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Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

This study has been completed.
Sponsor:
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:
NCT00028262
First received: December 17, 2001
Last updated: September 21, 2016
Last verified: September 2016
Results First Received: January 9, 2015  
Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Infantile Neronal Ceroid Lipofuscinosis
Intervention: Drug: Cystagon

  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
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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
Drug Cystagon and N-acetylcysteine No text entered.

Participant Flow:   Overall Study
    Drug Cystagon and N-acetylcysteine
STARTED   10 
COMPLETED   9 
NOT COMPLETED   1 



  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.
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Reporting Groups
  Description
Drug Cystagon and N-acetylcysteine No text entered.

Baseline Measures
   Drug Cystagon and N-acetylcysteine 
Overall Participants Analyzed 
[Units: Participants]
 10 
Age 
[Units: Participants]
 
<=18 years   10 
Between 18 and 65 years   0 
>=65 years   0 
Gender 
[Units: Participants]
 
Female   5 
Male   5 
Ethnicity (NIH/OMB) 
[Units: Participants]
 
Hispanic or Latino   1 
Not Hispanic or Latino   8 
Unknown or Not Reported   1 
Race (NIH/OMB) 
[Units: Participants]
 
American Indian or Alaska Native   0 
Asian   0 
Native Hawaiian or Other Pacific Islander   0 
Black or African American   0 
White   10 
More than one race   0 
Unknown or Not Reported   0 


  Outcome Measures

1.  Primary:   Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells.   [ Time Frame: 10 years ]


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


Results Point of Contact:  
Name/Title: Dr. Anil Mukherjee
Organization: National Institute of Child Health and Human Development
phone: +1 301 496 7213
e-mail: mukherja@exchange.nih.gov


Publications of Results:
Other 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: NCT00028262     History of Changes
Other Study ID Numbers: 010086
01-CH-0086
Study First Received: December 17, 2001
Results First Received: January 9, 2015
Last Updated: September 21, 2016
Health Authority: United States: Federal Government