MedTrace Logo

Gene Therapy Trial for CLN6 Batten Disease

NCT07582484 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-14

No results posted yet for this study

Summary

The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms.

Participants will:

Receive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study

Conditions

  • CLN6
  • Batten Disease
  • Batten's Disease
  • Neuronal Ceroid Lipofuscinosis CLN6
  • Neuronal Ceroid Lipofuscinosis

Interventions

DRUG

scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)

self-complementary adeno-associated viral vector, serotype 9 (scAAV9), which contains the human CLN6 gene under the control of a hybrid CMV/CB promoter

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-08-31
Primary Completion
2027-08-31
Completion
2028-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

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 NCT07582484 on ClinicalTrials.gov