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NCT01161576 · ClinicalTrials.gov registry record · Phase 1
Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)
A Phase 1 study, sponsored by Weill Medical College of Cornell University.
- Completed
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
NCT01161576 is a Phase 1 study that has completed, run by Weill Medical College of Cornell University. The registered enrollment target is 12 participants.
The verdict
NCT01161576, a Phase 1 study, has completed, sponsored by Weill Medical College of Cornell University.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
Study Summary
This is a proposed follow up study on the investigators previous gene transfer human clinical trial entitled "Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis" (Weill Cornell IRB# 0401007010). As in the previous study, the investigators propose to administer a biologic by direct gene transfer into the brain and assess its safety on children with a fatal genetic disease of the central nervous system (CNS). The disease is Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL, a form of Batten disease). This will be accomplished by using delivery of a gene (method called gene transfer) to administer to the brain an experimental drug called AAVRh.10CUhCLN2, a gene transfer vector.
Interventions
- BIOLOGICAL AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies
- BIOLOGICAL AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2010-08-19 |
| Est. Completion | 2020-12-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01161576
The ClinicalTrials.gov registry entry for NCT01161576 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Weill Medical College of Cornell University, which has 701 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies is the first listed.
NCT01161576 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01161576 about?
NCT01161576 is a clinical study titled "Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)". This is a proposed follow up study on the investigators previous gene transfer human clinical trial entitled "Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinos...
What is the current status of trial NCT01161576?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2010-08-19. Estimated completion is 2020-12-31.
What interventions are being tested in trial NCT01161576?
The interventions under investigation include: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies (BIOLOGICAL), AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies (BIOLOGICAL).
Who is sponsoring clinical trial NCT01161576?
This trial is sponsored by Weill Medical College of Cornell University, which has 701 total clinical trials registered on ClinicalTrials.gov.
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