Trial Outcomes & Findings for A Study of N-Acetyl Cysteine in Children With Autism (NCT NCT00627705)
NCT ID: NCT00627705
Last Updated: 2017-05-18
Results Overview
The Dosage Record and Treatment Emergent Symptom Scale (DOTES) provides information on the presence, frequency, and severity of side effects reported during the course of the trial.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
43 participants
Primary outcome timeframe
4, 8, and 12 weeks
Results posted on
2017-05-18
Participant Flow
Recruitment started in March 2009 and ended in September 2010. This study was conducted in the Autism \& Developmental Disabilities Clinic in the Division of Child \& Adolescent Psychiatry, Lucile Packard Children's Hospital at Stanford University.
Fifty-one potential subjects inquired about the study. Forty-three of the subjects signed a consent form. Seven subjects were excluded because they did not meet criteria for autistic disorder. Three subjects decided not to participate in the study before baseline measures were obtained. Thirty-three subjects were randomized in the study.
Participant milestones
| Measure |
N-Acetyl Cysteine
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
18
|
|
Overall Study
COMPLETED
|
13
|
12
|
|
Overall Study
NOT COMPLETED
|
2
|
6
|
Reasons for withdrawal
| Measure |
N-Acetyl Cysteine
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
|---|---|---|
|
Overall Study
Unwilling to take the compound (taste)
|
1
|
3
|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
2
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
Baseline Characteristics
A Study of N-Acetyl Cysteine in Children With Autism
Baseline characteristics by cohort
| Measure |
N-Acetyl Cysteine
n=15 Participants
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
n=18 Participants
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Total
n=33 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
15 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
33 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
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
31 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
18 participants
n=7 Participants
|
33 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 4, 8, and 12 weeksPopulation: We analyzed subjects who had follow-up data available.
The Dosage Record and Treatment Emergent Symptom Scale (DOTES) provides information on the presence, frequency, and severity of side effects reported during the course of the trial.
Outcome measures
| Measure |
N-Acetyl Cysteine
n=14 Participants
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
n=15 Participants
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
|---|---|---|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Total Number of Subjects with GI adverse Events
|
11 participants
|
7 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Constipation
|
3 participants
|
2 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Nausea/Vomiting
|
6 participants
|
3 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Diarrhea
|
3 participants
|
1 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Increased Appetite
|
2 participants
|
0 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Decreased Appetite
|
2 participants
|
3 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Akathisia
|
1 participants
|
0 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Excitement/Agitation
|
2 participants
|
3 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Increased Motor Activity
|
2 participants
|
3 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Tremor
|
0 participants
|
1 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Syncope/Dizziness
|
0 participants
|
1 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Depressive Affect
|
1 participants
|
0 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Nasal Congestion
|
4 participants
|
6 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Increased Salivation
|
0 participants
|
2 participants
|
|
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Sweating
|
0 participants
|
1 participants
|
PRIMARY outcome
Timeframe: 12 weeksPopulation: We analyzed subjects who had follow-up data available.
Score range 1-7 (lower score mean more improvement compared to baseline)
Outcome measures
| Measure |
N-Acetyl Cysteine
n=14 Participants
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
n=15 Participants
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
|---|---|---|
|
The Clinical Global Rating Scale (CGRS) Improvement Subscale Score
|
3.2 score (range 1-7)
Standard Deviation 0.9
|
2.9 score (range 1-7)
Standard Deviation 1.1
|
PRIMARY outcome
Timeframe: 12 weeksPopulation: Data not collected.
Data not collected. The laboratory was not able to measure Glutathione levels.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: baseline and 12 weeksPopulation: We analyzed subjects who had follow-up data available.
Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability
Outcome measures
| Measure |
N-Acetyl Cysteine
n=14 Participants
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
n=15 Participants
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
|---|---|---|
|
Irritability Subscale of the Aberrant Behavior Checklist (ABC)
Baseline ABC-I Score
|
16.9 Score (range 0-45)
Standard Deviation 7.9
|
14.8 Score (range 0-45)
Standard Deviation 9.6
|
|
Irritability Subscale of the Aberrant Behavior Checklist (ABC)
Week 12 ABC-I Score
|
7.2 Score (range 0-45)
Standard Deviation 5.7
|
13.1 Score (range 0-45)
Standard Deviation 9.9
|
SECONDARY outcome
Timeframe: 4, 8, and 12 weeksPopulation: Measure not analyzed.
Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeksPopulation: We analyzed subjects who had follow-up data available.
SRS total score (range 0-195); higher scores mean more social impairment
Outcome measures
| Measure |
N-Acetyl Cysteine
n=14 Participants
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
n=15 Participants
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
|---|---|---|
|
Social Responsiveness Scale (SRS)
Baseline SRS Total Score
|
111.9 SRS total score (range 0-195)
Standard Deviation 28.3
|
104.7 SRS total score (range 0-195)
Standard Deviation 28.1
|
|
Social Responsiveness Scale (SRS)
Week 12 SRS Total Score
|
93.8 SRS total score (range 0-195)
Standard Deviation 26.7
|
98.5 SRS total score (range 0-195)
Standard Deviation 37.8
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Data not collected. Measure not analyzed.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 12 weeksPopulation: Data not collected. The measure was not analyzed. The lab was not able to measure Glutathione for the study.
Outcome measures
Outcome data not reported
Adverse Events
N-Acetyl Cysteine
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Sugar Pill
Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
N-Acetyl Cysteine
n=14 participants at risk
active compound N-Acetyl Cysteine
N-Acetyl Cysteine: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
Sugar Pill
n=15 participants at risk
Placebo or sugar pill
Placebo - sugar pill: Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks
Entire intervention lasts for 12 weeks (drug administration is continuous).
|
|---|---|---|
|
Gastrointestinal disorders
Constipation
|
21.4%
3/14 • Number of events 3 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
13.3%
2/15 • Number of events 2 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
Gastrointestinal disorders
Nausea/Vomiting
|
42.9%
6/14 • Number of events 6 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
20.0%
3/15 • Number of events 3 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
Gastrointestinal disorders
Diarrhea
|
21.4%
3/14 • Number of events 3 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
6.7%
1/15 • Number of events 1 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
Gastrointestinal disorders
Increased Appetite
|
14.3%
2/14 • Number of events 2 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
0.00%
0/15 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
Gastrointestinal disorders
Decreased Appetite
|
14.3%
2/14 • Number of events 2 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
20.0%
3/15 • Number of events 3 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Akathisia
|
7.1%
1/14 • Number of events 1 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
0.00%
0/15 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Excitement/ Agitation
|
14.3%
2/14 • Number of events 2 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
20.0%
3/15 • Number of events 3 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Increased Motor Activity
|
14.3%
2/14 • Number of events 2 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
20.0%
3/15 • Number of events 3 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Tremor
|
0.00%
0/14 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
6.7%
1/15 • Number of events 1 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Syncope/Dizziness
|
0.00%
0/14 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
6.7%
1/15 • Number of events 1 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
Psychiatric disorders
Depressive Affect
|
7.1%
1/14 • Number of events 1 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
0.00%
0/15 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Nasal Congestion
|
28.6%
4/14 • Number of events 4 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
40.0%
6/15 • Number of events 6 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Increased Salivation
|
0.00%
0/14 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
13.3%
2/15 • Number of events 2 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
|
General disorders
Sweating
|
0.00%
0/14 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
6.7%
1/15 • Number of events 1 • Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
|
Additional Information
Antonio Hardan, MD
Stanford University School of Medicine
Phone: 650-736-1235
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place