Trial Outcomes & Findings for Pilot Study of Sleep Therapy and Biomarkers in Children With Autism Spectrum Disorders (NCT NCT02487082)

Last Updated: 2024-06-06

Results Overview

Subscale of the Aberrant Behavior Checklist (ABC). Subscales were derived by factor analysis (n=927). The checklist was first developed for use with severely mentally retarded individuals. More recently it has become widely used in research with children with autism. The ABC checklist is considered sensitive to drug effects. The Lethargy/Social Withdrawal subscale contains items such as: "Listless, sluggish, inactive" ; "Withdrawn, prefers solitary activities"; "Does not try to communicate by words or gestures"; "Shows few social reactions to others". The subscale consists of 16 items, each rated on a 4 point scale (0 = not at all, 3 = severe). Possible scores range from 0 to 48. Higher scores mean more lethargy and/or more social withdrawal.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Subscale rating was made by parent/caregiver every 5 weeks for a total of 5 times. Due to small N, only the endpoint of the study is reported here. Study endpoint (Visit 5) was after at least 18 wks, including 2 wks of washout, up to a maximum of 27 wks.

Results posted on

2024-06-06

Participant Flow

Most patients were recruited from the clinical practice of the Principal Investigator. We also posted flyers in the hospital outpatient clinics and sent "Dear Physician" recruitment letters to other pediatric neurologists and psychologists in our catchment area.

Participant milestones

Participant milestones
Measure	Group A Group A received an initial 5 week Run-In Period where other medications were withdrawn, and Placebo medication was given. Instructions for improving sleep hygiene were given during this phase. This Run-In Period was followed by Phase 1 (5 weeks) with active study medication (donepezil and melatonin) with dose based on subject age/weight. This was followed by Phase 2 (5 weeks) with continued study medication. At the conclusion of Phase 2, study medication was discontinued and a variable washout period (2-12 weeks) followed, ending with the next scheduled clinic visit with the Principal Investigator.	Group B Group B received an initial 5 week Run-In Period where other medications were withdrawn, and Placebo medication was given. Instructions for improving sleep hygiene were given during this phase. This Run-In Period was followed by Phase 1 (5 weeks)where Placebo medication was continued. This was followed by Phase 2 (5 weeks) with active study medication (donepezil and melatonin) with dose based on subject age/weight. At the conclusion of Phase 2, study medication was discontinued and a variable washout period (2-12 weeks) followed, ending with the next scheduled clinic visit with the Principal Investigator.
Run-In Phase (5 Weeks) STARTED	4	8
Run-In Phase (5 Weeks) COMPLETED	3	6
Run-In Phase (5 Weeks) NOT COMPLETED	1	2
Phase 1 (5 Weeks) STARTED	3	6
Phase 1 (5 Weeks) COMPLETED	2	6
Phase 1 (5 Weeks) NOT COMPLETED	1	0
Phase 2 (5 Weeks) STARTED	2	6
Phase 2 (5 Weeks) COMPLETED	2	6
Phase 2 (5 Weeks) NOT COMPLETED	0	0
Wash-Out Phase (2-12 Weeks) STARTED	2	6
Wash-Out Phase (2-12 Weeks) COMPLETED	2	6
Wash-Out Phase (2-12 Weeks) NOT COMPLETED	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Group A Group A received an initial 5 week Run-In Period where other medications were withdrawn, and Placebo medication was given. Instructions for improving sleep hygiene were given during this phase. This Run-In Period was followed by Phase 1 (5 weeks) with active study medication (donepezil and melatonin) with dose based on subject age/weight. This was followed by Phase 2 (5 weeks) with continued study medication. At the conclusion of Phase 2, study medication was discontinued and a variable washout period (2-12 weeks) followed, ending with the next scheduled clinic visit with the Principal Investigator.	Group B Group B received an initial 5 week Run-In Period where other medications were withdrawn, and Placebo medication was given. Instructions for improving sleep hygiene were given during this phase. This Run-In Period was followed by Phase 1 (5 weeks)where Placebo medication was continued. This was followed by Phase 2 (5 weeks) with active study medication (donepezil and melatonin) with dose based on subject age/weight. At the conclusion of Phase 2, study medication was discontinued and a variable washout period (2-12 weeks) followed, ending with the next scheduled clinic visit with the Principal Investigator.
Run-In Phase (5 Weeks) Withdrawal by Subject	1	2
Phase 1 (5 Weeks) Withdrawal by Subject	1	0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group A n=4 Participants Melatonin and donepezil (melatonin 3mg and donepezil 1.25-5mg based on age/weight each night) or placebo for 15 weeks Melatonin and Donepezil: The duration depends on group assignment. Placebo: The duration depends on group assignment.	Group B n=8 Participants Melatonin and donepezil (melatonin 3mg and donepezil 1.25-5mg based on age/weight each night) or placebo for 15 weeks Melatonin and Donepezil: The duration depends on group assignment. Placebo: The duration depends on group assignment.	Total n=12 Participants Total of all reporting groups
Age, Continuous	11 years n=4 Participants	9.5 years n=8 Participants	9.5 years n=12 Participants
Sex: Female, Male Female	0 Participants n=4 Participants	1 Participants n=8 Participants	1 Participants n=12 Participants
Sex: Female, Male Male	4 Participants n=4 Participants	7 Participants n=8 Participants	11 Participants n=12 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	4 participants n=4 Participants	8 participants n=8 Participants	12 participants n=12 Participants
ABC Lethargy/Social Withdrawal	8 units on a scale n=4 Participants	10 units on a scale n=8 Participants	9.5 units on a scale n=12 Participants
ABC Stereotypic Behavior	7.5 units on a scale n=4 Participants	7.5 units on a scale n=8 Participants	7.5 units on a scale n=12 Participants
Sleep Duration (hours)	8.2 hours n=4 Participants	7 hours n=8 Participants	7.1 hours n=12 Participants

PRIMARY outcome

Timeframe: Subscale rating was made by parent/caregiver every 5 weeks for a total of 5 times. Due to small N, only the endpoint of the study is reported here. Study endpoint (Visit 5) was after at least 18 wks, including 2 wks of washout, up to a maximum of 27 wks.