MedTrace Logo

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

NCT00151216 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2020-07-24

No results posted yet for this 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.

Conditions

  • Batten Disease
  • Late Infantile Neuronal Ceroid Lipofuscinosis

Interventions

BIOLOGICAL

AAV2CUhCLN2 (3x10^12 particle units)

gene transfer; one-time administration via neuro surgery procedure

Sponsors & Collaborators

  • Nathan's Battle Foundation

    collaborator OTHER

  • Weill Medical College of Cornell University

    lead OTHER

Principal Investigators

  • Ronald G. Crystal, MD · Weill Medical College of Cornell University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-06-30
Primary Completion
2019-06-30
Completion
2019-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00151216 on ClinicalTrials.gov