Trial Outcomes & Findings for Memantine Treatment in Fragile X-Associated Tremor/Ataxia Syndrome (NCT NCT00584948)

Last Updated: 2017-05-30

Results Overview

The BDS-II is a 9-item, 27-point instrument that measures executive function as the capacity for behavioral and attentional self-regulation. Total score is a sum of the 9 items, with a range of 0-27, in which a higher score indicates a better performance.

Recruitment status

COMPLETED

Study phase

Target enrollment

94 participants

Primary outcome timeframe

One Year

Results posted on

2017-05-30

Participant Flow

Participant milestones

Participant milestones
Measure	Memantine Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study.	Placebo Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study
Overall Study STARTED	47	47
Overall Study COMPLETED	34	36
Overall Study NOT COMPLETED	13	11

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Memantine Treatment in Fragile X-Associated Tremor/Ataxia Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Memantine n=47 Participants Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study.	Placebo n=47 Participants Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study	Total n=94 Participants Total of all reporting groups
Age, Continuous	64.7 years STANDARD_DEVIATION 9.7 • n=99 Participants	66.3 years STANDARD_DEVIATION 7.0 • n=107 Participants	65.5 years STANDARD_DEVIATION 8.4 • n=206 Participants
Sex: Female, Male Female	15 Participants n=99 Participants	19 Participants n=107 Participants	34 Participants n=206 Participants
Sex: Female, Male Male	32 Participants n=99 Participants	28 Participants n=107 Participants	60 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) White	47 Participants n=99 Participants	47 Participants n=107 Participants	94 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=99 Participants	4 Participants n=107 Participants	5 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	46 Participants n=99 Participants	43 Participants n=107 Participants	89 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Region of Enrollment United States	47 participants n=99 Participants	47 participants n=107 Participants	94 participants n=206 Participants

PRIMARY outcome

Timeframe: One Year

Outcome measures

Outcome measures
Measure	Memantine n=34 Participants Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study.	Placebo n=36 Participants Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study
Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) Baseline	17.44 units on a scale Standard Deviation 5.19	16.12 units on a scale Standard Deviation 5.43
Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) One Year	15.66 units on a scale Standard Deviation 4.11	15.72 units on a scale Standard Deviation 3.93

PRIMARY outcome

Timeframe: 1 year

The CATSYS is a set of computer assisted diagnostic instruments that can measure intention tremor, postural tremor, postural sway, manual coordination and reaction time. The tremor intensity is defined as the root mean square of accelerations, recorded in the 0.9 Hz to 15.0 Hz band during the test period. Unit is measured in m/s2

Outcome measures

Outcome measures
Measure	Memantine n=34 Participants Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study.	Placebo n=36 Participants Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study
Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale Baseline	1.31 m/s^2 Standard Deviation 1.02	1.05 m/s^2 Standard Deviation 0.73
Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale One Year	1.77 m/s^2 Standard Deviation 1.78	1.89 m/s^2 Standard Deviation 2.19

Adverse Events

Memantine

Serious events: 2 serious events

Other events: 6 other events

Deaths: 0 deaths

Placebo

Serious events: 3 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Memantine n=47 participants at risk Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study.	Placebo n=47 participants at risk Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study
Musculoskeletal and connective tissue disorders Motor Vehicle Accident	2.1% 1/47 • Number of events 1	0.00% 0/47
Respiratory, thoracic and mediastinal disorders Lung Cancer	2.1% 1/47 • Number of events 1	0.00% 0/47
Reproductive system and breast disorders Ovarian Cancer	0.00% 0/47	2.1% 1/47 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pneumonia	0.00% 0/47	2.1% 1/47 • Number of events 1
Gastrointestinal disorders Appendicitis	0.00% 0/47	2.1% 1/47 • Number of events 1

Other adverse events

Other adverse events
Measure	Memantine n=47 participants at risk Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study.	Placebo n=47 participants at risk Week 1: Take 5mg tab every morning. Week 2: Take 5mg tab every morning and evening. Week 3: Take 10mg tab in the morning and 5 mg in the evening. Week 4: Take 10 mg tab in the morning and evening, and remain on this dose through the remainder of the study
Gastrointestinal disorders Constipation	12.8% 6/47 • Number of events 10	4.3% 2/47 • Number of events 4

Additional Information

Randi J Hagerman, MD

University of California, Davis Medical Center

Phone: 916-703-0247

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place