Trial Outcomes & Findings for An Open-Label Study of N-Acetyl Cysteine in Children With Autism (NCT NCT00676195)

Results Overview

Number of Participants with adverse events reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) during the course of the study as measured at time points (4, 8, and 12 weeks).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

24 participants

Primary outcome timeframe

4, 8, and 12 weeks

Results posted on

2017-03-29

Participant Flow

Participant milestones

Participant milestones
Measure	N-Acetyl Cysteine N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows: Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day
Overall Study STARTED	24
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	N-Acetyl Cysteine N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows: Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day
Overall Study Lost to Follow-up	1
Overall Study Adverse Event	1
Overall Study Physician Decision	1

Baseline Characteristics

An Open-Label Study of N-Acetyl Cysteine in Children With Autism

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	N-Acetyl Cysteine n=24 Participants N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows: Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day
Age, Categorical <=18 years	24 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants
Region of Enrollment United States	24 participants n=5 Participants

PRIMARY outcome

Timeframe: 4, 8, and 12 weeks

Number of Participants with adverse events reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) during the course of the study as measured at time points (4, 8, and 12 weeks).

Outcome measures

Outcome measures
Measure	N-Acetyl Cysteine n=24 Participants N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows: Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day
Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES)	17 number of participants

SECONDARY outcome

Timeframe: 12 weeks

Population: No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 weeks

Population: No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 4, 8, and 12 weeks

Population: No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 weeks

Population: No analyses were conducted since it is an open-label trial and data was analyzed in the double-blind phase. Additionally, the main reason for the this phase is to allow participants who were on the placebo during the double-blind phase of NCT00627705 to receive the compound.

Outcome measures

Outcome data not reported

Adverse Events

N-Acetyl Cysteine

Serious events: 0 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	N-Acetyl Cysteine n=24 participants at risk N-Acetyl Cysteine: Dosage of orally administered N-Acetyl Cysteine is as follows: Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day
Gastrointestinal disorders Nausea/Vomiting	8.3% 2/24 • Number of events 3 • Baseline and 4, 8 and 12 Weeks.
Gastrointestinal disorders Diarrhea	12.5% 3/24 • Number of events 3 • Baseline and 4, 8 and 12 Weeks.
Gastrointestinal disorders Decreased Appetite	20.8% 5/24 • Number of events 6 • Baseline and 4, 8 and 12 Weeks.
Psychiatric disorders Depressive Affect	12.5% 3/24 • Number of events 3 • Baseline and 4, 8 and 12 Weeks.
General disorders Nasal Congestion	25.0% 6/24 • Number of events 6 • Baseline and 4, 8 and 12 Weeks.
General disorders Insomnia	16.7% 4/24 • Number of events 4 • Baseline and 4, 8 and 12 Weeks.
General disorders Decreased Motor Activity	8.3% 2/24 • Number of events 2 • Baseline and 4, 8 and 12 Weeks.
General disorders Grinding Teeth	8.3% 2/24 • Number of events 2 • Baseline and 4, 8 and 12 Weeks.

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