Trial Outcomes & Findings for Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17 (NCT NCT00754052)

Results Overview

Mean change from Baseline from Visit 1 (baseline) to Visit 3 (Week 10 or early termination) in VABS-11/PCRF, a sum of the 9 sub-domain v-scores (3 scores for each of the communication, daily living skills, and socialization domains) using last observation carried forward was planned.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

8 participants

Primary outcome timeframe

Baseline (Day 0) to Visit 3 (Week 10) or at early termination

Results posted on

2018-04-23

Participant Flow

Participant milestones

Participant milestones
Measure	Donepezil Hydrochloride Blinded oral donepezil hydrochloride, was started at 2.5 mL daily, followed by 2-week titration intervals over a period of 6 weeks until a maximum dose of 5 milligrams per kilograms per day (mg/kg/day) or 10 mg/kg/day was reached. Due to early termination of the study no participant reached either targeted maximum.	Placebo Participants received matching placebo in a 1:1:1 ratio.
Overall Study STARTED	5	3
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	5	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Donepezil Hydrochloride Blinded oral donepezil hydrochloride, was started at 2.5 mL daily, followed by 2-week titration intervals over a period of 6 weeks until a maximum dose of 5 milligrams per kilograms per day (mg/kg/day) or 10 mg/kg/day was reached. Due to early termination of the study no participant reached either targeted maximum.	Placebo Participants received matching placebo in a 1:1:1 ratio.
Overall Study Study terminated by Sponsor	5	3

Baseline Characteristics

Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Donepezil Hydrochloride n=5 Participants Blinded oral donepezil hydrochloride, was started at 2.5 mL daily, followed by 2-week titration intervals over a period of 6 weeks until a maximum dose of 5 mg/kg/day or 10 mg/kg/day was reached. Due to early termination of the study no participant reached either targeted maximum.	Placebo n=3 Participants Participants received matching placebo in a 1:1:1 ratio.	Total n=8 Participants Total of all reporting groups
Age, Continuous	12.8 Years n=5 Participants	15.0 Years n=7 Participants	13.6 Years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (Day 0) to Visit 3 (Week 10) or at early termination

Population: Because the study was terminated early, only 8 subjects were enrolled. Analyses were not performed due to study medication exposure being limited and variable, limited efficacy data were collected, and no participant reached their maximum targeted dose.

Mean change from Baseline from Visit 1 (baseline) to Visit 3 (Week 10 or early termination) in VABS-11/PCRF, a sum of the 9 sub-domain v-scores (3 scores for each of the communication, daily living skills, and socialization domains) using last observation carried forward was planned.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Day 0) to Visit 3 (Week 10) or early termination

Population: Because the study was terminated early, only 8 subjects were enrolled. Analyses were not performed due to study medication exposure being limited and variable, limited efficacy data were collected, and no participant reached their maximum targeted dose.

Additional analyses of the VABS-II/PCRF were planned.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Day 0) to Visit 3 (Week 10) or early termination

Population: Because the study was terminated early, only 8 subjects were enrolled. Analyses were not performed due to study medication exposure being limited and variable, limited efficacy data were collected, and no participant reached their maximum targeted dose.

TOVER, a subject performance-based measure of expressive language function was planned.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline (Day 0) to Visit 3 (Week 10) or early termination

Population: Because the study was terminated early, only 8 subjects were enrolled. Analyses were not performed due to study medication exposure being limited and variable, limited efficacy data were collected, and no participant reached their maximum targeted dose.

Forward Memory and Attention Sustained sub-tests of the Leiter International Performance Scale - Revised (Leiter-R), a cognitive assessment instrument for children and adolescents that is not language dependent was planned.

Outcome measures

Outcome data not reported

Adverse Events

Donepezil Hydrochloride

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Donepezil Hydrochloride n=5 participants at risk Blinded oral donepezil hydrochloride, was started at 2.5 mL daily, followed by 2-week titration intervals over a period of 6 weeks until a maximum dose of 5 mg/kg/day or 10 mg/kg/day was reached. Due to early termination of the study no participant reached either targeted maximum.	Placebo n=3 participants at risk Participants received matching placebo in a 1:1:1 ratio.
Gastrointestinal disorders Nausea	20.0% 1/5 • Adverse events (AEs) and treatment emergent AEs (TEAEs) were collected from the time of screening (prior to start of study medication) until termination of the study. The Safety Population included all 8 participants who received at least 1 dose of study medication.	33.3% 1/3 • Adverse events (AEs) and treatment emergent AEs (TEAEs) were collected from the time of screening (prior to start of study medication) until termination of the study. The Safety Population included all 8 participants who received at least 1 dose of study medication.
General disorders Diarrhea	20.0% 1/5 • Adverse events (AEs) and treatment emergent AEs (TEAEs) were collected from the time of screening (prior to start of study medication) until termination of the study. The Safety Population included all 8 participants who received at least 1 dose of study medication.	33.3% 1/3 • Adverse events (AEs) and treatment emergent AEs (TEAEs) were collected from the time of screening (prior to start of study medication) until termination of the study. The Safety Population included all 8 participants who received at least 1 dose of study medication.
Infections and infestations Ear infection	20.0% 1/5 • Adverse events (AEs) and treatment emergent AEs (TEAEs) were collected from the time of screening (prior to start of study medication) until termination of the study. The Safety Population included all 8 participants who received at least 1 dose of study medication.	0.00% 0/3 • Adverse events (AEs) and treatment emergent AEs (TEAEs) were collected from the time of screening (prior to start of study medication) until termination of the study. The Safety Population included all 8 participants who received at least 1 dose of study medication.

Additional Information

Eisai Medical Services

Eisai Inc.

Phone: 1-888-422-4743

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER