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

NCT ID: NCT02197104

Last Updated: 2022-11-02

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-01-31
• Study Completion Date: 2017-01-31

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.

Conditions

Fragile X Tremor/Ataxia Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Citocoline

The intervention will be 1000mg of citicoline in capsule form once daily.

Group Type: EXPERIMENTAL

citocoline

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

CDP-choline

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 90 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Rush University Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Deborah Hall, MD

Associate Professor Neurological Sciences

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Deborah A Hall, MD PhD

Role: PRINCIPAL_INVESTIGATOR

Rush University Medical Center

Locations

Rush University Medical Center

Chicago, Illinois, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 32053612 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

CITO-123

Identifier Type: -

Identifier Source: org_study_id