Clinical trial NCT02197104: Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
Official title: Phase 2 Study of Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Tremor/Ataxia Syndrome
Lead sponsor: Rush University Medical Center
Sponsor class: OTHER
First posted: 2014-07-22
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

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.

Primary outcomes:
measure: FXTAS Rating Scale Score
timeFrame: 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
Study officials:
affiliation: Rush University Medical Center
name: Deborah A Hall, MD PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Hall DA, Robertson EE, Leehey M, McAsey A, Ouyang B, Berry-Kravis E, O'Keefe JA. Open-label pilot clinical trial of citicoline for fragile X-associated tremor/ataxia syndrome (FXTAS). PLoS One. 2020 Feb 13;15(2):e0225191. doi: 10.1371/journal.pone.0225191. eCollection 2020.