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 6 To 10 (NCT NCT00754013)

Results Overview

The primary objective of the study was evaluation of the efficacy and safety of donepezil hydrochloride in the treatment of the cognitive dysfunction exhibited by children with Down syndrome (DS), aged 6 to 10, as assessed by analysis of VABS-II/PCRF in the domains of communication, daily living skills, and socialization, and as assessed by standard safety measurements.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

9 participants

Primary outcome timeframe

Visit 0 (Screen), Visits 1 (Baseline), 2, and 3 (or Early Termination).

Results posted on

2020-01-28

Participant Flow

This study planned to recruit subjects at 26 centers in the United States. However, the study was terminated early and only 5 centers enrolled subjects during the period of 06 Oct 2008 to 10 Dec 2008.

Participant milestones

Participant milestones
Measure	Donepezil Donepezil was titrated to a dose of approximately 0.1-0.2 mg/kg/day as liquid containing 1 mg/1 mL of donepezil.	Placebo Donepezil matched placebo was titrated in the similar way as the donepezil arm.
Overall Study STARTED	4	5
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	4	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Donepezil Donepezil was titrated to a dose of approximately 0.1-0.2 mg/kg/day as liquid containing 1 mg/1 mL of donepezil.	Placebo Donepezil matched placebo was titrated in the similar way as the donepezil arm.
Overall Study Lost to Follow-up	1	1
Overall Study didn't return after study termed	3	4

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 6 To 10

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Donepezil n=4 Participants Donepezil was titrated to a dose of approximately 0.1-0.2 mg/kg/day as liquid containing 1 mg/1 mL of donepezil.	Placebo n=5 Participants Donepezil matched placebo was titrated in the similar way as the donepezil arm.	Total n=9 Participants Total of all reporting groups
Age, Continuous	9.0 years STANDARD_DEVIATION 0.8 • n=5 Participants	8.0 years STANDARD_DEVIATION 1.4 • n=7 Participants	8.5 years STANDARD_DEVIATION 0.3 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized White (n)	4 participants n=5 Participants	5 participants n=7 Participants	9 participants n=5 Participants

PRIMARY outcome

Timeframe: Visit 0 (Screen), Visits 1 (Baseline), 2, and 3 (or Early Termination).

Population: The planned efficacy analysis was on the intent-to-treat (ITT) population. This study was terminated early. Primary efficacy data were not analyzed since only 9 of the 140 planned subjects had been enrolled.

The primary objective of the study was evaluation of the efficacy and safety of donepezil hydrochloride in the treatment of the cognitive dysfunction exhibited by children with Down syndrome (DS), aged 6 to 10, as assessed by analysis of VABS-II/PCRF in the domains of communication, daily living skills, and socialization, and as assessed by standard safety measurements.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Visit 1 (Baseline) to Visit 3 (Week 10 or early termination).

Population: This study was terminated early. Secondary efficacy data were not analyzed since only 9 of the 140 planned subjects had been enrolled.

The planned secondary objectives of the study included further evaluation of efficacy as assessed by additional analyses of the VABS-II/PCRF, by analyses of the Test of Verbal Expression and Reasoning (TOVER), a subject-performance-based measure of expressive language function, and by the 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. In addition, observed case analyses of these assessments at Week 4 and Week 10 were planned.

Outcome measures

Outcome data not reported

Adverse Events

Donepezil

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Donepezil n=4 participants at risk Donepezil was titrated to a dose of approximately 0.1-0.2 mg/kg/day as liquid containing 1 mg/1 mL of donepezil.	Placebo n=5 participants at risk Donepezil matched placebo was titrated in the similar way as the donepezil arm.
Gastrointestinal disorders Bruxism	0.00% 0/4	20.0% 1/5
Gastrointestinal disorders Tongue disorder	0.00% 0/4	20.0% 1/5
Infections and infestations Upper respiratory infection	0.00% 0/4	20.0% 1/5
Nervous system disorders Headache	25.0% 1/4	20.0% 1/5
Psychiatric disorders Abnormal behavior	0.00% 0/4	20.0% 1/5

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