Citocoline for Treatment of FXTAS

• Status: Completed
• Trial end date: 2017-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Rush University Medical Center

Criteria

Inclusion Criteria:

• Clinical diagnosis of FXTAS

• Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG are within normal limits

Exclusion Criteria:

• Legal incapacity or limited legal capacity

• Presence of severe renal disease (BUN 50% greater than normal or creatinine clearance <60 mL/min) or hepatic disease.

• Abnormal creatine kinase and/or platelet count in the past 6 months (as determined by lab reports obtained from primary care physicians or conducted at baseline).

• Women of childbearing potential who are pregnant at the time of screening or who will not use adequate protection during participation of the study.

• Allergy/sensitivity to the drug of its formulations.

• Concurrent participation in another clinical study.

• Active substance use or dependence.

• Serious illness (requiring systematic treatment/or hospitalization) until the subject either completes therapy or is clinically stable on therapy, in the opinion of the site investigator, for at least 60 days prior to study entry.

• Inability or unwillingness of the subject or legal guardian/representative to give written informed consent.