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Evaluating The Safety Of Donepezil Hydrochloride (Aricept) For Up To 1 Year In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome - Follow-Up To A 10-Week, Double-Blind, Placebo-Controlled Trial

Phase 2
Terminated
Conditions
Down Syndrome
Interventions
Registration Number
NCT00675025
Lead Sponsor
Eisai Inc.
Brief Summary

The purpose of this study is to determine the safety of donepezil hydrochloride (Aricept) in children with Down syndrome who have finished the preceding 10-week, double-blind study of donepezil hydrochloride. Medical tests for drug safety will be conducted at each clinic visit.

Detailed Description

All children in this study will take donepezil hydrochloride once a day, and will come back to the clinic at 8, 24 and 42 weeks after the study has begun. In between these clinic visits, the clinical staff will telephone the child's parent or caregiver to discuss drug safety observations and possible changes in drug dose. At the end of the study (42 weeks), psychological testing will also be given to determine how well the child is functioning.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
117
Inclusion Criteria
  • Diagnosis of Down syndrome (established during study E2020-A001-220).
  • Completion of study E2020-A001-219 (NCT00570128 [also known as A2501059]) with no ongoing seroius adverse events and no severe drug reactions.
Exclusion Criteria
  • Weight less than 20 kg.
  • Clinically significant conditions affecting absorption, distribution or metabolism of the study medication.
  • No reliable parent or caregiver, or unwillingness or inability of parent or caregiver to fulfill the requirements of the study.
  • Females of childbearing potential who are not practicing an effective means of birth control.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Prior Donepezil-DBDonepezil hydrochloride (Aricept)All participants started with a dose of 2.5 mg/day (2.5 mL/day). Dose escalations occurred in 2.5 mg/day increments every 2 weeks (steady state levels assumed to have been reached) to a maximum dose of 10 mg/day, according to the participant's weight schedule and the Investigator's judgment of safety and tolerability. Re-titration was done to maintain the blinding of the double-blind study (E2020-A001-219). Doses could be decreased due to tolerability and could be increased or decreased to maintain a maximum dose of 0.1 to 0.2 mg/kg/day based on the participant's weight at clinic visits during the study duration.
Primary Outcome Measures
NameTimeMethod
Change From Visit 1 (Baseline) to Visit 4 or Early Termination in the Vineland Adaptive Behavior Scales, 2nd Edition, Parent/Caregiver Rating Form (VABS-II/PCRF) Sum of the 9 Sub-domain V-scoresVisit 1 (baseline); Early Termination Visit (Week 36)

VABS-II/PCRF assessed participant's adaptive behaviors on 3 domains (each has 3 sub-domains): Communication (receptive, expressive, written), Daily Living Skills (personal, domestic, community), Socialization (interpersonal relationships, play a leisure time, coping skills). Parent/caregiver rated participant's behavior for sub-domains from 0 (never present) to 2 (always present). Raw scores from sub-domains converted into standardized score(V-scale scores) ranged:1-24 for each sub-domain, mean=15,standard deviation(SD)=3,higher scores=higher level of adaptive functioning and were summed to obtain V-scale composite score ranged 9-216, mean=100,SD=15,higher scores=higher level of adaptive functioning, positive change=improvement in adaptive functioning. Composite and individual analyses, both raw and standardized scores, were not performed due to lack of significant differences between donepezil and placebo in parent study.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (29)

Metrohealth Medical Center

🇺🇸

Cleveland, Ohio, United States

Phoenix Children's Hospital

🇺🇸

Miami, Florida, United States

UCSD Pediatric Pharmacology Research Unit

🇺🇸

San Diego, California, United States

Miami Children's Hospital - Medical Genetics and Neuro-Developmental Center

🇺🇸

Miami, Florida, United States

Miami Children's Hospital

🇺🇸

Miami, Florida, United States

Vanderbilt Children's Hospital

🇺🇸

Nashville, Tennessee, United States

Child Neurology Associates, PC

🇺🇸

Little Rock, Arkansas, United States

Midwest Children's Health Research Institute

🇺🇸

Los Angeles, California, United States

Children's Hospital and Research Center at Oakland

🇺🇸

Oakland, California, United States

University of California, Irvine Medical Center

🇺🇸

Orange, California, United States

Neufeld Medical Group, Inc.

🇺🇸

Fort Myers, Florida, United States

Clinical Studies Centers, LLC

🇺🇸

Saint Petersburg, Florida, United States

Northwest Clinical Research Center

🇺🇸

Saint Louis, Missouri, United States

Hurley Medical Center

🇺🇸

Flint, Michigan, United States

Saint Mary's Health Care

🇺🇸

Grand Rapids, Michigan, United States

Road Runner Research

🇺🇸

Atlanta, Georgia, United States

Clinical Research Center of New Jersey

🇺🇸

Voorhees, New Jersey, United States

Meridien Research

🇺🇸

Lincoln, Nebraska, United States

Office of Lazlo Mate

🇺🇸

Tulsa, Oklahoma, United States

Division of Genetics and Developmental Behavior

🇺🇸

Durham, North Carolina, United States

Valko and Associates

🇺🇸

Toledo, Ohio, United States

Medical Univ of South Carolina

🇺🇸

Charleston, South Carolina, United States

Down Syndrome Clinic of Houston

🇺🇸

Houston, Texas, United States

Alamo City Clinical Research, LLC

🇺🇸

San Antonio, Texas, United States

Community Research Foundation

🇺🇸

San Antonio, Texas, United States

Neuropsychiatric Research Center of Southwest Florida

🇺🇸

Bellevue, Washington, United States

Rocky Mountain Pediatrics, P.C.

🇺🇸

Lakewood, Colorado, United States

Regions Hospital

🇺🇸

Saint Paul, Minnesota, United States

Washington University School of Medicine

🇺🇸

Grand Rapids, Michigan, United States

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