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Randomized Controlled Study of Donepezil in Fragile X Syndrome

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ClinicalTrials.gov Identifier: NCT01120626
Recruitment Status : Completed
First Posted : May 11, 2010
Results First Posted : March 17, 2016
Last Update Posted : March 17, 2016
Sponsor:
Collaborators:
National Institute of Mental Health (NIMH)
Autism Speaks
Information provided by (Responsible Party):
Allan Reiss, Stanford University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Fragile X Syndrome
Interventions Drug: donepezil
Drug: sugar pill
Enrollment 45
Recruitment Details  
Pre-assignment Details 45 participants consented/enrolled in study. Of these, 3 failed to meet inclusion criteria at the baseline visit and thus were not randomized to receive study drug.
Arm/Group Title Donepezil Sugar Pill
Hide Arm/Group Description

donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

donepezil: donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill: sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

Period Title: Overall Study
Started 20 22
Completed 19 22
Not Completed 1 0
Reason Not Completed
Withdrawal by Subject             1             0
Arm/Group Title Donepezil Sugar Pill Total
Hide Arm/Group Description

donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

donepezil: donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill: sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

Total of all reporting groups
Overall Number of Baseline Participants 20 22 42
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 22 participants 42 participants
<=18 years
10
  50.0%
9
  40.9%
19
  45.2%
Between 18 and 65 years
10
  50.0%
13
  59.1%
23
  54.8%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants 22 participants 42 participants
Female
7
  35.0%
8
  36.4%
15
  35.7%
Male
13
  65.0%
14
  63.6%
27
  64.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 20 participants 22 participants 42 participants
20 22 42
Contingency Naming Test (CNT) Performance Score   [1] 
Mean (Standard Deviation)
Unit of measure:  Correct responses per minute
Number Analyzed 20 participants 22 participants 42 participants
CNT Performance Rule 2 52.63  (18.54) 38.77  (26.49) 45.37  (23.83)
CNT Performance Rule 3 23.08  (12.56) 13.77  (13.44) 18.21  (13.71)
[1]
Measure Description: Baseline Contingency Naming Test (CNT) performance score on Rule 2 (naming shapes) and on Rule 3 (If the inside shape matches the outside shape, name the color, otherwise, name the outside shape). Performance score is the number of correct responses per minute, calculated by dividing the number of correct responses by the time taken to complete the 27 items, and multiplying by 60.
Aberrant Behavior Checklist (ABC)   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 20 participants 22 participants 42 participants
19.70  (15.34) 30.77  (25.54) 21.26  (19.50)
[1]
Measure Description: The Aberrant Behavior Checklist is a 58-item symptom checklist for assessing problem behaviors. The ABC was rated by each participant's parent. Each item is rated on a four-point Likert scale ranging from 0 (not at all a problem) to 3 (the problem is severe in degree). The ABC Total score (range 0-174) is the sum of all individual item scores. Higher score indicates more maladaptive behaviors/worse outcome.
1.Primary Outcome
Title Contingency Naming Test (CNT) Performance Score
Hide Description Week 12 Contingency Naming Test (CNT) performance score on Rule 2 (naming shapes) and on Rule 3 (If the inside shape matches the outside shape, name the color, otherwise, name the outside shape). Performance score is the number of correct responses per minute, calculated by dividing the number of correct responses by the time taken to complete the 27 items, and multiplying by 60. Higher scores indicate faster and more accurate responding.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
41 of 42 randomized participants completed the 12-week randomized controlled trial. 37 of 42 randomized participants completed CNT Rule 2 at week 12. 34 of 42 randomized participants completed CNT Rule 3 at week 12.
Arm/Group Title Donepezil Sugar Pill
Hide Arm/Group Description:

donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

donepezil: donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill: sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

Overall Number of Participants Analyzed 19 18
Mean (Standard Deviation)
Unit of Measure: correct responses per minute
CNT Performance Score Rule 2 37.05  (14.60) 40.50  (31.11)
CNT Performance Score Rule 3 66.94  (46.92) 61.56  (23.06)
2.Secondary Outcome
Title Aberrant Behavior Checklist (ABC)
Hide Description The Aberrant Behavior Checklist is a 58-item symptom checklist for assessing problem behaviors. The ABC was rated by each participant's parent. Each item is rated on a four-point Likert scale ranging from 0 (not at all a problem) to 3 (the problem is severe in degree). The ABC Total score (range 0-174) is the sum of all individual item scores. Higher score indicates more maladaptive behaviors/worse outcome.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
41 of 42 randomized participants completed the 12-week randomized controlled trial. 39 of 42 participants were administered the ABC at week 12.
Arm/Group Title Donepezil Sugar Pill
Hide Arm/Group Description:

donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

donepezil: donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill: sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

Overall Number of Participants Analyzed 18 21
Mean (Standard Deviation)
Unit of Measure: units on a scale
17.56  (13.65) 24.43  (23.27)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Donepezil Sugar Pill
Hide Arm/Group Description

donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

donepezil: donepezil (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

sugar pill: sugar pill (2.5 mg to 10.0 mg per day for 12 weeks)

All-Cause Mortality
Donepezil Sugar Pill
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Donepezil Sugar Pill
Affected / at Risk (%) Affected / at Risk (%)
Total   0/20 (0.00%)   0/22 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Donepezil Sugar Pill
Affected / at Risk (%) Affected / at Risk (%)
Total   0/20 (0.00%)   0/22 (0.00%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Allan Reiss, MD
Organization: Stanford University
Phone: 650-498-4538
Responsible Party: Allan Reiss, Stanford University
ClinicalTrials.gov Identifier: NCT01120626     History of Changes
Other Study ID Numbers: SU-02042010-4923
Autism Speaks 5907 ( Other Grant/Funding Number: Autism Speaks 5907 )
R34MH085899-01A1 ( U.S. NIH Grant/Contract )
SPO#42922 ( Other Identifier: Stanford University Research Management Group )
SPO#45612 ( Other Identifier: Stanford University Research Management Group )
eProtocol 13773 (SQL 96239) ( Other Identifier: Stanford University IRB )
First Submitted: May 7, 2010
First Posted: May 11, 2010
Results First Submitted: November 19, 2015
Results First Posted: March 17, 2016
Last Update Posted: March 17, 2016