Trial Outcomes & Findings for Proof of Concept Study of an Oral Orthotic to Reduce Tic Severity in Chronic Tic Disorder and Tourette Syndrome (NCT NCT02067819)

NCT ID: NCT02067819

Last Updated: 2022-05-24

Results Overview

The primary clinical outcome measures included the Yale Global Tic Severity Scale (YGTSS). The YGTSS is a semi structured interview designed to elicit information regarding the character \& anatomical distribution of tics observed during the course of 1 week interval, prior to clinical assessment. Following completion of the interview \& construction of a "tic inventory", clinicians rate severity of motor and photic tics along with 5 separate dimensions (frequency, intensity, number, complexity, interference). The YGTSS is comprised of a list of motor and vocal tics \& the participant endorses which tics are currently present. Motor and vocal tics are then separately rated for Number, Frequency, Intensity, Complexity, Interference \& Impairment, each on a 6 point likert scale ranging from 0 (none) to 5 (Severe).This results in a range of 0-25 for motor tics \& 0-25 for vocal tics, for a total score range of 0-50. A score of 0 indicating a lack of tics \& 50 represent the most severe tics.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

21 participants

Primary outcome timeframe

Baseline Visit, Week 2 Visit

Results posted on

2022-05-24

Participant Flow

Participant milestones

Participant milestones
Measure
Active Oral Orthotic Treatment
Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study. Active Oral Orthotic Treatment: Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Placebo Oral Orthotic Treatment
Control participants will receive an identical sham splint to the active condition, but not adjusted to the recommended height for the given participant. Participants will be asked to wear the orthotic 24/7 for the duration of the study. Placebo Oral Orthotic Treatment: After two weeks, this treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Overall Study
STARTED
11
10
Overall Study
COMPLETED
6
7
Overall Study
NOT COMPLETED
5
3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Proof of Concept Study of an Oral Orthotic to Reduce Tic Severity in Chronic Tic Disorder and Tourette Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Active Oral Orthotic Treatment
n=11 Participants
Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study. Active Oral Orthotic Treatment: Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Placebo Oral Orthotic Treatment
n=10 Participants
Control participants will receive an identical sham splint to the active condition, but not adjusted to the recommended height for the given participant. Participants will be asked to wear the orthotic 24/7 for the duration of the study. Placebo Oral Orthotic Treatment: After two weeks, this treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Total
n=21 Participants
Total of all reporting groups
Age, Continuous
12.27 years
STANDARD_DEVIATION 5.293 • n=99 Participants
12.5 years
STANDARD_DEVIATION 4.90 • n=107 Participants
12.38 years
STANDARD_DEVIATION 5.1 • n=206 Participants
Sex: Female, Male
Female
4 Participants
n=99 Participants
4 Participants
n=107 Participants
8 Participants
n=206 Participants
Sex: Female, Male
Male
7 Participants
n=99 Participants
6 Participants
n=107 Participants
13 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=99 Participants
2 Participants
n=107 Participants
2 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
4 Participants
n=99 Participants
3 Participants
n=107 Participants
7 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
7 Participants
n=99 Participants
5 Participants
n=107 Participants
12 Participants
n=206 Participants

PRIMARY outcome

Timeframe: Baseline Visit, Week 2 Visit

Population: 4 opted not to participate after randomization but prior to receiving the orthotic. 1 participant dropped out after baseline visit due to belief that oral orthotic was responsible for disruptive behavior at school. 1 participant was removed from study at Baseline by PI due to medication change for bipolar d/o. 1 participant dropped out after Week 2 due to discomfort when wearing the oral orthotic.1 patient failed to respond to attempted contact from the study team following baseline visit

The primary clinical outcome measures included the Yale Global Tic Severity Scale (YGTSS). The YGTSS is a semi structured interview designed to elicit information regarding the character \& anatomical distribution of tics observed during the course of 1 week interval, prior to clinical assessment. Following completion of the interview \& construction of a "tic inventory", clinicians rate severity of motor and photic tics along with 5 separate dimensions (frequency, intensity, number, complexity, interference). The YGTSS is comprised of a list of motor and vocal tics \& the participant endorses which tics are currently present. Motor and vocal tics are then separately rated for Number, Frequency, Intensity, Complexity, Interference \& Impairment, each on a 6 point likert scale ranging from 0 (none) to 5 (Severe).This results in a range of 0-25 for motor tics \& 0-25 for vocal tics, for a total score range of 0-50. A score of 0 indicating a lack of tics \& 50 represent the most severe tics.

Outcome measures

Outcome measures
Measure
Active Oral Orthotic Treatment
n=6 Participants
Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study. Active Oral Orthotic Treatment: Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Placebo Oral Orthotic Treatment
n=7 Participants
Control participants will receive an identical sham splint to the active condition, but not adjusted to the recommended height for the given participant. Participants will be asked to wear the orthotic 24/7 for the duration of the study. Placebo Oral Orthotic Treatment: After two weeks, this treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Change in Global Severity of Tics and Clinical Condition as Measured by YGTSS
20.19 score on a scale
Standard Error 1.06
25.82 score on a scale
Standard Error 0.99

SECONDARY outcome

Timeframe: Baseline Visit, Week 2 Visit

Population: 4 opted not to participate after randomization but prior to receiving the orthotic. 1 participant dropped out after baseline visit due to belief that oral orthotic was responsible for disruptive behavior at school. 1 participant was removed from study at Baseline by PI due to medication change for bipolar d/o. 1 participant dropped out after Week 2 due to discomfort when wearing the oral orthotic.1 patient failed to respond to attempted contact from the study team following baseline visit

To assess global severity of tics and change in the clinical condition over time, the Clinical Global Impressions Scale - Severity (CGI-S) was utilized. The Severity of Illness has a low score of 0 and high score is 7. The low score of 0 represents that the subject was not assessed and the high score of 7 represents among the most extremely ill subjects.

Outcome measures

Outcome measures
Measure
Active Oral Orthotic Treatment
n=6 Participants
Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study. Active Oral Orthotic Treatment: Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Placebo Oral Orthotic Treatment
n=7 Participants
Control participants will receive an identical sham splint to the active condition, but not adjusted to the recommended height for the given participant. Participants will be asked to wear the orthotic 24/7 for the duration of the study. Placebo Oral Orthotic Treatment: After two weeks, this treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Change in Number, Frequency, and Intensity of Motor and Vocal Tics as Measured by the Clinical Global Impressions Scale - Severity
4.57 score on a scale
Standard Error 0.25
5.27 score on a scale
Standard Error 0.23

Adverse Events

Active Oral Orthotic Treatment

Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths

Placebo Oral Orthotic Treatment

Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Active Oral Orthotic Treatment
n=8 participants at risk
Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study. Active Oral Orthotic Treatment: Active treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Placebo Oral Orthotic Treatment
n=9 participants at risk
Control participants will receive an identical sham splint to the active condition, but not adjusted to the recommended height for the given participant. Participants will be asked to wear the orthotic 24/7 for the duration of the study. Placebo Oral Orthotic Treatment: After two weeks, this treatment group will receive an occlusal splint adjusted to the appropriate therapeutic height (based on an initial fitting). Participants will be instructed to wear the orthotic 24/7 (or as close as possible) for the duration of the study.
Social circumstances
mouth soreness
75.0%
6/8 • Number of events 6 • Ten Weeks
Participants reported all side effects or adverse events in a diary at home, as well as on a structured adverse events questionnaire during study visits. Study personnel additionally inquired about adverse events at each visit. The total number of participants at risk for an adverse event was 17, because 17 participants received the oral orthotic (8 were active, 9 were placebo).
66.7%
6/9 • Number of events 6 • Ten Weeks
Participants reported all side effects or adverse events in a diary at home, as well as on a structured adverse events questionnaire during study visits. Study personnel additionally inquired about adverse events at each visit. The total number of participants at risk for an adverse event was 17, because 17 participants received the oral orthotic (8 were active, 9 were placebo).

Additional Information

Shannon Bennett, PhD

Weill Cornell Medicine

Phone: 646-962-2820

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place