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

This study has been completed.
Sponsor:
Collaborators:
University of Oxford
Washington University School of Medicine
Information provided by (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
First received: September 10, 2009
Last updated: April 30, 2013
Last verified: April 2013
Results First Received: May 23, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator);   Primary Purpose: Treatment
Condition: Niemann-Pick Disease, Type C
Intervention: Drug: N-Acetyl Cysteine

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
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

Participant Flow:   Overall Study
    Placebo Phase A/NAC Phase B     NAC Phase A/Placebo Phase B  
STARTED     17     18  
COMPLETED     15     15  
NOT COMPLETED     2     3  
Adverse Event                 0                 3  
Withdrawal by Subject                 1                 0  
Physician Decision                 1                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
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
Total Total of all reporting groups

Baseline Measures
    Placebo Phase A/NAC Phase B     NAC Phase A/Placebo Phase B     Total  
Number of Participants  
[units: participants]
  17     18     35  
Age  
[units: participants]
     
<=18 years     12     13     25  
Between 18 and 65 years     5     5     10  
>=65 years     0     0     0  
Gender  
[units: participants]
     
Female     6     11     17  
Male     11     7     18  
Region of Enrollment  
[units: participants]
     
United States     16     18     34  
Costa Rica     1     0     1  



  Outcome Measures

1.  Primary:   Oxysterol Levels   [ Time Frame: Six months ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Dr. Forbes D Porter, Senior Investigator
Organization: National Institute of Child Health and Human Development; NIH
phone: (301) 435-4432
e-mail: fdporter@mail.nih.gov


Publications:

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
Study First Received: September 10, 2009
Results First Received: May 23, 2012
Last Updated: April 30, 2013
Health Authority: United States: Food and Drug Administration