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

Primary Purpose

Fragile X Tremor/Ataxia Syndrome

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

citocoline

About this trial

This is an interventional treatment trial for Fragile X Tremor/Ataxia Syndrome

Eligibility Criteria

18 Years - 90 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Rush University Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Citocoline

Arm Description

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

Outcomes

Primary Outcome Measures

FXTAS Rating Scale Score

The FXTAS Rating Scale (FXTAS-RS), which was designed to measure the severity of motor signs in FXTAS patients, was administered to each participant. The FXTAS-RS was designed to measure the severity of motor signs of FXTAS, tremor, ataxia, and parkinsonism. The scale was developed from a combination of three separate measures: the Clinical Rating Scale for Tremor (CRST), the International Cooperative Ataxia Rating Scale (ICARS), and the Unified Parkinson's Disease Rating Scale (UPDRS). The resultant scale has 44 items and a possible total score of 0-226. A higher score indicates worse symptoms. For this study, improvement was defined as a 20% improvement on the FXTAS-RS.

Secondary Outcome Measures

Full Information

NCT ID

NCT02197104

First Posted

July 14, 2014

Last Updated

November 1, 2022

Sponsor

Rush University Medical Center

1. Study Identification

Unique Protocol Identification Number

NCT02197104

Brief Title

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

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Completed

Study Start Date

January 2015 (undefined)

Primary Completion Date

January 2017 (Actual)

Study Completion Date

January 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Rush University Medical Center

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Fragile X Tremor/Ataxia Syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Citocoline

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

citocoline

Other Intervention Name(s)

CDP-choline

Intervention Description

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.

Primary Outcome Measure Information:

Title

FXTAS Rating Scale Score

Description

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

90 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Deborah A Hall, MD PhD

Organizational Affiliation

Rush University Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Rush University Medical Center

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60612

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

32053612

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.

Results Reference

derived

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

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