Fragile X-Associated Tremor/Ataxia Syndrome, Fragile X Premutation Carriers
Conditions
Keywords
FXTAS, Fragile X Premutation
Brief summary
The purpose of this study is to determine if memantine is effective in treating symptoms of Fragile X-associated Tremor Ataxia Syndrome.
Interventions
DRUGMemantine
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.
DRUGPlacebo
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.
Sponsors
National Institute on Aging (NIA)
Forest Laboratories
University of California, Davis
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
30 Years to 80 Years
Healthy volunteers
Yes
Inclusion criteria
* Fragile X premutation carrier (CGG repeat 55-200)with neurological symptoms; Clinical FXTAS Stage 1-5
Exclusion criteria
* Previous reaction to memantine
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) | One Year | 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. |
| Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale | 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 |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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. | 47 |
| 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 | 47 |
| Total | 94 |
Baseline characteristics
| Characteristic | Total | Memantine | Placebo |
|---|---|---|---|
| Age, Continuous | 65.5 years STANDARD_DEVIATION 8.4 | 64.7 years STANDARD_DEVIATION 9.7 | 66.3 years STANDARD_DEVIATION 7 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 5 Participants | 1 Participants | 4 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 89 Participants | 46 Participants | 43 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 94 Participants | 47 Participants | 47 Participants |
| Region of Enrollment United States | 94 participants | 47 participants | 47 participants |
| Sex: Female, Male Female | 34 Participants | 15 Participants | 19 Participants |
| Sex: Female, Male Male | 60 Participants | 32 Participants | 28 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 6 / 47 | 2 / 47 |
| serious Total, serious adverse events | 2 / 47 | 3 / 47 |
Outcome results
Primary
Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II)
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.
Time frame: One Year
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Memantine | 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 |
| Memantine | 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 |
| Placebo | Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) | One Year | 15.72 units on a scale | Standard Deviation 3.93 |
| Placebo | Change From Baseline in Executive Functioning as Measured by the Behavioral Dyscontrol Scale II (BDS-II) | Baseline | 16.12 units on a scale | Standard Deviation 5.43 |
Primary
Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale
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
Time frame: 1 year
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Memantine | Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale | Baseline | 1.31 m/s^2 | Standard Deviation 1.02 |
| Memantine | Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale | One Year | 1.77 m/s^2 | Standard Deviation 1.78 |
| Placebo | Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale | Baseline | 1.05 m/s^2 | Standard Deviation 0.73 |
| Placebo | Change From Baseline in Intention Tremor as Measured by the CATSYS Tremor Scale | One Year | 1.89 m/s^2 | Standard Deviation 2.19 |