Trial Outcomes & Findings for A Study of Glutathione in Children With Autism Spectrum Disorder (NCT NCT05954052)

Results Overview

The Aberrant Behavior Checklist (ABC) measures psychiatric symptoms and behavioral disturbances in individuals with IDD across five subscales: Irritability (0-45), Social Withdrawal (0-48), Stereotypic Behavior (0-21), Hyperactivity (0-48), and Inappropriate Speech (0-12). Each subscale score is summed to calculate a total score ranging from 0 to 174. Higher scores indicate worse outcomes, with no specific cutoff values, as the measure is used alongside diagnostic tools like the Autism Diagnostic Observation Schedule (ADOS). The ABC is designed to establish baseline symptoms and monitor changes over time, where a decrease in scores reflects improvement, and an increase indicates worsening symptoms.The Aberrant Behavior Checklist (ABC) consists of five subscales: Irritability (0-45), Lethargy (0-48), Stereotypic Behavior (0-21), Hyperactivity (0-48), and Inappropriate Speech (0-12). Higher scores represent more severe symptoms. The total ABC score ranges from 0 to 174

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

6 participants

Primary outcome timeframe

baseline and 12 weeks. To clarify, all pre-specified Primary and Secondary Outcome Measures were collected at baseline and the 12-week endpoint only. No intermediate assessments were conducted.

Results posted on

2025-09-18

Participant Flow

Participant milestones

Participant milestones
Measure	Glutathione Oral Supplementation Glutathione will be the only study medication dispensed to subjects for this study. Subjects will be told to take the first dose of study medication after breakfast each day and the second dose in the evening after dinner. The proposed dose range for Glutathione in this study will be 1000mg-3000mg/day based on the subject's weight. Glutathione: Giving supplement orally
Overall Study STARTED	6
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Glutathione Oral Supplementation Glutathione will be the only study medication dispensed to subjects for this study. Subjects will be told to take the first dose of study medication after breakfast each day and the second dose in the evening after dinner. The proposed dose range for Glutathione in this study will be 1000mg-3000mg/day based on the subject's weight. Glutathione: Giving supplement orally
Overall Study Adverse Event	1

Baseline Characteristics

A Study of Glutathione in Children With Autism Spectrum Disorder

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Glutathione Oral Supplementation n=6 Participants Glutathione will be the only study medication dispensed to subjects for this study. Subjects will be told to take the first dose of study medication after breakfast each day and the second dose in the evening after dinner. The proposed dose range for Glutathione in this study will be 1000mg-3000mg/day based on the subject's weight. Glutathione: Giving supplement orally
Age, Categorical <=18 years	6 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
Age, Continuous	14.67 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	6 Participants n=5 Participants

PRIMARY outcome

Timeframe: baseline and 12 weeks. To clarify, all pre-specified Primary and Secondary Outcome Measures were collected at baseline and the 12-week endpoint only. No intermediate assessments were conducted.

Population: All participants who received at least one dose of oral glutathione and had both baseline and Week 12 (or endpoint) Aberrant Behavior Checklist (ABC) assessments available (n=5). One participant discontinued after 2 weeks due to increased irritability and was excluded from the change analysis

The Aberrant Behavior Checklist (ABC) measures psychiatric symptoms and behavioral disturbances in individuals with IDD across five subscales: Irritability (0-45), Social Withdrawal (0-48), Stereotypic Behavior (0-21), Hyperactivity (0-48), and Inappropriate Speech (0-12). Each subscale score is summed to calculate a total score ranging from 0 to 174. Higher scores indicate worse outcomes, with no specific cutoff values, as the measure is used alongside diagnostic tools like the Autism Diagnostic Observation Schedule (ADOS). The ABC is designed to establish baseline symptoms and monitor changes over time, where a decrease in scores reflects improvement, and an increase indicates worsening symptoms.The Aberrant Behavior Checklist (ABC) consists of five subscales: Irritability (0-45), Lethargy (0-48), Stereotypic Behavior (0-21), Hyperactivity (0-48), and Inappropriate Speech (0-12). Higher scores represent more severe symptoms. The total ABC score ranges from 0 to 174

Outcome measures

Outcome measures
Measure	Glutathione n=5 Participants We have one arm who took supplement
Aberrant Child Checklist, Looking at Change in the Subscale of the ABC ABC: Irritability	18.60 units on a scale Interval 15.56 to 20.64
Aberrant Child Checklist, Looking at Change in the Subscale of the ABC ABC: Lethargy	14.40 units on a scale Interval 10.99 to 17.81
Aberrant Child Checklist, Looking at Change in the Subscale of the ABC ABC: Stereotypy	8.80 units on a scale Interval 7.01 to 10.59
Aberrant Child Checklist, Looking at Change in the Subscale of the ABC ABC: Hyperactivity	14.80 units on a scale Interval 13.25 to 16.35
Aberrant Child Checklist, Looking at Change in the Subscale of the ABC ABC: Inappropriate Speech	6.40 units on a scale Interval 5.12 to 7.68

SECONDARY outcome

Timeframe: We only have the Social Responsiveness Scale measurement at baseline.

Population: All participants who were enrolled and completed the baseline Social Responsiveness Scale (SRS) assessment (n=6)

The Social Responsiveness Scale, Second Edition (SRS-2) is a 65-item caregiver-rated questionnaire that measures the severity of social impairments associated with autism spectrum disorder (ASD) in children and adolescents (ages 2 years 6 months to 18 years). It assesses five domains: Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests and Repetitive Behavior, as well as a Total Score. T-scores typically range from approximately 30 to 90+, with scores capped at 90 in cases of severe impairment. Higher T-scores indicate greater severity of social impairment (worse outcome), while lower T-scores indicate fewer social difficulties (better outcome). Clinically Relevant Thresholds T-score ≤ 59: Within normal limits T-score 60-65: Mild range (borderline, subclinical difficulties) T-score 66-75: Moderate range (clinically significant impairment) T-score ≥ 76: Severe range (marked impairment)

Outcome measures

Outcome measures
Measure	Glutathione n=6 Participants We have one arm who took supplement
Social Responsiveness Scale Social Awareness	75.5 T-score for this instrument. Interval 58.25 to 86.75
Social Responsiveness Scale Social Cognition	85.0 T-score for this instrument. Interval 78.5 to 89.25
Social Responsiveness Scale Social Communication	90.00 T-score for this instrument. Interval 64.5 to 90.0
Social Responsiveness Scale Social Motivation	82.0 T-score for this instrument. Interval 75.0 to 83.75
Social Responsiveness Scale Autism Mannerisms	89.0 T-score for this instrument. Interval 77.5 to 89.75
Social Responsiveness Scale Total Severity	89 T-score for this instrument. Interval 73.0 to 90.0

OTHER_PRE_SPECIFIED outcome

Timeframe: At baseline and the end of the trial (12 weeks)

The Clinical Global Impression (CGI) is a clinician-rated instrument developed for use in NIMH-sponsored clinical trials to provide a brief global assessment of illness severity, improvement, and therapeutic efficacy. The three commonly reported CGI scales are: CGI-Severity (CGI-S): Rates current illness severity from 1 (Normal, not at all ill) to 7 (Among the most extremely ill patients). CGI-Improvement (CGI-I): Rates change in clinical status from baseline on a 7-point scale, ranging from 1 (Very much improved) to 7 (Very much worse). CGI-Efficacy Index (CGI-E): Balances therapeutic efficacy with adverse effects, rated on a 4 × 4 grid, where higher scores reflect minimal efficacy and/or significant side effects. Lower CGI-S and CGI-I scores indicate better outcomes (less illness severity and/or greater improvement). Higher CGI-E scores reflect worse outcomes (limited efficacy and/or significant side effects). Higher CGI-E scores reflect worse outcomes

Outcome measures

Outcome measures
Measure	Glutathione n=6 Participants We have one arm who took supplement
Change in Clinical Global Impression Scale Pre-Treatment CGI Severity	4 score on a scale Interval 3.25 to 4.75
Change in Clinical Global Impression Scale Post-Treatment CGI Severity	4.5 score on a scale Interval 3.25 to 5.0
Change in Clinical Global Impression Scale CGI Improvement	3 score on a scale Interval 3.0 to 3.75
Change in Clinical Global Impression Scale Efficacy Index	9 score on a scale Interval 9.0 to 11.25

POST_HOC outcome

Timeframe: 12 weeks

Pre-treatment CGI-S ratings ranged from 2 (borderline ill) to 5 (markedly ill). One subject's CGI-S changed from 4 (moderately ill) to 5 (markedly ill); others had no change.

Outcome measures

Outcome data not reported

POST_HOC outcome

Timeframe: 12 weeks

1 subject scored 4 (no change), four scored 3 (minimally improved), and one scored 6 (much worse).

Outcome measures

Outcome data not reported

POST_HOC outcome

Timeframe: 12 weeks

two patients had no therapeutic changes, four had minimal efficacy, five had no side effects, and one dropped out due to significant side effects

Outcome measures

Outcome data not reported

Adverse Events

Glutathione

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Glutathione n=6 participants at risk We have one arm who took supplement
Nervous system disorders 1	16.7% 1/6 • Number of events 1 • 12 weeks collected using interview with the family and patient
Nervous system disorders irritability	16.7% 1/6 • Number of events 1 • 12 weeks collected using interview with the family and patient

Other adverse events

Other adverse events
Measure	Glutathione n=6 participants at risk We have one arm who took supplement
Nervous system disorders 1	16.7% 1/6 • Number of events 1 • 12 weeks collected using interview with the family and patient

Additional Information

Karam Radwan, MD

University of Chicago

Phone: 7738346686

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place