Phase 2
N=10
Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
Fragile X Tremor/Ataxia Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02197104 ↗Enrolled (actual)
10
Serious AEs
60.0%
Results posted
Nov 2022
Primary outcome: Primary: FXTAS Rating Scale Score — 45.8 score on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- citocoline (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Rush University Medical Center
- Primary completion
- Jan 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY FXTAS Rating Scale Score |
45.8 | — |
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.
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT02197104). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.