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Memantine for fragile X-associated tremor/ataxia syndrome: a randomized, double-blind, placebo-controlled trial
Andreea L Seritan1, Danh V Nguyen, Yi Mu
12230 Stockton Blvd, Sacramento, CA 95817 andreea.seritan@ucdmc.ucdavis.edu.
Objective:
Memantine, an uncompetitive N-methyl-d-aspartate receptor antagonist, is currently approved by the US Food and Drug Administration for the treatment of moderate to severe Alzheimer's disease. Anecdotal reports have suggested that memantine may improve neurologic and cognitive symptoms of individuals with the neurodegenerative disease fragile X-associated tremor/ataxia syndrome (FXTAS); however, its efficacy and safety in this population have not been assessed in a controlled trial.
Method:
Individuals with FXTAS aged 34-80 years were enrolled in a randomized, double-blind, placebo-controlled, 1-year trial between September 2007 and August 2012. Inclusion required definite, probable, or possible FXTAS in clinical stages 1-5 according to previously published criteria. Primary outcome measures were the Behavioral Dyscontrol Scale (BDS) score and CATSYS intention tremor severity.
Results:
Ninety-four participants were randomized from 205 screened; of those, 43 and 45 started treatment with memantine (titrated to 10 mg twice daily) and placebo, respectively. Thirty-four participants receiving memantine and 36 receiving placebo completed the 1-year endpoint assessment (n = 70). Intention-to-treat analysis showed no improvement with respect to intention tremor severity (mean [SD] values with memantine vs placebo: 1.05 [0.73] vs 1.89 [2.19], P = .047) or BDS score (16.12 [5.43] vs 15.72 [3.93], P = .727) at follow-up. Post hoc analyses of participants with early FXTAS (stage ≤ 3), those with late FXTAS (stage > 3), and those in different age groups (≤ 65 years and > 65 years) also indicated no significant improvement. More frequent mild adverse events were observed in the placebo group, while more frequent moderate adverse events occurred in the memantine group (P = .007).
Conclusion:
This randomized, double-blind, placebo-controlled trial of memantine for individuals with FXTAS showed no benefit compared to placebo with respect to the selected outcome measures.
Trial Registration:
ClinicalTrials.gov identifier: NCT00584948.
Insights
Memantine did not improve neurologic or cognitive symptoms in fragile X-associated tremor/ataxia syndrome (FXTAS). This randomized trial found no benefit compared to placebo for FXTAS patients.
Area of Science:
- Neuroscience
- Pharmacology
Background:
- Fragile X-associated tremor/ataxia syndrome (FXTAS) is a neurodegenerative disorder.
- Memantine is an N-methyl-d-aspartate receptor antagonist approved for Alzheimer's disease.
- Anecdotal evidence suggested potential benefits of memantine for FXTAS symptoms.
Purpose of the Study:
- To evaluate the efficacy and safety of memantine in individuals with FXTAS.
- To assess memantine's impact on neurologic and cognitive symptoms in FXTAS patients through a controlled trial.
Main Methods:
- A randomized, double-blind, placebo-controlled trial involving 94 participants with FXTAS (aged 34-80).
- Participants received either memantine (titrated to 10 mg twice daily) or placebo for 1 year.
- Primary outcomes included the Behavioral Dyscontrol Scale (BDS) score and CATSYS intention tremor severity.
Main Results:
- No significant improvement in intention tremor severity (P = .047) or BDS score (P = .727) was observed in the memantine group compared to placebo.
- Post hoc analyses across different FXTAS stages and age groups also showed no significant benefits.
- Moderate adverse events were more frequent in the memantine group (P = .007).
Conclusions:
- Memantine demonstrated no benefit over placebo for the primary outcome measures in individuals with FXTAS.
- The trial did not support the use of memantine for treating FXTAS symptoms.
- Further research may be needed to explore other therapeutic avenues for FXTAS.
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