NAC will be started at 500 mg by mouth twice daily for the first 2 weeks, then increased to 500 mg in the am and 1000 mg in the pm for week 3, and then increased to 1000 mg am and pm for weeks 4 through 8. Subjects will be maintained at the highest tolerated dose.

Autistic Disorder

Seizures

Irritability

N-Acetyl Cysteine (NAC)

Nationwide Children's Hospital

Treatment Resistant Epilepsy and N-Acetyl Cysteine

Pilot Study of N-acetyl Cysteine for Refractory Generalized Epilepsy in Children With Autism

screening to enrollment and retention rates will be measured

parent and child will be queried about changes in physical and mental health between study visits using a standardized form.

parent report form on symptoms and behaviors in autism

blood is collected and analyzed for oxidative changes in lipids, proteins.

Barbara Gracious

Principal Investigator

These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial

Placebo Phase A/NAC Phase B

These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B

NAC Phase A/Placebo Phase B

Total

Sex: Female, Male

Region of Enrollment

NAC Phase

Placebo Phase

Oxysterol Levels

Overall Study

Dr. Forbes D Porter, Senior Investigator

Niemann-Pick Disease, Type C

900mg effervescent tablet; Dosed as 15 mg/kg/day (maximum dose 900 mg per day) for one week, advanced to 30 mg/kg/day (maximum dose 1800 mg per day) for the second week, and then advanced to 60 mg/kg/day (maximum dose 2700 mg) for the remainder of the trial (6 additional weeks).

N-Acetyl Cysteine

National Institutes of Health Clinical Center, 9000 Rockville Pike

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

University of Oxford

Washington University School of Medicine

National Human Genome Research Institute (NHGRI)

NAC given plus 17Oh progesterone caproate

Preterm Labour

Arm 2, control group: given hydroxyl progesterone caproate (150mgm) every 3 days starting from the 28th week of pregnancy till completed 36 weeks of gestation.
Arm 1 patients were given hydroxyl progesterone caproate (150mgm) weekly injections plus NAC 0.6 gm (Sedico, Egypt) orally daily starting from the 28th week of pregnancy till delivery or completed 36th week of pregnancy.

cysteine/seizures

Treatment Resistant Epilepsy and N-Acetyl Cysteine

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

N-Acetyl Cysteine for Prevention of Preterm Birth

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