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Study for the Treatment for CLN7 Disease

NCT04737460 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2025-12-31

No results posted yet for this study

Summary

This is a phase 1 open-label, single-administration of gene therapy agent AAV9/CLN7, administered intrathecally into the lumbar spinal cord region of pediatric patients with CLN7 Batten disease.

This study consists of a one-time injection of AAV9/CLN7. There are two Cohorts with a low dose and a high dose.

The primary objective for this clinical study is to evaluate safety. The secondary objective is to determine the efficacy of AAV9/CLN7.

The secondary outcome measures include motor, cognition and intelligence assessments.

The exploratory outcome measures include visual impairment assessment, cognitive evaluations, Brain magnetic resonance imaging (MRI), electroencephalogram (EEG), electrocardiogram (ECG) and echocardiogram (ECHO).

Conditions

  • CLN7

Interventions

GENETIC

AAV9/CLN7

Enrollees will receive gene therapy via a viral vector

Sponsors & Collaborators

  • Benjamin Greenberg

    lead OTHER

Principal Investigators

  • Benjamin Greenberg, MD · University of Texas Southwestern Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-05-04
Primary Completion
2028-02-01
Completion
2029-02-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT04737460 on ClinicalTrials.gov