Donepezil HCl & Cognitive Deficits in Autism

• Sponsor: University of Pittsburgh (Other)
• Overall Status: Completed
• CT.gov ID: NCT00047697
• Collaborator: National Institute of Mental Health (NIMH) (NIH)
• Enrollment: 34
• Location: 1
• Arms: 2
• Duration (Months): 46
• Patients Per Site Per Month: 0.7

Study Details

Study Description

Brief Summary

This 11-week study will examine the safety and effectiveness of the medication donepezil (Aricept®) compared to placebo for treating cognitive deficits in children and adolescents with Autism Spectrum Disorder.

Condition or Disease	Intervention/Treatment	Phase
Autistic Disorder	Drug: Donepezil HCl; Drug: Placebo	Phase 2

Detailed Description

Children with autism spectrum disorder (ASD) often have impaired communication, problems with social interaction, and repetitive and stereotyped patterns of behavior. While most research has attempted to treat the behavioral deficits commonly associated with ASD, few studies have attempted to improve the core features of this disorder. A recent study found that donepezil HCl helped to improve speech production, attention span, and ability to express emotions in a group of children with autism. This study will provide an opportunity to conduct further testing of the effects of donepezil HCl on the cognitive deficits presumed to underlie the core features of ASD.

This study begins at Week 1 with a baseline assessment. Participants are then randomly assigned to either donepezil HCl or placebo. Participants will start with either a 5mg/day dose of donepezil HCl or placebo followed by a cognitive assessment after 4 weeks on this dose. Participants will then have their dose increased to 10mg/day. Another cognitive assessment will be given after 4 weeks on this dose.

Study Design

• Study Type: Interventional
• Actual Enrollment: 34 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Donepezil HCl: Treating Cognitive Deficits in Autism
• Study Start Date: Oct 1, 2002
• Actual Primary Completion Date: Aug 1, 2006
• Actual Study Completion Date: Aug 1, 2006

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Donepezil HCl Donepezil HCL 5 mg and 10 mg	Drug: Donepezil HCl; Participants will start with 5 mg per day dose of donepezil HCl, then have their dose increased to 10mg per day after 4 weeks.; Other Names: Aricept
Placebo Comparator: Placebo Placebo	Drug: Placebo; Placebo used in placed of Donepezil HCL

Outcome Measures

Primary Outcome Measures

1. Cognitive Assessment: TMT [8 weeks]

   TMT: Trial-Making Test. Time (sec) Range: 0 - 300. Lower = better

2. Cognitive Assessment: EOWVT Standard Score [8 weeks]

   Expressive One Word Vocabulary Test (standard score) Range: 55-140. Higher = better

3. Cognitive Assessment: CVLT [8 weeks]

   California Verbal Learning Test (percent of correct answers) Range: 0-100. Higher = better

Eligibility Criteria

Criteria

• Ages Eligible for Study: 8 Years to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Autism Spectrum Disorder (ASD)

• Asperger's Disorder

• IQ of 75 or above

• Baseline assessment tests within the acceptable range

Exclusion Criteria:

• Bipolar disorder, schizophrenia, schizoaffective disorder, or psychotic disorder

• Seizure disorder requiring the use of anticonvulsant medications

• Congenital rubella, cytomegalovirus, or tuberous sclerosis

• Certain medications prescribed for management of behavior (please contact the investigator for a complete list)

• Medications/preparations that are known to interact with donepezil HCl

• Significant medical illness, endocrinopathies, cardiovascular disease, or severe chronic malnutrition

• Pregnancy or sexually active females not using a reliable method of contraception

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Western Psychiatric Institute & Clinic	Pittsburgh	Pennsylvania	United States	15213

Sponsors and Collaborators

• University of Pittsburgh
• National Institute of Mental Health (NIMH)

Investigators

• Principal Investigator: Benjamin L. Handen, PhD, University of Pittsburgh, School of Medicine, Department of Psychiatry

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Benjamin L Handen, PhD, BCBA-D, Assistant Professor of Psychiatry & Pediatric, University of Pittsburgh
• ClinicalTrials.gov Identifier: NCT00047697
• Other Study ID Numbers: R21MH064941; R21MH064941; DSIR CT-M3
• First Posted: Oct 16, 2002
• Last Update Posted: Oct 5, 2017
• Last Verified: Sep 1, 2017

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Additional relevant MeSH terms: Autistic Disorder; Cognition Disorders; Cognitive Dysfunction; Donepezil

Study Results

Participant Flow

Arm/Group Title	Donepezil HCL	Placebo
Arm/Group Description	Subjects placed on 5 and 10 mg of donepezil	Subjects placed on 5 mg or 10 mg of placebo
Period Title: Overall Study
STARTED	18	16
COMPLETED	18	14
NOT COMPLETED	0	2

Baseline Characteristics

Arm/Group Title	Donepezil HCL	Placebo	Total
Arm/Group Description	Donepezil HCl: Participants will start with 5 mg per day dose of donepezil HCl, then have their dose increased to 10mg per day after 4 weeks.	Participants will start with 5 mg/day of placebo, then have their doses increased to 10 mg/day of placebo after 4 weeks.	Total of all reporting groups
Overall Participants	18	16	34
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	11.6	11.8	11.7
Sex: Female, Male (Count of Participants)
Female	1 5.6%	2 12.5%	3 8.8%
Male	17 94.4%	14 87.5%	31 91.2%
Cognitive Assessment: TMT (Seconds) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Seconds]	123.6 (56.5)	154.9 (77.8)	138.3 (66.5)
Cognitive Assessment: EOWVT Standard score (units on a scale) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [units on a scale]	104.6 (22.4)	108.7 (17.0)	106.5 (19.9)
Cognitive Assessment: CVLT (Correct answers, percentage) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Correct answers, percentage]	40.7 (14.1)	47.1 (10.3)	43.7 (12.3)

Outcome Measures

1. Primary Outcome

Title	Cognitive Assessment: TMT
Description	TMT: Trial-Making Test. Time (sec) Range: 0 - 300. Lower = better
Time Frame	8 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Donepezil HCl	Placebo
Arm/Group Description	Participants will start with 5 mg per day dose of donepezil HCl, then have their dose increased to 10mg per day after 4 weeks.	Placebo used in placed of Donepezil HCL
Measure Participants	18	16
Mean (Standard Deviation) [seconds]	118.3 (79.1)	104.7 (53.4)

2. Primary Outcome

Title	Cognitive Assessment: EOWVT Standard Score
Description	Expressive One Word Vocabulary Test (standard score) Range: 55-140. Higher = better
Time Frame	8 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Donepezil HCl	Placebo
Arm/Group Description	Participants will start with 5 mg per day dose of donepezil HCl, then have their dose increased to 10mg per day after 4 weeks.	Placebo used in place of Donepezil HCL
Measure Participants	18	16
Mean (Standard Deviation) [units on a scale]	109.7 (21.0)	114.5 (16.1)

3. Primary Outcome

Title	Cognitive Assessment: CVLT
Description	California Verbal Learning Test (percent of correct answers) Range: 0-100. Higher = better
Time Frame	8 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Donepezil HCl	Placebo
Arm/Group Description	Participants will start with 5 mg per day dose of donepezil HCl, then have their dose increased to 10mg per day after 4 weeks.	Placebo used in place of Donepezil HCL
Measure Participants	18	16
Mean (Standard Deviation) [percentage of correct answers]	50.2 (11.0)	51.3 (14.7)

Adverse Events

Time Frame	AEs were collected for 10 weeks (and for longer if a subject had an active AE at the end of the 10-week trial).
Arm/Group Title	Donepezil HCL		Placebo
Arm/Group Description	5 mg of donepezil for 4 weeks, followed by 10 mg of donepezil for 6 weeks.		Placebo substituted for Donepezil HCL
All Cause Mortality
	Donepezil HCL		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/18 (0%)		0/16 (0%)
Serious Adverse Events
	Donepezil HCL		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/18 (0%)		0/16 (0%)
Other (Not Including Serious) Adverse Events
	Donepezil HCL		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/18 (0%)		0/16 (0%)

Limitations/Caveats

The trial may not have been long enough to obtain clinically significant effects

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.