Status:
COMPLETED
Genotype - Phenotype Correlations of LINCL
Lead Sponsor:
Weill Medical College of Cornell University
Collaborating Sponsors:
Nathan's Battle Foundation
Conditions:
Batten Disease
Late-Infantile Neuronal Ceroid Lipfuscinosis
Eligibility:
All Genders
2-18 years
Brief Summary
In a separate protocol the Department of Genetic Medicine is proposing to carry out a study using gene transfer to treat the central nervous system (CNS) manifestations of late infantile neuronal cero...
Detailed Description
This proposed clinical protocol is designed to assess genotype - phenotype correlations of LINCL, including a preliminary assessment regarding the genotype - phenotype correlations of progressive CNS ...
Eligibility Criteria
Inclusion
- A definitive diagnosis of late infantile neuronal ceroid lipofuscinosis
- Between the age of 2 and 18 years
- Not previously participated in a gene transfer study for LINCL.
- Parents of study participants must agree to comply in good faith with the conditions of the study, including attending all of the required baseline and follow-up assessments.
- Both parents or legal guardians must give consent for their child's participation in the research study.
Exclusion
- Individuals with heart disease that would be a risk for anesthesia.
- Concurrent participation in any other FDA approved Investigational New Drug clinical protocol is not allowed, although the Principal Investigator will work with other doctors to accommodate specific requests (e.g., a study of nutritional supplements probably would not be a disqualification).
- Individuals who have a contraindication to MRI/MRS assessment including: (1) heart pacemaker and/or related implants; (2) metal fragment/chip in the eye or other sites; (3) an aneurysm clip in their brain; and (4) metallic inner ear implants.
Key Trial Info
Start Date :
March 1 2004
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 1 2009
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT00151268
Start Date
March 1 2004
End Date
September 1 2009
Last Update
July 29 2016
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