Trial Outcomes & Findings for A Randomized, Placebo-controlled, Tourette Syndrome Study. (NCT NCT00206323)

Results Overview

A component of the Yale Global Tic Severity Scale (YGTSS), the change from baseline in Total Tic Score (TTS) at visit 5 (day 70) is the pre-defined primary endpoint. The Total Tic Score (TTS) is a summation of the Total Motor Tic and Total Phonic Tic Scores. The Overall Impairment Rating is rated on a 50-point scale anchored by 0 (No impairment) and 50 (Severe impairment).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

29 participants

Primary outcome timeframe

baseline and Day 70

Results posted on

2020-02-12

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo/Sugar Pill Placebo or sugar pill placebo/sugar pill: placebo	Topiramate Topiramate versus placebo Topiramate (drug): Topiramate 25 mg titrated to 200 mg
Overall Study STARTED	14	15
Overall Study COMPLETED	8	12
Overall Study NOT COMPLETED	6	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo/Sugar Pill Placebo or sugar pill placebo/sugar pill: placebo	Topiramate Topiramate versus placebo Topiramate (drug): Topiramate 25 mg titrated to 200 mg
Overall Study Lack of Efficacy	4	1
Overall Study Adverse Event	1	1
Overall Study Lost to Follow-up	1	1

Baseline Characteristics

A Randomized, Placebo-controlled, Tourette Syndrome Study.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo/Sugar Pill n=14 Participants Placebo or sugar pill placebo/sugar pill: placebo	Topiramate n=15 Participants Topiramate versus placebo Topiramate (drug): Topiramate 25 mg titrated to 200 mg	Total n=29 Participants Total of all reporting groups
Age, Categorical <=18 years	14 Participants n=5 Participants	15 Participants n=7 Participants	29 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	14.1 years STANDARD_DEVIATION 8.35 • n=5 Participants	18.8 years STANDARD_DEVIATION 10.93 • n=7 Participants	16.5 years STANDARD_DEVIATION 9.89 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	14 Participants n=7 Participants	26 Participants n=5 Participants

PRIMARY outcome

Timeframe: baseline and Day 70

A component of the Yale Global Tic Severity Scale (YGTSS), the change from baseline in Total Tic Score (TTS) at visit 5 (day 70) is the pre-defined primary endpoint. The Total Tic Score (TTS) is a summation of the Total Motor Tic and Total Phonic Tic Scores. The Overall Impairment Rating is rated on a 50-point scale anchored by 0 (No impairment) and 50 (Severe impairment).

Outcome measures

Outcome measures
Measure	Placebo/Sugar Pill n=13 Participants Placebo or sugar pill placebo/sugar pill: placebo	Topiramate n=14 Participants Topiramate versus placebo Topiramate (drug): Topiramate 25 mg titrated to 200 mg
Change From Baseline in Total Tic Score at Day 70	-5.0 units on a scale Standard Deviation 9.88	-14.29 units on a scale Standard Deviation 10.47

Adverse Events

Placebo/Sugar Pill

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Topiramate

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Placebo/Sugar Pill n=14 participants at risk Placebo or sugar pill placebo/sugar pill: placebo	Topiramate n=15 participants at risk Topiramate versus placebo Topiramate (drug): Topiramate 25 mg titrated to 200 mg
General disorders headache	21.4% 3/14 • Number of events 3	20.0% 3/15 • Number of events 3
Gastrointestinal disorders diarrhea	7.1% 1/14 • Number of events 1	20.0% 3/15 • Number of events 3
Gastrointestinal disorders abdominal pain	14.3% 2/14 • Number of events 2	13.3% 2/15 • Number of events 2
General disorders drowsiness	14.3% 2/14 • Number of events 2	13.3% 2/15 • Number of events 2
General disorders cognitive slowing	0.00% 0/14	6.7% 1/15 • Number of events 1
Renal and urinary disorders kidney stone	0.00% 0/14	6.7% 1/15 • Number of events 1

Additional Information

Dr. Joseph Jankovic

Baylor College of Medicine

Phone: 713-798-6556

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place