Trial Outcomes & Findings for N-acetylcysteine (NAC) for Children With Tourette Syndrome (NCT NCT01172288)

Last Updated: 2017-02-10

Results Overview

Yale Global Tic Severity Scale is a standard psychiatric measure that rates tics from 0 (no tics) to 100 (most severe tics). It separately rates motor tics and vocal tics in 5 subscales (number, frequency, intensity, complexity and interference) where the maximum severity score for motor tics is 25 and for vocal tics is 25. Giving us the Total Tic Severity Score maximum of 50. The additional Impairment Scale rates the degree of disability caused by the tics ranging from 0 (none) to 50 (severe). When these two scores are added we get the Yale Global Tic Severity Scale Score.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

31 participants

Primary outcome timeframe

12 weeks

Results posted on

2017-02-10

Participant Flow

Participant milestones

Participant milestones
Measure	N-Acetylcysteine N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Overall Study STARTED	17	14
Overall Study COMPLETED	16	13
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	N-Acetylcysteine N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Overall Study Adverse Event	1	0
Overall Study Protocol Violation	0	1

Baseline Characteristics

N-acetylcysteine (NAC) for Children With Tourette Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	N-Acetylcysteine n=17 Participants N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo n=14 Participants Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.	Total n=31 Participants Total of all reporting groups
Age, Continuous	12.4 years STANDARD_DEVIATION 1.4 • n=113 Participants	11.5 years STANDARD_DEVIATION 2.8 • n=163 Participants	11.9 years STANDARD_DEVIATION 2.17 • n=160 Participants
Gender Female	5 Participants n=113 Participants	2 Participants n=163 Participants	7 Participants n=160 Participants
Gender Male	12 Participants n=113 Participants	12 Participants n=163 Participants	24 Participants n=160 Participants
Age of onset	6.8 years STANDARD_DEVIATION 1.4 • n=113 Participants	6.1 years STANDARD_DEVIATION 2.4 • n=163 Participants	6.4 years STANDARD_DEVIATION 1.17 • n=160 Participants

PRIMARY outcome

Timeframe: 12 weeks

Outcome measures

Outcome measures
Measure	N-Acetylcysteine n=17 Participants N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo n=14 Participants Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Improvement in Tic Severity	24.3 units on a scale Standard Deviation 7.9	21.3 units on a scale Standard Deviation 4.6

SECONDARY outcome

Timeframe: 12 weeks

Premonitory Urge for Tics Scale (PUTS). Items are rated on a scale of 1-4 from "least" to "most." A total score is calculated by summing the scores of all items. Nine is the minimum possible score. A score of 12.5-24.5 indicates medium intensity of premonitory urges for tics. A score of 25-30.5 indicates high intensity which may be associated with marked impairment. Scores 31 and above indicate extremely high intensity with probable severe impairment. A score of 36 is the maximum score possible.

Outcome measures

Outcome measures
Measure	N-Acetylcysteine n=17 Participants N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo n=14 Participants Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Improvement of Premonitory Urges	24.7 units on a scale Standard Deviation 4.9	24.1 units on a scale Standard Deviation 4.8

SECONDARY outcome

Timeframe: 12 weeks

Childrens' Yale-Brown Obsessive-Compulsive Scale (CY-BOCS). 10-item scale. Each item is rated from 0-4. A sum total is calculated by adding items 1-10. 0-7: Subclinical. 8-15: Mild. 16-23: Moderate. 24-31: Severe. 32-40: Extreme.

Outcome measures

Outcome measures
Measure	N-Acetylcysteine n=17 Participants N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo n=14 Participants Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Improvement in OCD Severity	4.4 units on a scale Standard Deviation 8.0	11.5 units on a scale Standard Deviation 9.8

SECONDARY outcome

Timeframe: 12 weeks

Clinical Global Impression - Improvement Scale (CGI-I). The CGI is a 7-point scare that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 = Minimally worse; 6 = Much worse; 7 = Very much worse.

Outcome measures

Outcome measures
Measure	N-Acetylcysteine n=17 Participants N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo n=14 Participants Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Overall Improvement	3.3 units on a scale Standard Deviation 1.1	3.4 units on a scale Standard Deviation 1.1

SECONDARY outcome

Timeframe: 12 weeks

Number of participants with adverse events according to the Pediatric Adverse Events Rating Scale

Outcome measures

Outcome measures
Measure	N-Acetylcysteine n=17 Participants N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo n=14 Participants Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Number of Participants With Adverse Effects Irritability	0 participants	0 participants
Number of Participants With Adverse Effects Sadness or depressed mood	0 participants	1 participants
Number of Participants With Adverse Effects Fatigue	0 participants	0 participants
Number of Participants With Adverse Effects Insomnia	0 participants	0 participants
Number of Participants With Adverse Effects Decreased appetite	0 participants	0 participants
Number of Participants With Adverse Effects Chest pain	0 participants	0 participants
Number of Participants With Adverse Effects Syncope	0 participants	0 participants
Number of Participants With Adverse Effects Stomach ache	0 participants	2 participants
Number of Participants With Adverse Effects Nausea	0 participants	3 participants
Number of Participants With Adverse Effects Vomiting	0 participants	0 participants
Number of Participants With Adverse Effects Headache	1 participants	1 participants

Adverse Events

N-Acetylcysteine

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	N-Acetylcysteine n=17 participants at risk N-Acetylcysteine (NAC): 1 600mg capsule twice a day for 2 weeks and then 2 600mg capsules twice a day for the remaining 10 weeks of the trial.	Placebo n=14 participants at risk Placebo: 1 600mg Capsule twice a day for two weeks then 2 600mg capsules twice a day for the remaining 10 weeks of the study. Children receiving placebo will be offered the active intervention after the double-blind portion of the trial.
Psychiatric disorders Worsening of symptoms	5.9% 1/17 • Number of events 1 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.	0.00% 0/14 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.
Psychiatric disorders Sadness or depressive mood	0.00% 0/17 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.	7.1% 1/14 • Number of events 1 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.
Gastrointestinal disorders Stomach Ache	0.00% 0/17 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.	14.3% 2/14 • Number of events 2 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.
Gastrointestinal disorders Nausea	0.00% 0/17 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.	21.4% 3/14 • Number of events 3 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.
General disorders Headache	5.9% 1/17 • Number of events 1 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.	7.1% 1/14 • Number of events 1 • Every 4 weeks until the completion of the 12-week study Adverse events were systematically assessed with every assessment using the PAERS, unless they were brought to our attention by the subject between assessment periods.

Additional Information

Associate Professor

Yale University- School of Medicine

Phone: (203)737-4809

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place