Trial Outcomes & Findings for Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine (NCT NCT00975689)

Last Updated: 2013-05-06

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

35 participants

Primary outcome timeframe

Six months

Results posted on

2013-05-06

Participant milestones
Measure	Placebo Phase A/NAC Phase B These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial	NAC Phase A/Placebo Phase B These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B
Overall Study STARTED	17	18
Overall Study COMPLETED	15	15
Overall Study NOT COMPLETED	2	3

Reasons for withdrawal
Measure	Placebo Phase A/NAC Phase B These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial	NAC Phase A/Placebo Phase B These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B
Overall Study Adverse Event	0	3
Overall Study Withdrawal by Subject	1	0
Overall Study Physician Decision	1	0

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

Baseline characteristics by cohort
Measure	Placebo Phase A/NAC Phase B n=17 Participants These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial	NAC Phase A/Placebo Phase B n=18 Participants These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B	Total n=35 Participants Total of all reporting groups
Age, Categorical <=18 years	12 Participants n=99 Participants	13 Participants n=107 Participants	25 Participants n=206 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=99 Participants	5 Participants n=107 Participants	10 Participants n=206 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Sex: Female, Male Female	6 Participants n=99 Participants	11 Participants n=107 Participants	17 Participants n=206 Participants
Sex: Female, Male Male	11 Participants n=99 Participants	7 Participants n=107 Participants	18 Participants n=206 Participants
Region of Enrollment United States	16 participants n=99 Participants	18 participants n=107 Participants	34 participants n=206 Participants
Region of Enrollment Costa Rica	1 participants n=99 Participants	0 participants n=107 Participants	1 participants n=206 Participants

Timeframe: Six months

Population: Every patient in the trial received the study drug as well as the placebo.

Outcome measures
Measure	NAC Phase n=30 Participants	Placebo Phase n=30 Participants
Oxysterol Levels	28.09 ng/mL Standard Error 2.152	27.64 ng/mL Standard Error 2.045

Serious events: 3 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious events: 5 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events
Measure	Placebo Phase A/NAC Phase B n=17 participants at risk These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial	NAC Phase A/Placebo Phase B n=18 participants at risk These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B
Hepatobiliary disorders Elevated Liver Enzymes	0.00% 0/17	11.1% 2/18 • Number of events 2
Nervous system disorders Hospitalization for seizure	5.9% 1/17 • Number of events 1	11.1% 2/18 • Number of events 2
Respiratory, thoracic and mediastinal disorders Aspiration Pneumonia	5.9% 1/17 • Number of events 1	5.6% 1/18 • Number of events 1
Blood and lymphatic system disorders Decreased blood counts	5.9% 1/17 • Number of events 1	0.00% 0/18

Adverse event data not reported

National Institute of Child Health and Human Development; NIH

Phone: (301) 435-4432