A Preliminary Study to Evaluate Cysteamine Therapy in Human Subjects With Non-Alcoholic Steatohepatitis (NASH)

This study has been completed.
Sponsor:
Collaborator:
Raptor Pharmaceuticals Corp.
Information provided by (Responsible Party):
Joel Lavine, University of California, San Diego
ClinicalTrials.gov Identifier:
NCT00799578
First received: November 28, 2008
Last updated: December 13, 2013
Last verified: December 2013
Results First Received: December 13, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Fatty Liver
Intervention: Drug: Cysteamine

  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
Biopsy proven children with nonalcoholic fatty liver disease from fatty liver clinic at University of California, San Diego

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Not on other therapy for NAFLD

Reporting Groups
  Description
Cystagon-EC No text entered.

Participant Flow:   Overall Study
    Cystagon-EC  
STARTED     13  
COMPLETED     11  
NOT COMPLETED     2  



  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
Cystagon-EC No text entered.

Baseline Measures
    Cystagon-EC  
Number of Participants  
[units: participants]
  13  
Age  
[units: participants]
 
<=18 years     13  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  14  ± 1  
Gender  
[units: participants]
 
Female     1  
Male     12  
Region of Enrollment  
[units: participants]
 
United States     13  



  Outcome Measures

1.  Primary:   Normalization or >50% of Serum ALT Levels From Baseline   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Joel Lavine
Organization: Columbia University
phone: 212 304 5533
e-mail: jl3553@columbia.edu


Publications:


Responsible Party: Joel Lavine, University of California, San Diego
ClinicalTrials.gov Identifier: NCT00799578     History of Changes
Other Study ID Numbers: 07-1699
Study First Received: November 28, 2008
Results First Received: December 13, 2013
Last Updated: December 13, 2013
Health Authority: United States: Food and Drug Administration