Autistic Disorder
Conditions
Keywords
N-Acetyl Cysteine
Brief summary
The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism.
Detailed description
N-Acetyl Cysteine (NAC) is a compound that increases the levels of Glutathione, the body's main antioxidant. Glutathione is a compound in the blood that is part of a natural defense system (the antioxidant system). Anti-oxidants protect the body from damage caused by internal toxins called free radicals. It is possible that children with Autism tend to have lower levels of glutathione, an important compound in our bodies that helps combat the effects of toxic free radicals. We hope that by studying the antioxidant system in more detail, we will increase our understanding of the reasons why people develop Autism so that we can design better ways to treat individuals with this condition. This study is meant to test the safety tolerability of N-Acetyl Cysteine and its effectiveness in the treatment of behavioral difficulties in children with autism. It will also examine the possible benefit of this agent in improving the core deficits in autism such as social deficits.
Interventions
DRUGN-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
Sponsors
Stanford University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
Subjects will be eligible for this study if they participated in the Double-blind, randomized, placebo controlled study of N-Acetyl Cysteine in Autism study at Stanford University and meet all of the following criteria: 1. Outpatients between 3.0 and 12.11 years of age inclusive 2. Males and females who are physically healthy 3. diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation 4. Clinical Global Impression Severity rating of 4 5. Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis 6. Ability of subject to swallow the compound 7. Stable concomitant medications for at least 2 weeks 8. No planned changes in psychosocial interventions during the open-label N-Acetyl Cysteine trial
Exclusion criteria
1. Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified 2. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology) 3. Pregnancy or sexually active females 4. Subjects taking antioxidant agents and glutathione prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) | 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). |
Secondary
| Measure | Time frame |
|---|---|
| Social Responsiveness Scale (SRS) | 12 weeks |
| Sensory Profile Questionnaire (SPQ) | 12 weeks |
| Irritability Subscale of the Aberrant Behavior Checklist (ABC) | 4, 8, and 12 weeks |
| Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography | 12 weeks |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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 | 24 |
| Total | 24 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Physician Decision | 1 |
Baseline characteristics
| Characteristic | N-Acetyl Cysteine |
|---|---|
| Age, Categorical <=18 years | 24 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Region of Enrollment United States | 24 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 22 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 19 / 24 |
| serious Total, serious adverse events | 0 / 24 |
Outcome results
Primary
Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES)
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).
Time frame: 4, 8, and 12 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| N-Acetyl Cysteine | Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) | 17 number of participants |
Secondary
Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography
Time frame: 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.
Secondary
Irritability Subscale of the Aberrant Behavior Checklist (ABC)
Time frame: 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.
Secondary
Sensory Profile Questionnaire (SPQ)
Time frame: 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.
Secondary
Social Responsiveness Scale (SRS)
Time frame: 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.