Study of Glutathione, Vitamin C and Cysteine in Children With Autism and Severe Behavior Problems

Autism Clinical Trial

Official title:

Double-Blind, Placebo-Controlled, Crossover Study of Glutathione, Vitamin C and Cysteine in Children With Autism and Severe Behavior Problems

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00889538
• Other study ID #: Glutathione-KCPCRU-01
• Status: Completed
• Phase: N/A
• First received: April 28, 2009
• Last updated: December 6, 2016
• Start date: April 2009
• Est. completion date: September 2012

Study information

• Verified date: December 2016
• Source: University of Louisville
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

This is a double-blind, placebo-controlled crossover study to evaluate the safety and efficacy of glutathione alone or glutathione, vitamin C and NAC treatment in children with autism who also have severe behavior problems. The investigators hypothesis is that children with autism will show improvement in both learning capabilities and behavior with either glutathione, or glutathione, vitamin C and NAC therapy.

Description:

This is a prospective, single center, double-blind, randomized pilot study in children and adolescents with autism and severe behavior disorders. All subjects will undergo initial screening procedures to determine eligibility. They will then be randomized to either 8 weeks of placebo or 8 weeks of glutathione or glutathione, vitamin C and N-acetylcysteine. They will receive intravenous treatment weekly and will have ongoing behavioral studies during this period. Following 8 weeks of therapy, they will have a week without treatment then will cross-over to the alternate therapy for weekly intravenous infusions and behavioral testing. In addition to baseline hematology and chemistries, baseline oxidized and reduced glutathione will be measured. These parameters will be repeated at the end of each 8 week course of therapy.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: September 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years to 16 Years

Eligibility

Inclusion Criteria:

• Age 5 to 16 years, inclusive

• Diagnosed with autism using the ADI-R (Autism Diagnostic Interview-Revised)

• ABC Irritability/Agitation subscale score (Aberrant Behavior Checklist Subscale) = 18

• CGI-S score (Clinical Global Impression-Severity Scale) = 4

• Must be drug naïve or on a stable dose of medication(s) and educational/behavioral interventions for one month prior to participation

• Parent/legal guardian must provide written consent and required research authorization (i.e. HIPAA) prior to the performance of any study procedures.

Exclusion Criteria:

• Presence of another disorder on the autism spectrum including PDD-NOS (Pervasive Developmental Disorder Not Otherwise Specified), Asperger's, and Rett's Syndrome

• Patient with a known cause of autism such as Fragile X

• Evidence of significant renal dysfunction (e.g. GFR estimated by the Schwartz formula < 50 mL/min or serum creatinine > 2.5 X upper limit of normal for age)

• Evidence of significant hepatic dysfunction (serum transaminases > 2.5 X the upper limit of normal)

• Known hypersensitivity to glutathione, vitamin C or NAC

• Pregnant or lactating female

• Inability of subject and parent to be able to comply with requirements for study visits and procedures

• Presence of major mental illness

• History of antioxidant supplementation.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autism
• Autistic Disorder
• Mental Disorders
• Problem Behavior
• Severe Behavior Disorder

Intervention

Drug:
• placebo
50 mL of 1/2 normal saline IV
• glutathione
glutathione 600 mg IV
• glutathione, vit C and NAC
Glutathione 600 mg IV Vitamin C 2000 mg IV N-acetylcysteine 20 mg/kg IV (max dose = 600 mg) Mix above three in 1/2 NS for a total volume of 50 mL

Locations

Country	Name	City	State
United States	KCPCRU	Louisville	Kentucky

Sponsors (3)

Lead Sponsor	Collaborator
University of Louisville	Cumberland Pharmaceuticals, Norton Healthcare

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvement in both developmental skills and behavior with either glutathione or glutathione, Vitamin C and N-acetylcysteine therapy as compared to placebo therapy. Subjects will also be monitored using clinical and laboratory safety parameters.	Improvement in both developmental skills and behavior measured using validated scales in subjects receiving either glutathione, glutathione/Vitamin C/N-acetylcysteine or placebo measured at baseline, 9 weeks and at the end of study in a double-blind cross-over design.	4 months	Yes
Secondary	Response to glutathione (changes in behavior) will correlate with the glutathione level (GSH) and GSH:GSSG ratio		Baseline	No
Secondary	Response to glutathione (changes in behavior) will correlate with the glutathione level (GSH) and GSH:GSSG ratio		9 weeks	No
Secondary	Response to glutathione (changes in behavior) will correlate with the glutathione level (GSH) and GSH:GSSG ratio		18 weeks	No