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NCT00570128

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

Completed Phase 2 Results posted Last updated 19 April 2021
What this trial tests

Phase 2 trial testing Donepezil HCl in Down Syndrome in 129 participants. Completed in 5 September 2008.

Timeline
16 November 2007
Primary endpoint
5 September 2008
5 September 2008

Quick facts

Lead sponsorEisai Inc.
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingdouble
Primary purposetreatment
Enrollment129
Start date16 November 2007
Primary completion5 September 2008
Estimated completion5 September 2008
Sites31 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Eisai Inc. — full company profile →

Who can join

Adults 10 to 17, any sex, with Down Syndrome. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Mean Change From Baseline in V-Scale Composite Score (Sum of 9 Sub-Domains) of Vineland Adaptive Behavior Scales Second Edition-Parent Caregiver Rating Form (VABS-II/PCRF) at Week 10-Last Observation Carried Forward (LOCF) Primary · Baseline, Week 10

The VABS-II/PCRF instrument was used in this study to assess 3 domains (each with 3 sub-domains): communication (sub-domains: receptive, expressive, and writing), daily living skills (sub-domains: personal, domestic, community), and socialization (sub-domains: interpersonal relationships, play/leisure time, coping skills). Raw scores (2=always present, 1=sometimes present, 0=seldom or never present) rated by the parent/caregiver from each sub-domain were converted to standardized scores called V-scores, which are based on age and a national sample of normal children. Each sub-domain v-scale sc

Baseline
GroupValue95% CI
Donepezil HCl83.1± 15.5
Placebo85.7± 15.8
Mean change from baseline at Week 10
GroupValue95% CI
Donepezil HCl4.74± 9.2
Placebo4.22± 8.5
Mean Change From Baseline in V-Scale Composite Score (Sum of 9 Sub-domains) of Vineland Adaptive Behavior Scales Second Edition-Parent Caregiver Rating Form (VABS-II/PCRF) at Week 4 and 10-Observed Cases (OC) Secondary · Baseline, Week 4 and Week 10

The VABS-II/PCRF instrument was used in this study to assess 3 domains (each with 3 sub-domains): communication (sub-domains: receptive, expressive, writing), daily living skills (sub-domains: personal, domestic, community), and socialization (sub-domains: interpersonal relationships, play/leisure time, coping skills). Raw scores (2=always present, 1=sometimes present, 0=seldom or never present) rated by the parent/caregiver from each sub-domain were converted to standardized scores called V-scores, which are based on age and a national sample of normal children. Each sub-domain v-scale score

Baseline
GroupValue95% CI
Donepezil HCl83.1± 15.5
Placebo85.7± 15.8
Mean change from baseline at Week 4
GroupValue95% CI
Donepezil HCl1.5± 6.4
Placebo2.6± 8.5
Mean change from baseline at Week 10
GroupValue95% CI
Donepezil HCl5.1± 9.6
Placebo4.2± 8.6
Mean Change From Baseline in Test of Verbal Expression and Reasoning (TOVER) Total Score at Week 4 and 10-OC Secondary · Baseline, Week 4 and Week 10

The TOVER is a participant-performance-based measure of expressive language function and verbal reasoning in response to questions about a series of stylized pictures showing identifiable scenarios. The 64-item test was specifically designed to assess language function in children and adults with down syndrome (DS) across a broad range of functional ability. The test used 23 multi-colored pictures to stimulate verbal responses to questions. The test was short (completed in 15 minutes) and fast-paced (2 to 4 questions per picture). Total score ranging from 0 to 64, was derived from 64 questions

Baseline
GroupValue95% CI
Donepezil HCl20.7± 12.2
Placebo21.6± 11.4
Mean change from baseline at Week 4
GroupValue95% CI
Donepezil HCl1.2± 6.4
Placebo0.9± 8.5
Mean change from baseline at Week 10
GroupValue95% CI
Donepezil HCl2.6± 6.2
Placebo1.9± 5.5
Mean Change From Baseline in Test of Verbal Expression and Reasoning (TOVER) Total Score at Week 10-LOCF Secondary · Baseline, Week 10

The TOVER is a participant-performance-based measure of expressive language function and verbal reasoning in response to questions about a series of stylized pictures showing identifiable scenarios. The 64-item test was specifically designed to assess language function in children and adults with DS across a broad range of functional ability. The test used 23 multi-colored pictures to stimulate verbal responses to questions. The test was short (completed in 15 minutes) and fast-paced (2 to 4 questions per picture). Total score ranging from 0 to 64, was derived from 64 questions, where higher s

Baseline
GroupValue95% CI
Donepezil HCl20.7± 12.2
Placebo21.6± 11.4
Mean change from baseline at Week 10
GroupValue95% CI
Donepezil HCl2.4± 6.0
Placebo2.1± 5.5

Adverse events — posted to ClinicalTrials.gov

Time frame: Baseline up to 10 months. Reporting threshold: 2%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Donepezil HCl
Serious: 0/64 (0%)
Deaths: 0/64
Placebo
Serious: 1/65 (2%)
Deaths: 0/65

Serious adverse events (2 terms)

ReactionSystemDonepezil HClPlacebo
GastroenteritisInfections and infestations
DehydrationMetabolism and nutrition disorders
Other adverse events (26 terms — click to expand)

ReactionSystemDonepezil HClPlacebo
DiarrheaGastrointestinal disorders
VomitingGastrointestinal disorders
Upper respiratory tract infectionInfections and infestations
NauseaGastrointestinal disorders
HeadacheNervous system disorders
NasopharyngitisInfections and infestations
CoughRespiratory, thoracic and mediastinal disorders
Ear infectionInfections and infestations
PharyngitisInfections and infestations
SinusitisInfections and infestations
Decreased appetiteMetabolism and nutrition disorders
RashSkin and subcutaneous tissue disorders
Abdominal pain, upperGastrointestinal disorders
ConstipationGastrointestinal disorders
Fecal incontinenceGastrointestinal disorders
FatigueGeneral disorders
PyrexiaGeneral disorders
BronchitisInfections and infestations
GastroenteritisInfections and infestations
Gastroenteritis viralInfections and infestations
Viral infectionInfections and infestations
LethargyNervous system disorders
SomnolenceNervous system disorders
InsomniaPsychiatric disorders
Nasal congestionRespiratory, thoracic and mediastinal disorders
Rhinitis allergicRespiratory, thoracic and mediastinal disorders

Most-reported serious reactions: Gastroenteritis, Dehydration.

Data from ClinicalTrials.gov NCT00570128 adverse events section.

Sponsor's own description

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 (DS). Effectiveness will be measured by rating communication, daily living skills, and social skills and relationships in subjects aged 10 to 17.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Timing of therapies for Down syndrome: the sooner, the better.
    Stagni F, Giacomini A, Guidi S, Ciani E, et al · · 2015 · cited 82× · PMID 26500515 · DOI 10.3389/fnbeh.2015.00265
  2. Aging with Down Syndrome-Where Are We Now and Where Are We Going?
    Alldred MJ, Martini AC, Patterson D, Hendrix J, et al · · 2021 · cited 29× · PMID 34682809 · DOI 10.3390/jcm10204687
  3. Challenges and Opportunities for Translation of Therapies to Improve Cognition in Down Syndrome.
    Lee SE, Duran-Martinez M, Khantsis S, Bianchi DW, et al · · 2020 · cited 18× · PMID 31706840 · DOI 10.1016/j.molmed.2019.10.001

Verify or expand the search:

Other recruiting trials for Down Syndrome

Currently open trials in the same condition.

Other Eisai Inc. trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00570128.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing