AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

ClinicalTrials.gov ID: NCT01414985

Public ClinicalTrials.gov record NCT01414985. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced Jul 21, 2026, 2:22 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA to Children With LINCL With Uncommon Genotypes and/or Moderate to Severe Impairment

Study identification

NCT ID: NCT01414985
Recruitment status: Completed
Study type: Interventional
Phase: Phase 1, Phase 2
Lead sponsor: Weill Medical College of Cornell University; Other
Enrollment: 8 participants

Conditions and interventions

Conditions

Interventions

AAVrh.10CUCLN2 Biological

Biological

Eligibility (public fields only)

Age range: 3 Years to 18 Years
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Apr 14, 2010
Primary completion: Nov 29, 2015
Completion: Feb 7, 2017
Last update posted: Aug 30, 2020

2010 – 2017

United States locations

U.S. sites: 1
U.S. states: 1
U.S. cities: 1

Facility	City	State	ZIP	Site status
Weill Cornell Medical College	New York	New York	10065	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT01414985, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Aug 30, 2020 · Synced Jul 21, 2026

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The complete protocol, eligibility criteria, and contact information for NCT01414985 live on ClinicalTrials.gov.

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