MedDataX Logo

Trial Outcomes & Findings for Randomized Controlled Study of Donepezil in Fragile X Syndrome (NCT NCT01120626)

NCT ID: NCT01120626

Last Updated: 2016-03-17

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

45 participants

Primary outcome timeframe

Week 12

Results posted on

2016-03-17

Participant Flow

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.

Participant milestones

Participant milestones
Measure
Donepezil
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
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 Study
STARTED
20
22
Overall Study
COMPLETED
19
22
Overall Study
NOT COMPLETED
1
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Donepezil
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
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 Study
Withdrawal by Subject
1
0

Baseline Characteristics

Randomized Controlled Study of Donepezil in Fragile X Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Donepezil
n=20 Participants
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
n=22 Participants
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
n=42 Participants
Total of all reporting groups
Age, Categorical
<=18 years
10 Participants
n=93 Participants
9 Participants
n=4 Participants
19 Participants
n=27 Participants
Age, Categorical
Between 18 and 65 years
10 Participants
n=93 Participants
13 Participants
n=4 Participants
23 Participants
n=27 Participants
Age, Categorical
>=65 years
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Sex: Female, Male
Female
7 Participants
n=93 Participants
8 Participants
n=4 Participants
15 Participants
n=27 Participants
Sex: Female, Male
Male
13 Participants
n=93 Participants
14 Participants
n=4 Participants
27 Participants
n=27 Participants
Region of Enrollment
United States
20 participants
n=93 Participants
22 participants
n=4 Participants
42 participants
n=27 Participants
Contingency Naming Test (CNT) Performance Score
CNT Performance Rule 2
52.63 correct responses per minute
STANDARD_DEVIATION 18.54 • n=93 Participants
38.77 correct responses per minute
STANDARD_DEVIATION 26.49 • n=4 Participants
45.37 correct responses per minute
STANDARD_DEVIATION 23.83 • n=27 Participants
Contingency Naming Test (CNT) Performance Score
CNT Performance Rule 3
23.08 correct responses per minute
STANDARD_DEVIATION 12.56 • n=93 Participants
13.77 correct responses per minute
STANDARD_DEVIATION 13.44 • n=4 Participants
18.21 correct responses per minute
STANDARD_DEVIATION 13.71 • n=27 Participants
Aberrant Behavior Checklist (ABC)
19.70 units on a scale
STANDARD_DEVIATION 15.34 • n=93 Participants
30.77 units on a scale
STANDARD_DEVIATION 25.54 • n=4 Participants
21.26 units on a scale
STANDARD_DEVIATION 19.50 • n=27 Participants

PRIMARY outcome

Timeframe: Week 12

Population: 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.

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.

Outcome measures

Outcome measures
Measure
Donepezil
n=19 Participants
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
n=18 Participants
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)
Contingency Naming Test (CNT) Performance Score
CNT Performance Score Rule 2
37.05 correct responses per minute
Standard Deviation 14.60
40.50 correct responses per minute
Standard Deviation 31.11
Contingency Naming Test (CNT) Performance Score
CNT Performance Score Rule 3
66.94 correct responses per minute
Standard Deviation 46.92
61.56 correct responses per minute
Standard Deviation 23.06

SECONDARY outcome

Timeframe: Week 12

Population: 41 of 42 randomized participants completed the 12-week randomized controlled trial. 39 of 42 participants were administered the ABC at week 12.

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.

Outcome measures

Outcome measures
Measure
Donepezil
n=18 Participants
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
n=21 Participants
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)
Aberrant Behavior Checklist (ABC)
17.56 units on a scale
Standard Deviation 13.65
24.43 units on a scale
Standard Deviation 23.27

Adverse Events

Donepezil

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Sugar Pill

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Allan Reiss, MD

Stanford University

Phone: 650-498-4538

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place