approach. BMJ Open. 2024 Mar 23;14(3):e080097. doi: 10.1136/bmjopen-2023-080097.
32956407	Derived	Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep.

Label	URL
Cooperative International Research Group	View source

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Vamorolone 0.25 mg/kg/Day	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Oral administration of 0.25 mg/kg/day daily for 14 days.
FG001	Vamorolone 0.75 mg/kg/Day

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Jan 20, 2017	Jul 30, 2018

Arm/Group Information

ID	Title	Description
BG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
BG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
BG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
BG003	Dose Level Group 4	Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
BG004	Total	Total of all reporting groups

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03

Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug.

Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group

Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in safety population.

Posted

Number

Number of Events

Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003	Dose Level Group 4	Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.

Units	Counts
Participants	OG00012 OG00112 OG00212 OG003

Title

Denominators

Categories

Total Number of Adverse Events

Title	Measurements
	OG00016 OG00118 OG00213 OG003

Secondary

Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subject who receive at least dose of vamorolone study medication will be included in the safety population.

Posted

Mean

Standard Deviation

mg/ dL

Baseline, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamic Biomarkers (Insulin Resistance) -Fasting Glucose

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subject who receive at least dose of vamorolone study medication will be included in the safety population.

Posted

Mean

Standard Deviation

Week 2 % change from Baseline

Baseline, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

µIU/mL

Baseline , Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

Week 2 % change from Baseline

Baseline, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamic Biomarkers (Adrenal Axis Suppression)- First in Morning Cortisol

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Posted

Mean

Standard Deviation

mcg/dL

Week 2 (pre-dose)

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003	Dose Level Group 4	Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.

Secondary

Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

ng/mL

Baseline, Day 1, Week 2, Week 4

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

% change from Baseline

Baseline, Day 1, Week 2, Week 4

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

ng/mL

Baseline Day 1 Week 2 Week 4

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

% change from Baseline

Baseline, Day 1, Week 2, Week 4

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.

Secondary

Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

pg/mL

Baseline, Day 1, Week 4

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD.

Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.

Posted

Mean

Standard Deviation

% change from Baseline

Baseline, Day 1, Week 4

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003

Secondary

Pharmacokinetic (PK) Assessments (Tmax)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. tmax= time when plasma concentration is at maximum.

All subjects who receive at least one dose of vamorolone study medication and have sufficient quantifiable plasma concentration data for PK analysis will be included in the PK population. [Note that the PK population will be determined by the study pharmacokineticist upon review of the concentration information for each subject in each cohort.](streamdown:incomplete-link)

Posted

Mean

Standard Deviation

hour

Day 1, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.

Secondary

Pharmacokinetic (PK) Assessments (AUC Inf)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. AUC inf= Area under the concentration vs. time curve to time infinity.

Posted

Mean

Standard Deviation

[(hr)(ng)/mL]

Day 1, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.

Secondary

Pharmacokinetic (PK) Assessments CL (ml/hr/kg)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay

Posted

Mean

Standard Deviation

ml/hr/kg

Day 1, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003	Dose Level Group 4	Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.

Secondary

Pharmacokinetic (PK) Assessments t(1/2)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. t1/2= elimination half life.

Posted

Mean

Standard Deviation

hour

Day 1, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.

Secondary

Pharmacokinetic (PK) Assessments (Cmax)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay

Posted

Mean

Standard Deviation

ng/mL

Day 1, Week 2

ID	Title	Description
OG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
OG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
OG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
OG003	Dose Level Group 4	Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.

Secondary

Metabolites in Safety Testing (MIST) Assessment

A portion of each blood sample of the Week 2 (Day 14) pharmacokinetic assessment time points for the subjects receiving vamorolone 2 mg/kg/day was used for analysis of vamorolone metabolites.

Posted

Mean

Standard Deviation

% of total drug related exposure

Week 2 (Day 14)

ID	Title	Description
OG000	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.

Units	Counts
Participants	OG00012

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Dose Level Group 1	Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day. Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.	12	12	7	12
EG001	Dose Level Group 2	Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day. Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.	12	12	6	12
EG002	Dose Level Group 3	Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day. Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.	12	12	8	12
EG003	Dose Level Group 4	Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day. Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.	12	12	7	12

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Abdominal Pain	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0011 affected12 at risk EG0020 affected12 at risk EG0030 affected12 at risk
Abdominal Pain Upper	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Constipation	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Diarrhoea	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Faeces Discoloured	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Lip Swelling	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Pyrexia	General disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Enterobiasis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Gastroenteritis viral	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Lice Infestation	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Viral Upper Respiratory Tract Infection	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0003 affected12 at risk EG0012 affected12 at risk EG0021 affected12 at risk EG003
Fall	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0011 affected12 at risk EG0020 affected12 at risk EG003
Muscle Spasms	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Pain in Extremity	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0013 affected12 at risk EG0020 affected12 at risk EG003
Headache	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Lethargy	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0011 affected12 at risk EG0020 affected12 at risk EG003
Agitation	Psychiatric disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Emotional Disorder	Psychiatric disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0021 affected12 at risk EG003
Insomnia	Psychiatric disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Irritability	Psychiatric disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Restlessness	Psychiatric disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0011 affected12 at risk EG0020 affected12 at risk EG003
Chromaturia	Renal and urinary disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0011 affected12 at risk EG0020 affected12 at risk EG003
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0011 affected12 at risk EG0020 affected12 at risk EG003
Upper-airway cough syndrome	Respiratory, thoracic and mediastinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0012 affected12 at risk EG0020 affected12 at risk EG003
Eczema	Skin and subcutaneous tissue disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Rash Macular	Skin and subcutaneous tissue disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003
Flushing	Vascular disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected12 at risk EG0010 affected12 at risk EG0020 affected12 at risk EG003

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases
D009468	Neuromuscular Diseases
D009422	Nervous System Diseases
D040181	Genetic Diseases, X-Linked
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities