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Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

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ClinicalTrials.gov Identifier: NCT00028262
Recruitment Status : Completed
First Posted : December 18, 2001
Results First Posted : January 21, 2015
Last Update Posted : October 27, 2016
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) )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Infantile Neronal Ceroid Lipofuscinosis
Intervention Drug: Cystagon
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Drug Cystagon and N-acetylcysteine
Hide Arm/Group Description [Not Specified]
Period Title: Overall Study
Started 10
Completed 9
Not Completed 1
Arm/Group Title Drug Cystagon and N-acetylcysteine
Hide Arm/Group Description [Not Specified]
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
10
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
5
  50.0%
Male
5
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Hispanic or Latino
1
  10.0%
Not Hispanic or Latino
8
  80.0%
Unknown or Not Reported
1
  10.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
10
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
1.Primary Outcome
Title Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells.
Hide Description The GRODs in peripheral white blood cells from all patients before and during treatment were analyzed by transmission electron microscopy (TEM) at 30000xmagnification. Two investigators working independently of each other identified and counted the GRODs and the results were averaged.
Time Frame 10 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Out of 10 enrolled subjects, one subject did not complete study and was lost to follow up.
Arm/Group Title Drug Cystagon and N-acetylcysteine
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 9
Mean (95% Confidence Interval)
Unit of Measure: Average number of GRODs
-0.3317
(-0.3943 to -0.2691)
Time Frame 10 years.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Drug Cystagon and N-acetylcysteine
Hide Arm/Group Description [Not Specified]
All-Cause Mortality
Drug Cystagon and N-acetylcysteine
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Drug Cystagon and N-acetylcysteine
Affected / at Risk (%)
Total   0/9 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Drug Cystagon and N-acetylcysteine
Affected / at Risk (%)
Total   0/9 (0.00%) 
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
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
First Submitted: December 17, 2001
First Posted: December 18, 2001
Results First Submitted: January 9, 2015
Results First Posted: January 21, 2015
Last Update Posted: October 27, 2016