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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: Completed Phase 1 study, sponsored by Weill Medical College of Cornell University.
NCT01161576 is a Phase 1 study that has completed, run by Weill Medical College of Cornell University. The registered enrollment target is 12 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
A clinical rating, 12 point scale which combines assessment of feeding, gait, motor and language to give an overall assessment of various CNS functions.
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 |
What the finished NCT01161576 record still lists
NCT01161576 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
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 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01161576's enrollment target sits among peer trials
12 1852nd of 2000 higher than 90 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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