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)

Patients randomized to this arm will receive N-Acetylcysteine augmentation, at a standard dose (3000 mg daily), in addition to the medication regimen they are on at enrollment

Patients randomized to this arm will receive placebo, formulated to be indistinguishable from N-Acetylcysteine, in addition to the medication regimen they are on at study enrollment.

Patients randomized to this arm will receive N-Acetylcysteine augmentation, at a standard dose (3000 mg daily), in addition to the medication regimen they are on at enrollment
N-Acetylcysteine : 3000 mg PO (1200 mg AM, 1800 mg PM), 6 weeks

N-Acetylcysteine

Patients randomized to this arm will receive placebo, formulated to be indistinguishable from N-Acetylcysteine, in addition to the medication regimen they are on at study enrollment.
placebo : placebo, 2 capsules PO AM, 3 capsules PO PM, 6 weeks

Placebo

The Self-Harm Inventory is assessed by asking an individual to answer (yes or no) if they have ever "intentionally, or on purpose" tried to harm themselves. The inventory contains 22 questions and a 23rd marked "other" that allows the individual to indicate a self-harm behavior not previously mentioned.
The scoring of this instrument is determined by counting the number of endorsed self-harm behaviors out of the possible twenty-three asked. The maximum score any individual may achieve for the SHI is a 23. Any individual scoring 5 or greater is classified as suffering from BPD.
In this study, scoring on the SHI was primarily used to assess improvement of self-harming symptoms and throughout the study by comparing participant ratings from baseline and week 6. Positive numbers indicate a decrease (i.e. participant indicated less self-harming behavior) and negative numbers indicate an increase in self-harming behaviors reported.

Self-Harm Inventory (SHI) Score at 6 Weeks

The Hamilton Rating Scale for Depression is a multiple item questionnaire used to provide an indication of depression, and as a guide to evaluate recovery. Administered by a clinician, The questionnaire is designed for adults and is used to rate the severity of the patients depression by asking their mood, feelings of guilt, insomnia, agitation, weight change, suicidal ideation, and somatic symptoms. The scale also allows the clinician to assess the patient's level of retardation, and insight into their depression. Highest possible score is 52.
HAM-D Scoring 0-7 = Normal 8-13 = Mild Depression 14-18 = Moderate Depression 19-22 = Severed Depression
≥23 = Very Severe Depression
In this study, Baseline ratings were compared to those of week 6 to assess each participants change in depression throughout the study. A negative value indicates an increase in depression (i.e. the individual felt more depressed) and a positive value indicates a decrease in depression.

Hamilton Depression Rating Scale (HAM-D) at 6 Weeks

Self-Harm Inventory (SHI) Score at Baseline

Hamilton Depression Rating Scale (HAM-D) at Baseline

Christopher Pittenger, MD Ph.D

Borderline Personality Disorder

Self-Injurious Behavior

3000 mg PO (1200 mg AM, 1800 mg PM), 6 weeks

placebo, 2 capsules PO AM, 3 capsules PO PM, 6 weeks

placebo

Yale OCD Research Clinic

N-Acetylcysteine in Adjunct to DBT for the Treatment of Self-Injurious Behavior in BPD

A Double-Blind, Placebo-controlled Pilot Study of NAC Addition to Dialectical Behavioral Therapy for the Treatment of Self-Injurious Behavior Associated With Borderline Personality Disorder

Christopher Pittenger

Principal Investigator

Yale University

N-Acetylcysteine and CRRT Patients are assigned to N-Acetylcysteine and CRRT. The N-Acetylcysteine is blinded to everyone except pharmacy. The CRRT is open label,

Patients are assigned to N-Acetylcysteine and CRRT. The N-Acetylcysteine is blinded to everyone except pharmacy. The CRRT is open label as would be impossible to blind

Patients are assigned to placebo treatment and CRRT. The N-Acetylcysteine/placebo is blinded to everyone except pharmacy. The CRRT is open label.

Patients are assigned to Placebo and non-CRRT. This is the standard of care arm. The N-Acetylcysteine/placebo is blinded to everyone except pharmacy. The CRRT/non CRRT is open label as would be impossible to blind

Rhabdomyolysis

Patients are assigned to either N-Acetylcysteine or placebo. Dose is weight based Placebo is normal saline or D5W

N-Acetylcystine and Non CRRT

Patients are assigned to Placebo and CRRT. The N-Acetylcysteine/Placebo is blinded to everyone except pharmacy. The CRRT is open label

Placebo and CRRT

Patients are assigned to Placebo and non-CRRT. The N-Acetylcysteine/placebo is blinded to everyone except pharmacy. The CRRT/non CRRT is open label as would be impossible to blind

Placebo and Non CRRT

Royal Alexandra Hospital

King Fahad National Guard Hospital

Study of N-Acetylcysteine (NAC) and Continuous Renal Replacement Therapy (CRRT) for the Treatment of Rhabdomyolysis

A Randomized Factorial Trial of N-Acetylcysteine and Continuous Veno-Venous Hemo(Dia)Filtration for Rhabdomyolysis

Demetrios J. Kutsogiannis

MD MHS FRCPC

University of Alberta

Gambro Renal Products, Inc.

Drug Cystagon and N-acetylcysteine

Ethnicity (NIH/OMB)

Race (NIH/OMB)

The GRODs in peripheral white blood cells from all patients before and during treatment were analyzed by transmission electron microscopy (TEM) at 30000xmagnification. Two investigators working independently of each other identified and counted the GRODs and the results were averaged.

acetylcysteine/seizures

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

N-Acetylcysteine in Adjunct to DBT for the Treatment of Self-Injurious Behavior in BPD

Study of N-Acetylcysteine (NAC) and Continuous Renal Replacement Therapy (CRRT) for the Treatment of Rhabdomyolysis

Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

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