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Trial record 2 of 5 for:    "Lipid Storage Disease" | "Acetylcysteine"

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

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ClinicalTrials.gov Identifier: NCT00975689
Recruitment Status : Completed
First Posted : September 11, 2009
Results First Posted : July 12, 2012
Last Update Posted : May 6, 2013
Sponsor:
Collaborators:
University of Oxford
Washington University School of Medicine
National Human Genome Research Institute (NHGRI)
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Niemann-Pick Disease, Type C
Intervention Drug: N-Acetyl Cysteine
Enrollment 35
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Phase A/NAC Phase B NAC Phase A/Placebo Phase B
Hide Arm/Group Description These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B
Period Title: Overall Study
Started 17 18
Completed 15 15
Not Completed 2 3
Reason Not Completed
Adverse Event             0             3
Withdrawal by Subject             1             0
Physician Decision             1             0
Arm/Group Title Placebo Phase A/NAC Phase B NAC Phase A/Placebo Phase B Total
Hide Arm/Group Description These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B Total of all reporting groups
Overall Number of Baseline Participants 17 18 35
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 18 participants 35 participants
<=18 years
12
  70.6%
13
  72.2%
25
  71.4%
Between 18 and 65 years
5
  29.4%
5
  27.8%
10
  28.6%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants 18 participants 35 participants
Female
6
  35.3%
11
  61.1%
17
  48.6%
Male
11
  64.7%
7
  38.9%
18
  51.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 17 participants 18 participants 35 participants
United States 16 18 34
Costa Rica 1 0 1
1.Primary Outcome
Title Oxysterol Levels
Hide Description [Not Specified]
Time Frame Six months
Hide Outcome Measure Data
Hide Analysis Population Description
Every patient in the trial received the study drug as well as the placebo.
Arm/Group Title NAC Phase Placebo Phase
Hide Arm/Group Description:
[Not Specified]
[Not Specified]
Overall Number of Participants Analyzed 30 30
Mean (Standard Error)
Unit of Measure: ng/mL
28.09  (2.152) 27.64  (2.045)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo Phase A/NAC Phase B NAC Phase A/Placebo Phase B
Hide Arm/Group Description These patients were randomized to receive the placebo in Phase A and study drug (NAC) in Phase B of the trial These patients were randomized to receive study drug (NAC) in Phase A and placebo in Phase B
All-Cause Mortality
Placebo Phase A/NAC Phase B NAC Phase A/Placebo Phase B
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Phase A/NAC Phase B NAC Phase A/Placebo Phase B
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/17 (17.65%)      5/18 (27.78%)    
Blood and lymphatic system disorders     
Decreased blood counts *  1/17 (5.88%)  1 0/18 (0.00%)  0
Hepatobiliary disorders     
Elevated Liver Enzymes   0/17 (0.00%)  0 2/18 (11.11%)  2
Nervous system disorders     
Hospitalization for seizure * [1]  1/17 (5.88%)  1 2/18 (11.11%)  2
Respiratory, thoracic and mediastinal disorders     
Aspiration Pneumonia *  1/17 (5.88%)  1 1/18 (5.56%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
[1]
Unanticipated hospitalization
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Phase A/NAC Phase B NAC Phase A/Placebo Phase B
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/17 (0.00%)      0/18 (0.00%)    
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Forbes D Porter, Senior Investigator
Organization: National Institute of Child Health and Human Development; NIH
Phone: (301) 435-4432
EMail: fdporter@mail.nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) )
ClinicalTrials.gov Identifier: NCT00975689     History of Changes
Other Study ID Numbers: 090185
09-CH-0185
First Submitted: September 10, 2009
First Posted: September 11, 2009
Results First Submitted: May 23, 2012
Results First Posted: July 12, 2012
Last Update Posted: May 6, 2013