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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

Primary Purpose

Down Syndrome

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Donepezil hydrochloride (Aricept)
Sponsored by
Eisai Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Down Syndrome

Eligibility Criteria

10 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Phoenix Children's Hospital
  • Child Neurology Associates, PC
  • Midwest Children's Health Research Institute
  • Children's Hospital and Research Center at Oakland
  • University of California, Irvine Medical Center
  • UCSD Pediatric Pharmacology Research Unit
  • Rocky Mountain Pediatrics, P.C.
  • Neufeld Medical Group, Inc.
  • Miami Children's Hospital
  • Miami Children's Hospital - Medical Genetics and Neuro-Developmental Center
  • Phoenix Children's Hospital
  • Clinical Studies Centers, LLC
  • Northwest Clinical Research Center
  • Road Runner Research
  • Hurley Medical Center
  • Saint Mary's Health Care
  • Washington University School of Medicine
  • Regions Hospital
  • Northwest Clinical Research Center
  • Meridien Research
  • Clinical Research Center of New Jersey
  • Division of Genetics and Developmental Behavior
  • Metrohealth Medical Center
  • Valko and Associates
  • Office of Lazlo Mate
  • Medical Univ of South Carolina
  • Vanderbilt Children's Hospital
  • Down Syndrome Clinic of Houston
  • Alamo City Clinical Research, LLC
  • Community Research Foundation
  • Neuropsychiatric Research Center of Southwest Florida

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Prior Donepezil-DB

Arm Description

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.

Outcomes

Primary Outcome Measures

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-scores
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

Full Information

First Posted
May 7, 2008
Last Updated
March 26, 2021
Sponsor
Eisai Inc.
Collaborators
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT00675025
Brief Title
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
Official Title
An Open-label Study to Evaluate the Safety of Donepezil Hydrochloride((Aricept®) for up to 1 Year in the Treatment of Cognitive Dysfunction Exhibited by Children With Down Syndrome-Follow-up to a 10-Week,Double-Blind,Placebo-Controlled Trial
Study Type
Interventional

2. Study Status

Record Verification Date
March 2021
Overall Recruitment Status
Terminated
Why Stopped
Sufficient evidence of efficacy not met. Discontinuation not based on any safety concerns.
Study Start Date
April 4, 2008 (Actual)
Primary Completion Date
November 13, 2008 (Actual)
Study Completion Date
December 15, 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Eisai Inc.
Collaborators
Pfizer

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Down Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
117 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Prior Donepezil-DB
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Donepezil hydrochloride (Aricept)
Other Intervention Name(s)
Aricept
Intervention Description
Liquid form Aricept - 5 mg/5 mL donepezil hydrochloride.
Primary Outcome Measure Information:
Title
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-scores
Description
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.
Time Frame
Visit 1 (baseline); Early Termination Visit (Week 36)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Child Neurology Associates, PC
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
Midwest Children's Health Research Institute
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Children's Hospital and Research Center at Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
University of California, Irvine Medical Center
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
UCSD Pediatric Pharmacology Research Unit
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Rocky Mountain Pediatrics, P.C.
City
Lakewood
State/Province
Colorado
ZIP/Postal Code
80214
Country
United States
Facility Name
Neufeld Medical Group, Inc.
City
Fort Myers
State/Province
Florida
ZIP/Postal Code
33912
Country
United States
Facility Name
Miami Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155-3009
Country
United States
Facility Name
Miami Children's Hospital - Medical Genetics and Neuro-Developmental Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Phoenix Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Clinical Studies Centers, LLC
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33709
Country
United States
Facility Name
Northwest Clinical Research Center
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33407
Country
United States
Facility Name
Road Runner Research
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Hurley Medical Center
City
Flint
State/Province
Michigan
ZIP/Postal Code
48503
Country
United States
Facility Name
Saint Mary's Health Care
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Facility Name
Washington University School of Medicine
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Facility Name
Regions Hospital
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55101-2595
Country
United States
Facility Name
Northwest Clinical Research Center
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Meridien Research
City
Lincoln
State/Province
Nebraska
ZIP/Postal Code
68504
Country
United States
Facility Name
Clinical Research Center of New Jersey
City
Voorhees
State/Province
New Jersey
ZIP/Postal Code
08043
Country
United States
Facility Name
Division of Genetics and Developmental Behavior
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Metrohealth Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44109
Country
United States
Facility Name
Valko and Associates
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Facility Name
Office of Lazlo Mate
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74104
Country
United States
Facility Name
Medical Univ of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Vanderbilt Children's Hospital
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232-9225
Country
United States
Facility Name
Down Syndrome Clinic of Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Alamo City Clinical Research, LLC
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78258
Country
United States
Facility Name
Community Research Foundation
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78258
Country
United States
Facility Name
Neuropsychiatric Research Center of Southwest Florida
City
Bellevue
State/Province
Washington
ZIP/Postal Code
98004
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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