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NCT00151216 · ClinicalTrials.gov registry record · Phase 1
Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis
A Phase 1 study, sponsored by Weill Medical College of Cornell University.
- Completed
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT00151216: Completed Phase 1 study, sponsored by Weill Medical College of Cornell University.
NCT00151216 is a Phase 1 study that has completed, run by Weill Medical College of Cornell University. The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00151216, a Phase 1 study, has completed, sponsored by Weill Medical College of Cornell University.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
Study Summary
The aim of this study is to treat the signs and symptoms of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal inherited disease in the brain. This will be accomplished by using delivery of a gene (method called gene transfer) to administer to the brain an experimental drug called AAV2CUhCLN2, a gene transfer vector.
Primary Outcome
The neurological examination will be carried out using a standard format with input from the parents/legal guardians as relevant. The clinical rating scale, referred to as a "CNS disability scale" is made up of individual sum of 4 point scales for each of 3 activities; the ratings for each activity are summed, giving a "CNS disability score". Each of the activities are rated 0 to 3, where 0 is the most severe form of the disease. This scale, developed by Steinfeld et al, originally included the
Interventions
- BIOLOGICAL AAV2CUhCLN2 (3x10^12 particle units)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2004-06 |
| Est. Completion | 2019-06 |
| Phase | Phase 1 |
What the finished NCT00151216 record still lists
NCT00151216 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 0 conditions, and to 1 intervention - of which AAV2CUhCLN2 (3x10^12 particle units) is the first listed.
NCT00151216 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00151216 about?
NCT00151216 is a clinical study titled "Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis". The aim of this study is to treat the signs and symptoms of late infantile neuronal ceroid lipofuscinosis (LINCL), a fatal inherited disease in the brain. This will be accomplished by using delivery of a gene (method called gene transfer) to administer to the brain an experimental drug called AAV2CU...
What is the current status of trial NCT00151216?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2004-06. Estimated completion is 2019-06.
What interventions are being tested in trial NCT00151216?
The interventions under investigation include: AAV2CUhCLN2 (3x10^12 particle units) (BIOLOGICAL).
Who is sponsoring clinical trial NCT00151216?
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 NCT00151216's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 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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