Trial Outcomes & Findings for A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) (NCT NCT05185622)

NCT ID: NCT05185622

Last Updated: 2025-10-24

Results Overview

An Adverse Event is any untoward medical occurrence in a subject and does not necessarily have to have a causal relationship with the intervention. Pre-existing conditions that worsen during the study are to be reported as AEs.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 54 participants
• Primary outcome timeframe: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed
• Results posted on: 2025-10-24

Participant Flow

Participant milestones

Measure	Treatment Group 1 Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Overall Study STARTED	10	10	6	6	6	16
Overall Study COMPLETED	10	10	6	6	6	16
Overall Study NOT COMPLETED	0	0	0	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)

Baseline characteristics by cohort

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Total n=54 Participants Total of all reporting groups
Age, Categorical <=18 years	10 Participants n=5 Participants	10 Participants n=7 Participants	6 Participants n=5 Participants	6 Participants n=4 Participants	6 Participants n=21 Participants	16 Participants n=10 Participants	54 Participants n=115 Participants
Age, Categorical Between 18 and 65 years	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	0 Participants n=10 Participants	0 Participants n=115 Participants
Age, Categorical >=65 years	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	0 Participants n=10 Participants	0 Participants n=115 Participants
Sex: Female, Male Female	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	0 Participants n=10 Participants	0 Participants n=115 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	10 Participants n=7 Participants	6 Participants n=5 Participants	6 Participants n=4 Participants	6 Participants n=21 Participants	16 Participants n=10 Participants	54 Participants n=115 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	1 Participants n=21 Participants	2 Participants n=10 Participants	4 Participants n=115 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants	10 Participants n=7 Participants	6 Participants n=5 Participants	3 Participants n=4 Participants	4 Participants n=21 Participants	14 Participants n=10 Participants	47 Participants n=115 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants	1 Participants n=21 Participants	0 Participants n=10 Participants	3 Participants n=115 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	0 Participants n=10 Participants	0 Participants n=115 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants	2 Participants n=21 Participants	3 Participants n=10 Participants	14 Participants n=115 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	0 Participants n=10 Participants	0 Participants n=115 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	1 Participants n=115 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants	8 Participants n=7 Participants	5 Participants n=5 Participants	3 Participants n=4 Participants	3 Participants n=21 Participants	13 Participants n=10 Participants	37 Participants n=115 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	1 Participants n=115 Participants
Race (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	1 Participants n=21 Participants	0 Participants n=10 Participants	1 Participants n=115 Participants
Region of Enrollment Canada	10 participants n=5 Participants	10 participants n=7 Participants	6 participants n=5 Participants	6 participants n=4 Participants	6 participants n=21 Participants	16 participants n=10 Participants	54 participants n=115 Participants
Age at first symptom	20.8 months STANDARD_DEVIATION 5.67 • n=5 Participants	14.2 months STANDARD_DEVIATION 4.47 • n=7 Participants	30.0 months STANDARD_DEVIATION 15.18 • n=5 Participants	51.0 months STANDARD_DEVIATION 23.92 • n=4 Participants	32.2 months STANDARD_DEVIATION 14.26 • n=21 Participants	42.8 months STANDARD_DEVIATION 27.59 • n=10 Participants	31.7 months STANDARD_DEVIATION 21.88 • n=115 Participants

PRIMARY outcome

Timeframe: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

Population: Safety set

An Adverse Event is any untoward medical occurrence in a subject and does not necessarily have to have a causal relationship with the intervention. Pre-existing conditions that worsen during the study are to be reported as AEs.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Participants With Any Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)	7 Participants	9 Participants	6 Participants	4 Participants	3 Participants	12 Participants

PRIMARY outcome

Timeframe: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

Population: Safety set

Drug related Adverse Events are TEAEs whose Causality were labeled as 'DEFINITE', 'POSSIBLE' or 'PROBABLE

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Participants With Drug Related Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)	1 Participants	7 Participants	3 Participants	4 Participants	1 Participants	8 Participants

PRIMARY outcome

Timeframe: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

Population: Safety set

Severe or medically significant but not immediately life -threatening: hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living; incapacitating with inability to work or perform normal daily activity.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Participants With Severe Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)	0 Participants	0 Participants	0 Participants	1 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose of study drug)

Population: Safety set

A Serious Adverse Event (SAE) is defined as any AE regardless of causality that meets any of the following criteria:

• Results in death
• Is life-threatening
• Requires inpatient hospitalization or prolongation of an existing hospitalization
• Results in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions
• Results in a congenital anomaly/birth defect
• Is an important medical event that may jeopardize the subject and may require medical or surgical intervention to prevent one of the outcomes listed above

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Participants With Serious Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03)	0 Participants	0 Participants	0 Participants	1 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: From the date of the subject's written informed consent until the final Week 16 Visit or the subject's participation in the study was completed

Population: Safety set

Adverse Events leading to Study treatment discontinuation

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Participants With Adverse Events as Assessed by Common Terminology Criteria for Adverse Events Version 4.03 (CTCAE v4.03) Leading to Study Treatment Discontinuation	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: Safety set

Standing height will be assessed for subjects ages 2-<4 years; height calculated from ulnar length in subjects ages 7-<18.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Height (Absolute) From Baseline to Week 12	1.98 cm Standard Deviation 1.146	2.35 cm Standard Deviation 0.568	0.55 cm Standard Deviation 0.557	2.29 cm Standard Deviation 1.609	0.60 cm Standard Deviation 0.594	1.27 cm Standard Deviation 1.313

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: Safety set

Standing height will be assessed for subjects ages 2-<4 years; height calculated from ulnar length in subjects ages 7-<18.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Height (Percentile) From Baseline to Week 12	1.17 percentile Standard Deviation 11.538	2.09 percentile Standard Deviation 5.634	-3.89 percentile Standard Deviation 3.907	2.02 percentile Standard Deviation 8.338	-3.69 percentile Standard Deviation 3.408	-0.30 percentile Standard Deviation 1.390

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: Safety set

Standing height will be assessed for subjects ages 2-<4 years; height calculated from ulnar length in subjects ages 7-<18. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher height).

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Height (Z-score) From Baseline to Week 12	0.04 Z-score (score on a scale) Standard Deviation 0.361	0.14 Z-score (score on a scale) Standard Deviation 0.201	-0.12 Z-score (score on a scale) Standard Deviation 0.111	0.07 Z-score (score on a scale) Standard Deviation 0.253	-0.13 Z-score (score on a scale) Standard Deviation 0.159	0.00 Z-score (score on a scale) Standard Deviation 0.194

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: Safety set

Body weight will be assessed at each of the scheduled time points.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Weight (Absolute) From Baseline to Week 12	0.43 kg Standard Deviation 0.776	0.65 kg Standard Deviation 0.911	-0.18 kg Standard Deviation 3.259	3.47 kg Standard Deviation 3.133	1.53 kg Standard Deviation 1.188	1.94 kg Standard Deviation 2.598

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: Safety set

Body weight will be assessed at each of the scheduled time points.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Weight (Percentile) From Baseline to Week 12	-1.44 percentile Standard Deviation 12.835	-1.97 percentile Standard Deviation 12.504	-4.00 percentile Standard Deviation 11.672	15.20 percentile Standard Deviation 2.083	2.12 percentile Standard Deviation 4.235	4.22 percentile Standard Deviation 8.566

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: Safety set

Body weight will be assessed at each of the scheduled time points. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher weight).

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Weight (Z-score) From Baseline to Week 12	-0.01 Z-score (score on a scale) Standard Deviation 0.406	0.05 Z-score (score on a scale) Standard Deviation 0.430	-0.03 Z-score (score on a scale) Standard Deviation 0.438	0.55 Z-score (score on a scale) Standard Deviation 0.159	0.11 Z-score (score on a scale) Standard Deviation 0.147	0.22 Z-score (score on a scale) Standard Deviation 0.353

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: Safety set

Body Mass Index is a measure of weight adjusted for height.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Body Mass Index (BMI) (Absolute) From Baseline to Week 12	-0.09 kg/m2 Standard Deviation 0.933	-0.04 kg/m2 Standard Deviation 0.665	0.05 kg/m2 Standard Deviation 1.394	1.53 kg/m2 Standard Deviation 1.385	0.82 kg/m2 Standard Deviation 0.784	0.60 kg/m2 Standard Deviation 1.471

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: Safety set

Body Mass Index is a measure of weight adjusted for height.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Body Mass Index (BMI) (Percentile) From Baseline to Week 12	-2.85 percentile Standard Deviation 24.623	-1.61 percentile Standard Deviation 12.509	-3.00 percentile Standard Deviation 15.781	10.78 percentile Standard Deviation 7.312	6.22 percentile Standard Deviation 6.849	4.44 percentile Standard Deviation 11.498

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: Safety set

Body Mass Index is a measure of weight adjusted for height. The z-score (standard score) is the number of standard deviations by which the outcome measure is above or below the value in the general population. A Z-score of 0 represents the population mean. A measure above the normal value has a positive z-score (higher BMI).

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Body Mass Index (BMI) (Z-score) From Baseline to Week 12	-0.05 Z-score (score on a scale) Standard Deviation 0.764	-0.02 Z-score (score on a scale) Standard Deviation 0.495	0.27 Z-score (score on a scale) Standard Deviation 0.771	1.10 Z-score (score on a scale) Standard Deviation 0.584	0.29 Z-score (score on a scale) Standard Deviation 0.317	0.29 Z-score (score on a scale) Standard Deviation 0.483

PRIMARY outcome

Timeframe: Day 1, Week 2, Week 6, Week 12, Week 16

Population: Safety set

Change from Baseline to Week 12 in diastolic sitting blood pressure.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Diastolic Blood Pressure	-0.44 mmHg Standard Deviation 8.248	2.00 mmHg Standard Deviation 8.179	-3.17 mmHg Standard Deviation 7.333	5.80 mmHg Standard Deviation 6.979	1.50 mmHg Standard Deviation 6.950	-2.40 mmHg Standard Deviation 11.915

PRIMARY outcome

Timeframe: Day 1, Week 2, Week 6, Week 12, Week 16

Population: Safety set

Change from Baseline to Week 12 in systolic sitting blood pressure.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change in Systolic Blood Pressure	-2.33 mmHg Standard Deviation 12.923	-2.20 mmHg Standard Deviation 11.233	-2.50 mmHg Standard Deviation 6.504	5.00 mmHg Standard Deviation 13.266	2.50 mmHg Standard Deviation 5.958	0.07 mmHg Standard Deviation 11.591

PRIMARY outcome

Timeframe: Baseline through Week 16

Population: Safety set

Treatment emergent cushingoid features based on physical examination at all baseline, on-treatment and post-treatment assessments

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Participants With Treatment Emergent Cushingoid Features	0 Participants	0 Participants	0 Participants	1 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Day 1, Week 6, Week 12, Week 16

Population: Safety set

Each subject had blood drawn and urine collected for the standard hematology, chemistry and lipids clinical laboratory tests. In addition, fasting glucose and insulin, morning cortisol, as well as, in the additional 12 to <18 years age group, LH, FSH, TSH, FT4 were collected. HbA1c determination had also to be performed if urine glucose was positive and/or fasted glucose levels was above normal limits. Any treatment-emergent clinically significant abnormal laboratory test result was reporte

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result Cortisol decreased	0 Participants	1 Participants	2 Participants	0 Participants	0 Participants	2 Participants
Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result Blood glucose decreased	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants
Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result Blood insulin increased	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants
Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result Blood TSH increased	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants
Number of Participants With Clinically Significant Treatment-emergent Abnormal Clinical Laboratory Test Result Tyroxin free increased	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	1 Participants

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: Safety population

12-lead 1electrocardiogram (ECG) as recorded after subject has rested quietly in a supine position for at least 5 minutes. ECG components are QRS duration, PR [PQ] interval, RR interval, QT interval and QTc.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=5 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=15 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Categorical Analysis of QTcF at Week 12 =< 450 msec	10 Participants	10 Participants	6 Participants	6 Participants	5 Participants	15 Participants
Categorical Analysis of QTcF at Week 12 > 450 msec	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline - Week 12

Population: Safety set

Cataract was diagnosed by the presence of partial or complete opacity of the crystalline lens at Baseline and Week 12.

Outcome measures

Measure	Treatment Group 1 n=18 eye Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=19 eye Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=12 eye Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=12 eye Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=12 eye Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=32 eye Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Eyes With Cataract No Cataract : Baseline	18 eyes	19 eyes	12 eyes	12 eyes	8 eyes	17 eyes
Number of Eyes With Cataract No Cataract : Week 12	18 eyes	8 eyes	12 eyes	10 eyes	8 eyes	15 eyes
Number of Eyes With Cataract Cataract : Baseline	0 eyes	0 eyes	0 eyes	0 eyes	4 eyes	15 eyes
Number of Eyes With Cataract Cataract : Week 12	0 eyes	0 eyes	0 eyes	0 eyes	2 eyes	13 eyes

PRIMARY outcome

Timeframe: Baseline - Week 12

Population: Safety set

Glaucoma was diagnosed by ocular pressure at Baseline and Week 12.

Outcome measures

Measure	Treatment Group 1 n=18 eye Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=19 eye Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=12 eye Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=12 eye Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=12 eye Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=32 eye Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Number of Eyes With Glaucoma Glaucoma: Baseline	0 eyes	0 eyes	0 eyes	0 eyes	0 eyes	0 eyes
Number of Eyes With Glaucoma No Glaucoma: Baseline	18 eyes	19 eyes	12 eyes	12 eyes	8 eyes	17 eyes
Number of Eyes With Glaucoma No Glaucoma: Week 12	18 eyes	8 eyes	12 eyes	10 eyes	8 eyes	15 eyes
Number of Eyes With Glaucoma Suspected Glaucoma: Baseline	0 eyes	0 eyes	0 eyes	0 eyes	0 eyes	2 eyes
Number of Eyes With Glaucoma Suspected Glaucoma: Week 12	0 eyes	0 eyes	0 eyes	0 eyes	0 eyes	0 eyes
Number of Eyes With Glaucoma Glaucoma: Week 12	0 eyes	0 eyes	0 eyes	0 eyes	0 eyes	0 eyes

SECONDARY outcome

Timeframe: Day 1, Week 2

Population: PK analysis set

The plasma concentration of vamorolone was measured on Day 1 and Week 2 predose, and 1h, 2h and 6h postdose and also 4h and 8h post in the 7-18 year groups.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Day 1 - predose	0.0 ng / mL Standard Deviation 0.00	0.0 ng / mL Standard Deviation 0.00	0.0 ng / mL Standard Deviation 0.00	0.0 ng / mL Standard Deviation 0.00	0.0 ng / mL Standard Deviation 0.00	0.0 ng / mL Standard Deviation 0.00
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Day 1 - 1h	198.3 ng / mL Standard Deviation 196.67	471.3 ng / mL Standard Deviation 333.61	142.5 ng / mL Standard Deviation 225.74	519.1 ng / mL Standard Deviation 437.26	142.5 ng / mL Standard Deviation 225.74	519.1 ng / mL Standard Deviation 437.26
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Day 1 - 2h	236.7 ng / mL Standard Deviation 153.58	769.3 ng / mL Standard Deviation 527.67	216.4 ng / mL Standard Deviation 181.19	780.5 ng / mL Standard Deviation 457.91	216.4 ng / mL Standard Deviation 181.19	780.5 ng / mL Standard Deviation 457.91
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Day 1 - 4h	—	—	163.8 ng / mL Standard Deviation 103.74	711.1 ng / mL Standard Deviation 353.60	163.8 ng / mL Standard Deviation 103.74	711.1 ng / mL Standard Deviation 353.60
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Day 1 - 6h	125.7 ng / mL Standard Deviation 84.83	280.0 ng / mL Standard Deviation 249.99	148.0 ng / mL Standard Deviation 109.33	389.1 ng / mL Standard Deviation 307.57	148.0 ng / mL Standard Deviation 109.33	389.1 ng / mL Standard Deviation 307.57
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Day 1 - 8h	—	—	78.5 ng / mL Standard Deviation 41.66	170.6 ng / mL Standard Deviation 183.67	78.5 ng / mL Standard Deviation 41.66	170.6 ng / mL Standard Deviation 183.67
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Week 2 - predose	0.0 ng / mL Standard Deviation 0.00	0.0 ng / mL Standard Deviation 0.00	0.7 ng / mL Standard Deviation 2.06	2.9 ng / mL Standard Deviation 5.22	0.7 ng / mL Standard Deviation 2.06	2.9 ng / mL Standard Deviation 5.22
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Week 2 - 1h	346.0 ng / mL Standard Deviation 329.08	409.8 ng / mL Standard Deviation 385.65	192.0 ng / mL Standard Deviation 199.92	463.7 ng / mL Standard Deviation 348.19	192.0 ng / mL Standard Deviation 199.92	463.7 ng / mL Standard Deviation 348.19
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Week 2 - 2h	360.4 ng / mL Standard Deviation 222.93	829.4 ng / mL Standard Deviation 740.83	217.8 ng / mL Standard Deviation 149.31	686.3 ng / mL Standard Deviation 369.69	217.8 ng / mL Standard Deviation 149.31	686.3 ng / mL Standard Deviation 369.69
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Week 2 - 4h	—	—	207.6 ng / mL Standard Deviation 167.99	603.0 ng / mL Standard Deviation 346.88	207.6 ng / mL Standard Deviation 167.99	603.0 ng / mL Standard Deviation 346.88
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Week 2 - 6h	134.5 ng / mL Standard Deviation 110.99	272.0 ng / mL Standard Deviation 109.71	158.9 ng / mL Standard Deviation 105.34	300.2 ng / mL Standard Deviation 206.18	158.9 ng / mL Standard Deviation 105.34	300.2 ng / mL Standard Deviation 206.18
Pre-dose and Post-dose Plasma Concentration Measurements of Vamorolone at Day 1 and Week 2 Week 2 - 8h	—	—	55.8 ng / mL Standard Deviation 31.81	174.7 ng / mL Standard Deviation 137.78	55.8 ng / mL Standard Deviation 31.81	174.7 ng / mL Standard Deviation 137.78

SECONDARY outcome

Timeframe: Day 1, Week 2

Population: PK analysis set

Tmax is the time to reach the maximum observed concentration collected during a dosing interval

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=12 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=22 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Descriptive Statistics of PK Parameters - Tmax Day 1	2.0 h Interval 1.0 to 6.0	2.0 h Interval 1.0 to 6.0	2.0 h Interval 1.0 to 6.0	2.0 h Interval 1.0 to 6.0	—	—
Descriptive Statistics of PK Parameters - Tmax Week 2	1.9 h Interval 1.0 to 6.3	2.0 h Interval 1.1 to 6.0	2.9 h Interval 1.0 to 8.0	2.0 h Interval 1.0 to 6.0	—	—

SECONDARY outcome

Timeframe: Day 1, Week 2

Population: PK analysis set

Cmax is the maximum observed concentration

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=12 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=22 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Descriptive Statistics of PK Parameters - Cmax Day 1	246.8 ng/mL Standard Deviation 2.01	781.7 ng/mL Standard Deviation 1.77	254.5 ng/mL Standard Deviation 2.04	922.2 ng/mL Standard Deviation 1.60	—	—
Descriptive Statistics of PK Parameters - Cmax Week 2	349.3 ng/mL Standard Deviation 2.06	719.5 ng/mL Standard Deviation 2.23	334.5 ng/mL Standard Deviation 1.67	765.5 ng/mL Standard Deviation 1.59	—	—

SECONDARY outcome

Timeframe: Day 1, Week 2

Population: PK analysis set - Subjects aged 2 to 4 years

AUC 0-6 is the area under the concentration-time curve during the first 6 hours after dosing

Outcome measures

Measure	Treatment Group 1 n=8 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=8 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Descriptive Statistics of PK Parameters in Subjects Aged 2 to 4 Years - AUC 0-6 Day 1	816.3 ng.h/mL Standard Deviation 1.72	2216.2 ng.h/mL Standard Deviation 1.47	—	—	—	—
Descriptive Statistics of PK Parameters in Subjects Aged 2 to 4 Years - AUC 0-6 Week 2	1448.4 ng.h/mL Standard Deviation 1.38	2571.6 ng.h/mL Standard Deviation 2.02	—	—	—	—

SECONDARY outcome

Timeframe: Day 1, Week 2

Population: PK analysis set - subjects aged 7 to 18 years

AUC 0-8 is the area under the concentration-time curve after dosing extrapolated to infinity

Outcome measures

Measure	Treatment Group 1 n=6 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=13 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Descriptive Statistics of PK Parameters in Subjects Aged 7 to 18 Years - AUC 0-inf Day 1	1253.0 ng.h/mL Standard Deviation 1.65	4119.8 ng.h/mL Standard Deviation 1.53	—	—	—	—
Descriptive Statistics of PK Parameters in Subjects Aged 7 to 18 Years - AUC 0-inf Week 2	1528.6 ng.h/mL Standard Deviation 1.15	3587.7 ng.h/mL Standard Deviation 1.72	—	—	—	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set

The Bayley-III Gross Motor scale is a functional assessment, an accurate reflection of muscle strength for subjects with DMD ages 2 to <4 years. The minimum score value is 0 and the maximum score value is 72. Higher scores mean a better outcome.

Outcome measures

Measure	Treatment Group 1 n=9 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Bayley-III Gross Motor Scale (Ages 2 to <4 Years Only)	0.44 score on a scale Standard Deviation 1.130	2.50 score on a scale Standard Deviation 1.716	—	—	—	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set

Morning cortisol [adrenal suppression] samples were collected after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication at Day 1 and Week 12 Visits, before 10 AM local time

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=8 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=15 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Morning Cortisol Concentration	-184.700 nmol/L Standard Deviation 138.2020	-217.750 nmol/L Standard Deviation 103.9832	-110.667 nmol/L Standard Deviation 85.8596	-248.667 nmol/L Standard Deviation 186.9060	12.000 nmol/L Standard Deviation 49.3356	-34.933 nmol/L Standard Deviation 42.1485

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set

Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=9 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=5 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=13 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Type 1 Collagen C-telopeptide [CTX1])	28.300 ng/L Standard Deviation 387.1423	138.000 ng/L Standard Deviation 257.6640	93.500 ng/L Standard Deviation 235.7446	101.800 ng/L Standard Deviation 365.3542	631.833 ng/L Standard Deviation 155.3621	273.000 ng/L Standard Deviation 335.4937

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set

Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=9 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=5 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=13 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Osteocalcin)	-3.340 ug/L Standard Deviation 21.0550	2.422 ug/L Standard Deviation 10.6960	14.817 ug/L Standard Deviation 18.7328	-1.120 ug/L Standard Deviation 15.8528	31.700 ug/L Standard Deviation 11.6915	16.546 ug/L Standard Deviation 9.1486

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set

Samples for CTX1, osteocalcin and P1NP were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=9 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=5 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=14 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Bone Turnover Biomarkers (Serum Aminoterminal Propeptide of Type I Collagen [P1NP] )	-39.870 ug/L Standard Deviation 188.7787	14.478 ug/L Standard Deviation 117.5704	19.067 ug/L Standard Deviation 78.6139	-82.840 ug/L Standard Deviation 178.0726	318.850 ug/L Standard Deviation 99.0038	149.671 ug/L Standard Deviation 106.2874

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set

Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication.

Outcome measures

Measure	Treatment Group 1 n=10 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Glucose)	-0.160 mmol/L Standard Deviation 0.2912	-0.243 mmol/L Standard Deviation 0.6309	-0.063 mmol/L Standard Deviation 0.3524	-0.335 mmol/L Standard Deviation 0.3815	0.195 mmol/L Standard Deviation 0.5727	-0.254 mmol/L Standard Deviation 0.3909

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set

Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication. The Baseline sample for HbA1c measurement may have been collected non-fasting.

Outcome measures

Measure	Treatment Group 1 n=9 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=9 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=5 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=5 Participants Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=11 Participants Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Hemoglobin A1c [HbA1c])	0.1 percent Standard Deviation 0.26	0.0 percent Standard Deviation 0.15	0.0 percent Standard Deviation 0.16	0.0 percent Standard Deviation 0.10	0.0 percent Standard Deviation 0.11	-0.1 percent Standard Deviation 0.12

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 12

Population: Safety set - No subjects were analyzed in Group 1 and 5

Glucose, HbA1c and insulin were collected at the Day 1 and Week 12 Visits after the subject has fasted for ≥ 6 hours and prior to administration of the daily dose of study medication.

Outcome measures

Measure	Treatment Group 1 n=9 Participants Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=3 Participants Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 Participants Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=9 Participants Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Change From Baseline to Week 12 in Insulin Resistance Biomarkers (Insulin)	6.333 pmol/L Standard Deviation 20.5183	0.667 pmol/L Standard Deviation 33.0051	46.333 pmol/L Standard Deviation 51.9256	-26.333 pmol/L Standard Deviation 51.1493	—	—

Adverse Events

Treatment Group 1

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Treatment Group 2

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Treatment Group 3

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Treatment Group 4

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Treatment Group 5

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Treatment Group 6

Serious events: 1 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Measure	Treatment Group 1 n=10 participants at risk Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 participants at risk Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 participants at risk Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 participants at risk Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 participants at risk Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 participants at risk Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Gastrointestinal disorders Vomiting	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Musculoskeletal and connective tissue disorders Rhabdomyolysis	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).

Other adverse events

Measure	Treatment Group 1 n=10 participants at risk Patients in Treatment Group 1 must be ages 2-\<4 years and will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Treatment Group 1 will be enrolled prior to Treatment Group 2. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 2 n=10 participants at risk Patients in Treatment Group 2 must be ages 2-\<4 years and will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Treatment Group 2 will be enrolled after Treatment Group 1. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 3 n=6 participants at risk Patients in Treatment Group 3 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 3 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 4 n=6 participants at risk Patients in Treatment Group 4 must be ages 7-\<18 years and must be steroid untreated at entry. Treatment Group 4 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 5 n=6 participants at risk Patients in Treatment Group 5 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 5 will receive Vamorolone at 2.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.	Treatment Group 6 n=16 participants at risk Patients in Treatment Group 6 must be ages 7-\<18 years and must be on a stable dose of steroid for 3 months prior to entry. Treatment Group 6 will receive Vamorolone at 6.0 mg/kg/day for the duration of the study. Vamorolone: Oral administration of vamorolone for 12 weeks.
Blood and lymphatic system disorders Lymphadenopathy	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Endocrine disorders Adrenal insufficiency	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Endocrine disorders Adrenal suppression	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	50.0% 5/10 • Number of events 5 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Endocrine disorders Cushingoid	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Eye disorders Cataract	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Abdominal pain	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Abdominal pain upper	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	20.0% 2/10 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 3 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Abnormal faeces	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Diarrhoea	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	30.0% 3/10 • Number of events 3 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Faeces discoloured	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Nausea	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Toothache	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	20.0% 2/10 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Gastrointestinal disorders Vomiting	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	12.5% 2/16 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
General disorders Asthenia	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	33.3% 2/6 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
General disorders Energy increased	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
General disorders Fatigue	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
General disorders Pyrexia	10.0% 1/10 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Immune system disorders Hypersensitivity	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Infections and infestations COVID-19	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Infections and infestations Gastroenteritis viral	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Infections and infestations Nasopharyngitis	40.0% 4/10 • Number of events 8 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	30.0% 3/10 • Number of events 3 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	33.3% 2/6 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	12.5% 2/16 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Infections and infestations Upper respiratory tract infection	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Injury, poisoning and procedural complications Arthropod bite	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Injury, poisoning and procedural complications Fall	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	20.0% 2/10 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Injury, poisoning and procedural complications Head injury	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Injury, poisoning and procedural complications Joint injury	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Injury, poisoning and procedural complications Skin abrasion	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Investigations Blood glucose decreased	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Investigations Blood insulin increased	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Investigations Blood thyroid stimulating hormone increased	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Investigations Cortisol decreased	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	33.3% 2/6 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	12.5% 2/16 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Investigations Thyroxine free increased	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Investigations Weight increased	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Metabolism and nutrition disorders Increased appetite	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	33.3% 2/6 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	12.5% 2/16 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Musculoskeletal and connective tissue disorders Musculoskeletal stiffness	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	20.0% 2/10 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	33.3% 2/6 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Nervous system disorders Headache	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 3 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	25.0% 4/16 • Number of events 5 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Nervous system disorders Hypoaesthesia	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Psychiatric disorders Aggression	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Psychiatric disorders Behaviour disorder	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Psychiatric disorders Compulsive lip biting	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Psychiatric disorders Disruptive mood dysregulation disorder	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	12.5% 2/16 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Psychiatric disorders Irritability	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Psychiatric disorders Mood swings	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Respiratory, thoracic and mediastinal disorders Cough	20.0% 2/10 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	6.2% 1/16 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	10.0% 1/10 • Number of events 2 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Skin and subcutaneous tissue disorders Miliaria	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Skin and subcutaneous tissue disorders Rash	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	16.7% 1/6 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).
Skin and subcutaneous tissue disorders Urticaria	10.0% 1/10 • Number of events 1 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/10 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/6 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).	0.00% 0/16 • AEs were collected through Week 16 Visit or the subject's participation in the study was completed (SAEs through 30 days after final dose study drug).

Additional Information

Dr Shabir Hasham

Santhera

Phone: +41 61 906 89 50

Email: Shabir.Hasham@santhera.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place