# Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

> **NCT00151216** · PHASE1 · COMPLETED · sponsor: **Weill Medical College of Cornell University** · enrollment: 10 (actual)

## Conditions studied

- Batten Disease
- Late Infantile Neuronal Ceroid Lipofuscinosis

## Interventions

- **BIOLOGICAL:** AAV2CUhCLN2 (3x10^12 particle units)

## Key facts

- **NCT ID:** NCT00151216
- **Lead sponsor:** Weill Medical College of Cornell University
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2004-06
- **Primary completion:** 2019-06
- **Final completion:** 2019-06
- **Target enrollment:** 10 (ACTUAL)
- **Last updated:** 2020-07-24

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00151216

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00151216, "Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis". Retrieved via AI Analytics 2026-09-16 from https://api.ai-analytics.org/clinical/NCT00151216. Licensed CC0.

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