Down SyndromePhase 2NCT00675025

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

A Phase 2 study testing Donepezil hydrochloride (Aricept) in people with Down Syndrome, run by Eisai Inc. at 31 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 10 to 17 years
Conditions the study is for: Down Syndrome
What is being tested?: Donepezil hydrochloride (Aricept)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 117 participants
Where: 31 sitesUnited States
Study dates: 4 Apr 2008to 15 Dec 2008
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 10 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

31 sites in 1 country.

City, postcode or hospital

31 sites in 1 country

United States31 sites

Road Runner Research
Atlanta, Georgia, 30342
Neuropsychiatric Research Center of Southwest Florida
Bellevue, Washington, 98004
Medical Univ of South Carolina
Charleston, South Carolina, 29425
Metrohealth Medical Center
Cleveland, Ohio, 44109
Division of Genetics and Developmental Behavior
Durham, North Carolina, 27710
Hurley Medical Center
Flint, Michigan, 48503
Neufeld Medical Group, Inc.
Fort Myers, Florida, 33912
Saint Mary's Health Care
Grand Rapids, Michigan, 49503
Washington University School of Medicine
Grand Rapids, Michigan, 49503
Down Syndrome Clinic of Houston
Houston, Texas, 77030
Rocky Mountain Pediatrics, P.C.
Lakewood, Colorado, 80214
Meridien Research
Lincoln, Nebraska, 68504
Child Neurology Associates, PC
Little Rock, Arkansas, 72205
Midwest Children's Health Research Institute
Los Angeles, California, 90048
Miami Children's Hospital
Miami, Florida, 33155-3009
Miami Children's Hospital - Medical Genetics and Neuro-Developmental Center
Miami, Florida, 33155
Phoenix Children's Hospital
Miami, Florida, 33155
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232-9225
Children's Hospital and Research Center at Oakland
Oakland, California, 94609
University of California, Irvine Medical Center
Orange, California, 92868
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Regions Hospital
Saint Paul, Minnesota, 55101-2595
Alamo City Clinical Research, LLC
San Antonio, Texas, 78258
Community Research Foundation
San Antonio, Texas, 78258
UCSD Pediatric Pharmacology Research Unit
San Diego, California, 92123
Northwest Clinical Research Center
St Louis, Missouri, 63110
Clinical Studies Centers, LLC
St. Petersburg, Florida, 33709
Valko and Associates
Toledo, Ohio, 43606
Office of Lazlo Mate
Tulsa, Oklahoma, 74104
Clinical Research Center of New Jersey
Voorhees Township, New Jersey, 08043
Northwest Clinical Research Center
West Palm Beach, Florida, 33407

Other trials in United States

About this study

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

Read the 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.

Conditions: Down Syndrome
Being tested: Donepezil hydrochloride (Aricept)
Medical subject headings: Down Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Donepezil hydrochloride (Aricept)Drug
Also called Aricept
Liquid form Aricept - 5 mg/5 mL donepezil hydrochloride.

Study groups in NCT00675025
Study group	Role	What they receive
Prior Donepezil-DB	Experimental	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. Receives: Drug: Donepezil hydrochloride (Aricept)

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Visit 1 (baseline); Early Termination Visit (Week 36)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

4 Apr 200815 Dec 2008

First posted8 May 2008 (estimated)
Study start4 Apr 2008
Primary completion13 Nov 2008
Study completion15 Dec 2008

Why it stopped: Sufficient evidence of efficacy not met. Discontinuation not based on any safety concerns.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Down SyndromeStudies of the same condition, open and under way.
More trials run by Eisai Inc.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 117 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00675025
Sponsor's protocol ID: E2020-A001-220
Other IDs: A2501060
Status last verified: Mar 2021
First submitted: 7 May 2008
Last updated: 29 Mar 2021
Results posted: Yes, on ClinicalTrials.gov

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 29 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00675025
Acronym / sponsor ID: E2020-A001-220
Conditions: Down Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Sufficient evidence of efficacy not met. Discontinuation not based on any safety concerns.
Enrolment: 117 participants (actual)
Lead sponsor: Eisai Inc.Industry (pharma, biotech, devices)
Collaborators: Pfizer
Study start: 4 Apr 2008 (actual)
Primary completion: 13 Nov 2008 (actual)
Study completion: 15 Dec 2008 (actual)
First posted: 8 May 2008 (estimated)
Results: Posted 29 Mar 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Prior Donepezil-DB	Experimental	Drug: Donepezil 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.

Who may qualify

Ages: 10 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (2 listed)

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.

Key exclusion criteria (4 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Donepezil hydrochloride (Aricept)Also: AriceptLiquid form Aricept - 5 mg/5 mL donepezil hydrochloride.	Drug	Prior Donepezil-DB

Primary outcome

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
Time frame: Visit 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.

Results and safety

Results posted to the registry on 29 Mar 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Prior Donepezil-DB	54	0
Prior Placebo-DB	63	0

Primary outcome: 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

Time frame: Visit 1 (baseline); Early Termination Visit (Week 36). Mean (Standard Deviation). Unit: units on a scale

Measure	Prior Donepezil-DB n = 44	Prior Placebo-DB n = 45
Baseline (Week 10 of DB study)	87.73 (16.58)	92.02 (17.51)
Early termination visit	89.37 (20.9)	91.25 (18.45)

Population analysed: Analyses were performed on the Intent-to-treat population. The VABS-II/PCRF composite score, individual analyses of each of the 3 domains and the 9 sub-domains by both raw and standardized scores were not performed due to a lack of significant differences between donepezil and placebo subgroups; the data are provided as listings.

Adverse events

Time frame: Baseline up to early termination visit (Week 36). Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Prior Donepezil-DB	0 of 54 (0%)	37 of 54 (68.5%)	0 of 54
Prior Placebo-DB	0 of 63 (0%)	48 of 63 (76.2%)	0 of 63

Limitations and caveats (from the sponsor): This was an open-label trial that was terminated early by the Sponsor.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

31 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Donepezil hydrochloride (Aricept) in people with Down Syndrome, run by Eisai Inc. at 31 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 29 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 31 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Mar 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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 (time frame: Visit 1 (baseline); Early Termination Visit (Week 36)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00675025.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2021.