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| ID | Type | Description | Link |
|---|---|---|---|
| 10142 |
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The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism. 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 NAC 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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| N-Acetyl Cysteine | Active Comparator | active compound N-Acetyl Cysteine |
|
| Sugar pill | Placebo Comparator | Placebo or sugar pill |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| N-Acetyl Cysteine | Drug | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES) | 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. | 4, 8, and 12 weeks |
| The Clinical Global Rating Scale (CGRS) Improvement Subscale Score | Score range 1-7 (lower score mean more improvement compared to baseline) | 12 weeks |
| Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC) | Data not collected. The laboratory was not able to measure Glutathione levels. | 12 weeks |
| Irritability Subscale of the Aberrant Behavior Checklist (ABC) | Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability | baseline and 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| The Aberrant Behavior Checklist Total Score (ABC) | Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score. | 4, 8, and 12 weeks |
| Social Responsiveness Scale (SRS) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Antonio Hardan, MD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University School of Medicine | Stanford | California | 94305 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22342106 | Result | Hardan AY, Fung LK, Libove RA, Obukhanych TV, Nair S, Herzenberg LA, Frazier TW, Tirouvanziam R. A randomized controlled pilot trial of oral N-acetylcysteine in children with autism. Biol Psychiatry. 2012 Jun 1;71(11):956-61. doi: 10.1016/j.biopsych.2012.01.014. Epub 2012 Feb 18. |
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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). |
| FG001 | 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). |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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). |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES) | 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. | We analyzed subjects who had follow-up data available. | Posted | Number | participants | 4, 8, and 12 weeks |
|
Baseline and 4, 8 and 12 Weeks
We analyzed all subjects who had follow-up data available.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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). |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
The sample size was relatively small and the age range was narrow, which limits the generalizability of the findings. Most subjects were taking psychotropic medications and were receiving behavioral interventions.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Antonio Hardan, MD | Stanford University School of Medicine | 650-736-1235 | hardanay@stanford.edu |
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| ID | Term |
|---|---|
| D001321 | Autistic Disorder |
| ID | Term |
|---|---|
| D000067877 | Autism Spectrum Disorder |
| D002659 | Child Development Disorders, Pervasive |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D000111 | Acetylcysteine |
| ID | Term |
|---|---|
| D003545 | Cysteine |
| D000603 | Amino Acids, Sulfur |
| D013457 | Sulfur Compounds |
| D009930 | Organic Chemicals |
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|
| Placebo - sugar pill | Other | 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). |
|
|
SRS total score (range 0-195); higher scores mean more social impairment |
| 12 weeks |
| Sensory Profile Questionnaire (SPQ) | 12 weeks |
| Glutathione (GSH) Metabolism Intermediates in Peripheral Blood | 12 weeks |
| Withdrawal by Subject |
|
| Lost to Follow-up |
|
| 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). |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | 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). |
|
|
| Primary | The Clinical Global Rating Scale (CGRS) Improvement Subscale Score | Score range 1-7 (lower score mean more improvement compared to baseline) | We analyzed subjects who had follow-up data available. | Posted | Mean | Standard Deviation | score (range 1-7) | 12 weeks |
|
|
|
|
| Primary | Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC) | Data not collected. The laboratory was not able to measure Glutathione levels. | Data not collected. | Posted | 12 weeks |
|
|
| Primary | Irritability Subscale of the Aberrant Behavior Checklist (ABC) | Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability | We analyzed subjects who had follow-up data available. | Posted | Mean | Standard Deviation | Score (range 0-45) | baseline and 12 weeks |
|
|
|
|
| Secondary | The Aberrant Behavior Checklist Total Score (ABC) | Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score. | Measure not analyzed. | Posted | 4, 8, and 12 weeks |
|
|
| Secondary | Social Responsiveness Scale (SRS) | SRS total score (range 0-195); higher scores mean more social impairment | We analyzed subjects who had follow-up data available. | Posted | Mean | Standard Deviation | SRS total score (range 0-195) | 12 weeks |
|
|
|
|
| Secondary | Sensory Profile Questionnaire (SPQ) | Data not collected. Measure not analyzed. | Posted | 12 weeks |
|
|
| Secondary | Glutathione (GSH) Metabolism Intermediates in Peripheral Blood | Data not collected. The measure was not analyzed. The lab was not able to measure Glutathione for the study. | Posted | 12 weeks |
|
|
| 0 |
| 14 |
| 13 |
| 14 |
| EG001 | 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). | 0 | 15 | 14 | 15 |
| Nausea/Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Increased Appetite | Gastrointestinal disorders | Systematic Assessment |
|
| Decreased Appetite | Gastrointestinal disorders | Systematic Assessment |
|
| Akathisia | General disorders | Systematic Assessment |
|
| Excitement/ Agitation | General disorders | Systematic Assessment |
|
| Increased Motor Activity | General disorders | Systematic Assessment |
|
| Tremor | General disorders | Systematic Assessment |
|
| Syncope/Dizziness | General disorders | Systematic Assessment |
|
| Depressive Affect | Psychiatric disorders | Systematic Assessment |
|
| Nasal Congestion | General disorders | Systematic Assessment |
|
| Increased Salivation | General disorders | Systematic Assessment |
|
| Sweating | General disorders | Systematic Assessment |
|
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| D000596 |
| Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |