NCT02197104

Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome

Phase 2 Study of Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Rush University Medical Center · Academic / Other
• Sex: All
• Age: 18 Years – 90 Years
• Healthy volunteers: Not accepted

Summary

The objective of this study is to determine if citocoline is effective for balance abnormalities and to stabilize cognitive decline in patients with fragile X-associated tremor ataxia syndrome. The study will test 1000mg twice daily of citocoline for 12 months in an open label pilot study, with study visits at baseline, 3, 6, and 12 months.

Detailed description

The purpose of this study was to determine if citicoline was safe for the treatment of tremor and balance abnormalities and to stabilize cognitive decline in patients with fragile X-associated tremor ataxia syndrome. Ten participants with diagnosed FXTAS were administered 1000 mg of citicoline once daily for 12 months. Outcome measures and neurological examination were performed at baseline, 3 months, 6 months, and 12 months. The primary outcome was safety. Secondary outcomes included change in FXTAS Rating Scale score, a battery of neuropsychological tests, an instrumented Timed up and go test, computerized dynamic posturography, 9-hole pegboard test, and balance confidence and psychiatric symptom questionnaires.

Conditions

• Fragile X Tremor/Ataxia Syndrome

Interventions

Type	Name	Description
DRUG	citocoline	Ten subjects will receive 1,000mg once daily of citicoline. Each subject will stay on the study drug for 12 months. The outcome measures will be assessed at baseline and then again at the end of month 3, month 6, and month 12. The subjects will continue taking the same study drug at the same dose throughout the entire trial.

Timeline

• Start date: 2015-01-01
• Primary completion: 2017-01-01
• Completion: 2017-01-01
• First posted: 2014-07-22
• Last updated: 2022-11-02
• Results posted: 2022-11-02

Locations

1 site across 1 country: United States