Trial Outcomes & Findings for Cannabidiol for ASD Open Trial (NCT NCT03900923)

Last Updated: 2024-05-31

Results Overview

The physician-rated CGI-I comprises one question asking how much a participant's condition has changed since baseline. The item is answered using a 7-point Likert scale, where 1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; and 7 = very much worse. The total score is the item response; lower scores indicate greater improvement.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

29 participants

Primary outcome timeframe

Week 6

Results posted on

2024-05-31

Participant Flow

Participant milestones

Participant milestones
Measure	3 mg/kg 98% Pure CBD Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Overall Study STARTED	6	9	8
Overall Study Participants Started Part 1: 3 mg/kg/Day	6	0	0
Overall Study Participants Completed Part 1: 3 mg/kg/Day	6	0	0
Overall Study Participants Started Part 1: 6 mg/kg/Day	0	9	0
Overall Study Participants Completed Part 1: 6 mg/kg/Day	0	9	0
Overall Study Participants Started Part 2: 9 mg/kg/Day	0	0	8
Overall Study Participants Completed Part 2: 9 mg/kg/Day	0	0	8
Overall Study COMPLETED	6	9	8
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cannabidiol for ASD Open Trial

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.	Total n=23 Participants Total of all reporting groups
Age, Continuous	10.32 years STANDARD_DEVIATION 2.18 • n=5 Participants	11.21 years STANDARD_DEVIATION 2.56 • n=7 Participants	12.16 years STANDARD_DEVIATION 3.4 • n=5 Participants	11.31 years STANDARD_DEVIATION 2.77 • n=4 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	8 Participants n=7 Participants	8 Participants n=5 Participants	22 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	4 Participants n=7 Participants	4 Participants n=5 Participants	8 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants	4 Participants n=7 Participants	4 Participants n=5 Participants	14 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 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
Race (NIH/OMB) Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 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
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	6 Participants n=7 Participants	7 Participants n=5 Participants	16 Participants n=4 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Region of Enrollment United States	6 participants n=5 Participants	9 participants n=7 Participants	8 participants n=5 Participants	23 participants n=4 Participants

PRIMARY outcome

Timeframe: Week 6

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Clinical Global Impression Scale-Improvement (CGI-I)	3.17 score on a scale Standard Deviation 0.75	2.56 score on a scale Standard Deviation 0.88	2.25 score on a scale Standard Deviation 0.71

SECONDARY outcome

Timeframe: Baseline, Week 6

The RBS-R is a 43-item self report used to measure the breadth of repetitive behavior in children. For each item, behaviors are rated on a 4-point scale: 0-Behavior does not occur, 1-Behavior occurs and is a mild problem, 2-Behavior occurs and is a moderate problem, 3-Behavior occurs and is a severe problem. On the last question, participants are asked to "lump together" all of the behaviors described in the questionnaire, and provide a rating for how much of a problem these repetitive behaviors are overall, on a scale from 1-100: 1-Not a problem at all, to 100-As bad as you can imagine. A decrease in scores indicates the repetitive behaviors became less of a problem overall. The total score is the sum of all items and ranges from 0 to 229.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Repetitive Behavior Scale-Revised (RBS-R) Score	-0.5 score on a scale Standard Deviation 9.52	-6.67 score on a scale Standard Deviation 9.41	-4.38 score on a scale Standard Deviation 8.26

SECONDARY outcome

Timeframe: Baseline, Week 6

The SRS-2 is a 65-item questionnaire assessing Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests and Repetitive Behavior. Items are scored on a 4-point scale (ranging from 1=not true to 4=almost always true). The raw score is the sum of responses and is converted to a T-score; the final T-score has a mean of 50 points with a standard deviation of 10 points, where lower scores indicate greater social responsiveness: * Less than or equal to 59 = Low-to-no symptom impacts * Between 60-65 = Mild-to-moderate defcits in social interaction * Between 66-75 = Moderate defcit in social interaction * Greater than or equal to 76 = Severe

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Social Responsiveness Scale, 2nd Edition (SRS-2), School-Age Form Score	-6.17 score on a scale Standard Deviation 5	-14 score on a scale Standard Deviation 12.55	-17.4 score on a scale Standard Deviation 5.5

SECONDARY outcome

Timeframe: Baseline, Week 6

The ABC Irritability subscale comprises 15 items rated on a 4-point scale (ranging from 0=no problem at all to 3 = problem is severe in degree). The total score is the sum of responses and ranges from 0 to 45; higher scores indicate greater presences of irritability related behavior.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Aberrant Behavior Checklist (ABC) - Irritability Subscale Score	-5.5 score on a scale Standard Deviation 5.4	-5.89 score on a scale Standard Deviation 7.51	-1.4 score on a scale Standard Deviation 3.1

SECONDARY outcome

Timeframe: Baseline, Week 6

The SCARED, Parent Version is a 41-item parent-report measure developed to measure child anxiety symptoms. Subscales include Somatic Symptoms/Panic Disorder, Generalized Anxiety Disorder, Separation Anxiety, Social Phobia, and School Phobia. Items are rated on a 3-point scale ranging from 0=not true or hardly ever true to 2=very true or often true. The total score is the sum of responses and ranges from 0 to 82; lower scores indicate lesser child anxiety symptoms.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Screen for Child Anxiety Related Disorders (SCARED), Parent Version Score	-6.33 score on a scale Standard Deviation 13.7	-16 score on a scale Standard Deviation 17.12	-3.25 score on a scale Standard Deviation 12.6

SECONDARY outcome

Timeframe: Baseline, Week 6

The SDSC is a 26-item parent-completed assessment of their child's sleep-wake rhythm. The questionnaire measures Disorders of Initiating and Maintaining Sleep, Sleep Breathing Disorders, Disorders of Arousal, Sleep Wake Transition Disorders, Disorders of Excessive Somnolence, and Sleep Hyperhydrosis. Items are rated on 5-point scale where 1=never and 5=always (daily). The total score is the sum of responses and ranges from 26 to 130; lower scores indicate healthier sleep-wake rhythms.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Sleep Disturbance Scale for Children (SDSC) Score	-7.5 score on a scale Standard Deviation 12.65	-6.89 score on a scale Standard Deviation 5.44	-0.13 score on a scale Standard Deviation 4.82

SECONDARY outcome

Timeframe: Baseline, Week 6

Used to measure adaptive functioning across three core domains (Communication, Daily Living Skills, and Socialization), and two optional domains (Motor Skills and Maladaptive Behavior); items are rated on a 3-point scale (0=never; 1=sometimes; 2=usually or often). The core domains sum to a total Adaptive Behavior Composite. The range of possible composite scores ranges from 20-140, with a higher score indicating higher levels of adaptive functioning.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Parent/Caregiver Form Score	-0.5 score on a scale Standard Deviation 3.89	4.33 score on a scale Standard Deviation 5.72	8.88 score on a scale Standard Deviation 7.32

SECONDARY outcome

Timeframe: Baseline, Week 6

1-item assessment of clinicians impression of severity of illness. Illness severity is rated on a 7-point scale ranging from 1=not at all to 7=among the most extremely ill.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Clinical Global Impression-Severity (CGI-S) Score	-0.5 score on a scale Standard Deviation 0.84	-1 score on a scale Standard Deviation 0.87	-0.5 score on a scale Standard Deviation 0.53

SECONDARY outcome

Timeframe: Baseline, Week 6

Parent/Caregiver form used to measure impact of autism interventions on family experience and quality of life. Items are rated on a 5-point scale where 1=always and 5=never. The total score is the sum of responses and ranges from 48 - 240; lower scores indicate better outcomes.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Autism Family Experience Questionnaire (AFEQ) Score	-3.17 score on a scale Standard Deviation 14.5	-14 score on a scale Standard Deviation 20.1	-8.88 score on a scale Standard Deviation 8.37

SECONDARY outcome

Timeframe: Baseline, Week 6

24-item Parent/Caregiver-completed form developed to detect symptoms of anxiety in youth with ASD. Items are rated on a 4-point scale (0=never and 3=always). The total score is the sum of responses and ranges from 0 to 72; lower scores indicate lesser symptoms of anxiety.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Anxiety Scale for Children - Autism Spectrum Disorder - Parent Versions (ASC-ASD-P) Score	-2.33 score on a scale Standard Deviation 4.76	-6.56 score on a scale Standard Deviation 9.84	-3.13 score on a scale Standard Deviation 6.15

SECONDARY outcome

Timeframe: Baseline, Week 6

24-item Child-completed form developed to detect symptoms of anxiety in youth with ASD. Items are rated on a 4-point scale (0=never and 3=always). The total score is the sum of responses and ranges from 0 to 72; lower scores indicate lesser symptoms of anxiety.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Anxiety Scale for Children - Autism Spectrum Disorder (ASC-ASD-C) - Child Version	-4 score on a scale Standard Deviation 6.81	-4.78 score on a scale Standard Deviation 5.61	-6.5 score on a scale Standard Deviation 11.2

SECONDARY outcome

Timeframe: Baseline, Week 6

Reflects clinicians impression of severity of illness on a 7-point scale ranging from 1=not present to 7="classic" autism.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Ohio State University (OSU) Autism Clinical Global Impressions: Severity	0 score on a scale Standard Deviation 0	-0.22 score on a scale Standard Deviation 0.67	-0.38 score on a scale Standard Deviation 0.52

SECONDARY outcome

Timeframe: Week 6

Reflects clinicians impression of improvement on a 7-point scale ranging from 1=very much improved to 7=very much worse.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
OSU Autism Clinical Global Impressions: Improvement	3.67 score on a scale Standard Deviation 0.82	2.78 score on a scale Standard Deviation 0.67	2.38 score on a scale Standard Deviation 0.52

SECONDARY outcome

Timeframe: Baseline, Week 6

38-item Parent/Caregiver-completed measure designed to assess rigid patterns of behavior commonly associated with ASD. Items are rated on a 6-point Likert scale where 0=Not at all a problem and 5=Very severe or extreme problem. The total score is the sum of responses and ranges from 0 to 228; higher scores indicate greater behavioral inflexibility.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Behavioral Inflexibility Scale (BIS) Score	-13 score on a scale Standard Deviation 28.61	-25.4 score on a scale Standard Deviation 40.54	-9.75 score on a scale Standard Deviation 13.73

SECONDARY outcome

Timeframe: Baseline, Week 6

24-item Parent/Caregiver-completed questionnaire measuring behavioral noncompliance in everyday settings over the past four weeks. Severity of non-compliance is rated on a 9-point Likert scale (where 1 = no interruption, and 9 = frequent/consistent interruption). The total score is the sum of responses and ranges from 24 to 216; higher scores indicate greater behavioral noncompliance.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD n=6 Participants Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 Participants Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Home Situations Questionnaire - Modified for ASD (HSQ-ASD) Score	-8.67 score on a scale Standard Deviation 40.51	-23.67 score on a scale Standard Deviation 20.02	-5.88 score on a scale Standard Deviation 6.73

SECONDARY outcome

Timeframe: Baseline, Week 6

Population: This outcome was not analyzed/assessed among participants in Part 1 of the trial (3 mg/kg and 6 mg/kg CBD arms).

SWAN is an 18-item Parent/Caregiver-completed measure designed to assess symptoms of ADHD in children and adolescents. Items are rated on a 7-point Likert scale, where Far Below Average = 3, Below Average = 2, Somewhat Below Average = 1, Average = 0, Somewhat Above Average = -1, Above Average = -2, and Far Above Average = -3. The total score is the sum of responses and ranges from -54 to 54; the lower the score, the higher the severity of ADHD symptoms.

Outcome measures

Outcome measures
Measure	3 mg/kg 98% Pure CBD Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=7 Participants Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Change in Strengths and Weaknesses of Attention-Deficit/Hyperactivity Disorder Symptoms and Normal Behavior (SWAN) Score	—	—	-2.71 score on a scale Standard Deviation 8.08

Adverse Events

3 mg/kg 98% Pure CBD

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

6 mg/kg 98% Pure CBD

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

9 mg/kg 98% Pure CBD

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Francisco X Castellanos, MD

NYU Langone Health

Phone: 646-754-5194

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Other adverse events
Measure	3 mg/kg 98% Pure CBD n=6 participants at risk Participants who received 98% pure CBD: 3 mg/kg/day in Part 1 of the study.	6 mg/kg 98% Pure CBD n=9 participants at risk Participants who received 98% pure CBD: 6 mg/kg/day in Part 1 of the study.	9 mg/kg 98% Pure CBD n=8 participants at risk Participants who received 98% pure CBD: 9 mg/kg/day in Part 2 of the study.
Cardiac disorders Palpitations/Tachycardia	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	25.0% 2/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Endocrine disorders Increased Salivation	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	50.0% 4/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Endocrine disorders Dry mouth	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Endocrine disorders Increased sweating	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Gastrointestinal disorders Appetite increase	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	25.0% 2/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Gastrointestinal disorders Appetite decrease	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
General disorders Weight gain	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Musculoskeletal and connective tissue disorders Muscle aches	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	11.1% 1/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Nervous system disorders Akathesia	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	37.5% 3/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Nervous system disorders Dystonia	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	11.1% 1/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Nervous system disorders Headache	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Nervous system disorders Increased stereotypies	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Nervous system disorders Paresthesias	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	11.1% 1/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Nervous system disorders Rigidity	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	11.1% 1/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Asthenia	33.3% 2/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Apathy	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Concentration difficulties	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	11.1% 1/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	25.0% 2/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Depressed Mood	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	11.1% 1/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Emotionality	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	33.3% 3/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Oppositionality	33.3% 2/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Increased sleep duration	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	44.4% 4/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	50.0% 4/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Decreased sleep duration	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Sleepiness/Sedation	50.0% 3/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Heavier Sleep	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	11.1% 1/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Increased Dream Activity	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	33.3% 3/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	50.0% 4/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Psychiatric disorders Sleep-onset Insomnia	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	12.5% 1/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Renal and urinary disorders Polyuria	16.7% 1/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	22.2% 2/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	25.0% 2/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.
Skin and subcutaneous tissue disorders Rash	0.00% 0/6 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	33.3% 3/9 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.	0.00% 0/8 • 6 weeks after participant received first dose. PI monitor for adverse events (AEs) at each follow-up visit. Study coordinator phone call to parents/caregivers to assess for AEs at weeks 1, 3, and 5. Participants and caregivers completed UKU Side Effects Rating Scale at weeks 2, 4, and 6. Participants were not assessed for all-cause mortality.