Trial Outcomes & Findings for Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome (NCT NCT02197104)

NCT ID: NCT02197104

Last Updated: 2022-11-02

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

10 participants

Primary outcome timeframe

12 months

Results posted on

2022-11-02

Participant Flow

Participant milestones

Participant milestones
Measure	Citocoline All participants received 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.
Overall Study STARTED	10
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Citocoline n=10 Participants All participants were white and non-Hispanic; there were nine men and one woman. This distribution is expected as there is a lower penetrance of FXTAS in women from X-inactivation. The subject with probable FXTAS was also the female subject. Nine participants completed the one-year follow-up and one participant with a low platelet count withdrew prior to the end of the study and outcome data was missing for this participant at 12 months, but all available data were used for analysis.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants
Age, Continuous	70 years STANDARD_DEVIATION 7.33 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States · Midwestern	9 Participants n=5 Participants
Region of Enrollment United States · East Coast	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Population: 9 patients with FXTAS who participated in the study for 12 months were analyzed.

Outcome measures

Outcome measures
Measure	Citocoline n=9 Participants Experimental group given the study drug, citocoline
FXTAS Rating Scale Score	45.8 score on a scale Standard Deviation 21.22

Adverse Events

Citocoline

Serious events: 6 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Citocoline n=10 participants at risk All participants received citocoline.
Gastrointestinal disorders Gastrointestinal disorders	20.0% 2/10 • Number of events 2 • 12 months Adverse event information was collected at baseline and then again at the end of month 3, month 6, and month 12.
Nervous system disorders Falls	30.0% 3/10 • Number of events 3 • 12 months Adverse event information was collected at baseline and then again at the end of month 3, month 6, and month 12.
Immune system disorders Low platelet count	10.0% 1/10 • Number of events 1 • 12 months Adverse event information was collected at baseline and then again at the end of month 3, month 6, and month 12.

Other adverse events

Other adverse events
Measure	Citocoline n=10 participants at risk All participants received citocoline.
Blood and lymphatic system disorders Bruising	10.0% 1/10 • Number of events 1 • 12 months Adverse event information was collected at baseline and then again at the end of month 3, month 6, and month 12.
Nervous system disorders Dizziness	20.0% 2/10 • Number of events 2 • 12 months Adverse event information was collected at baseline and then again at the end of month 3, month 6, and month 12.
Blood and lymphatic system disorders Leg edema	10.0% 1/10 • Number of events 1 • 12 months Adverse event information was collected at baseline and then again at the end of month 3, month 6, and month 12.
General disorders Weight gain	10.0% 1/10 • Number of events 1 • 12 months Adverse event information was collected at baseline and then again at the end of month 3, month 6, and month 12.

Additional Information

Dr. Deborah A Hall

Rush University Medical Center

Phone: 312-563-2900

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place