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Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome

This study has been completed.
Sponsor:
Collaborator:
Pfizer
Information provided by:
Eisai Inc.
ClinicalTrials.gov Identifier:
NCT00570128
First received: December 6, 2007
Last updated: August 4, 2009
Last verified: August 2009
  Purpose

The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome. Effectiveness will be measured by rating communication, daily living skills, and social skills and relationships in subjects aged 10 to 17.


Condition Intervention Phase
Down Syndrome
Drug: Donepezil hydrochloride
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: A 10-Week, Double-Blind, Placebo-Controlled Study To Evaluate The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome

Resource links provided by NLM:


Further study details as provided by Eisai Inc.:

Primary Outcome Measures:
  • Vineland-II Adaptive Behavior Scale (VABS-II); Parent/Caregiver Rating Form (PCRF) [ Time Frame: Screen, Baseline, Week 4 and Week 10 or Early Termination. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The Test of Verbal Expression and Reasoning (TOVER); Population PK (drug levels in the body), PD (drug effects) and safety will also be assessed. [ Time Frame: Baseline, Week 4 and Week 10 or Early Termination. ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 150
Study Start Date: December 2007
Study Completion Date: December 2008
Primary Completion Date: September 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1 Drug: Donepezil hydrochloride
Oral liquid.
Other Name: Aricept
Placebo Comparator: 2 Drug: Placebo
Oral liquid.

  Eligibility

Ages Eligible for Study:   10 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ages 10 to 17 years old, weight more than or equal to 20kg
  • Male and female
  • VABS-II/PCRF standard composite score >55
  • Diagnosis of DS (trisomy 21) documented by chromosomal analysis (karyotyping). If such documentation is not available at screening, karyotyping will be performed with the screening labs and must be documented prior to baseline visit.
  • Naïve to approved or unapproved cholinesterase inhibitors is preferred however, prior use of these medications is allowed, provided that the medication was discontinued at least 3 months prior to screening and that it was not discontinued for lack of tolerability or efficacy or for the sole purpose of enrolling the subject in the study.
  • Subjects residing in the community
  • Must be expected to complete all procedures scheduled during the Screening and Baseline visits including all efficacy and safety parameters.
  • Must speak English and be verbal and able to be understood most of the time and must not use other forms of communication, signs, symbol boards or devices to supplement his/her communication ability
  • Must have a parent or other reliable caregiver who agrees to accompany the subject to all clinic visits, provide information about the subject as required by the protocol, and ensure compliance with the medication schedule
  • a Parent or Caregiver must be a constant and reliable informant with sufficient contact with the subject to have detailed knowledge of the subject's adaptive behavior in order to be able to complete the VABS-II/PCRF accurately. The same individual should complete the form at every visit.
  • Should be in good general health with no medical conditions that are considered both clinically significant and unstable
  • Clinical laboratory values within normal limits or abnormalities considered not clinically significant by the investigator and sponsor
  • Stable Type I (insulin-dependent) or Type II diabetes are eligible provided they are monitored regularly prior to and during the study to ensure adequate glucose control (fasting blood glucose < 140mg/dl and glycosylated hemoglobin [hemoglobin A1c] <8% at screening).
  • Thyroid disease also may be included in the study provided they are euthyroid and stable on treatment for at least 3 months prior to screening.
  • History of seizure disorder is allowed provided that subjects are on stable treatment for at least 3 months and have not had a seizure within the past 6 months.
  • Independent in ambulation or ambulatory aided (i.e., walker or cane, wheelchair), vision and hearing (eyeglasses and/ or hearing aid permissible) sufficient for achieving VABS-II/PCRF composite standard scores >55 and for cooperating with examinations and the TOVER.

Exclusion Criteria:

  • Ages <10 or >17 years
  • Active or clinically significant conditions affecting absorption, distribution or metabolism of the study medication (e.g., inflammatory bowel disease, gastric or duodenal ulcers or severe lactose intolerance)
  • Known hypersensitivity to piperidine derivatives or cholinesterase inhibitors
  • Currently receiving cholinesterase inhibitors or who have received them in the 3 months prior to screening or with prior use >3 months prior to screening who stopped for lack of efficacy or tolerability
  • No reliable parent or caregiver, or subjects, or caregivers who are unwilling or unable to complete any of the outcome measures and fulfill the requirements of this study
  • Clinically significant obstructive pulmonary disease or asthma untreated or not controlled by treatment within 3 months prior to screening
  • Recent (less than or equal to 2 years) hematologic/oncologic disorders (mild anemia allowed)
  • Evidence of active, clinically significant, and unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system disease
  • Current DSM-IV-TR diagnosis of Major Depressive Disorder (MDD) or any current primary psychiatric diagnosis other than DS (as per DSM-IV)
  • Any condition which would make the subject or the caregiver, in the opinion of the investigator, unsuitable for the study
  • Unsuitability which includes female subjects who have begun menstruation and are thus of child-bearing potential, who may be sexually active and who are not practicing an effective means of birth control.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00570128

  Show 31 Study Locations
Sponsors and Collaborators
Eisai Inc.
Pfizer
Investigators
Study Director: Anita Murthy Eisai Inc.
  More Information

No publications provided

Responsible Party: Eisai Medical Services, Eisai Inc.
ClinicalTrials.gov Identifier: NCT00570128     History of Changes
Other Study ID Numbers: E2020-A001-219
Study First Received: December 6, 2007
Last Updated: August 4, 2009
Health Authority: United States: Food and Drug Administration

Keywords provided by Eisai Inc.:
Down Syndrome
trisomy 21

Additional relevant MeSH terms:
Down Syndrome
Cognition Disorders
Syndrome
Abnormalities, Multiple
Chromosome Disorders
Congenital Abnormalities
Delirium, Dementia, Amnestic, Cognitive Disorders
Disease
Genetic Diseases, Inborn
Intellectual Disability
Mental Disorders
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Pathologic Processes
Donepezil
Central Nervous System Agents
Cholinergic Agents
Cholinesterase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Nootropic Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014