Gene Therapy Trial for CLN6 Batten Disease

NCT07582484 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CGGF-CLN6-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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

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

Keywords

CLN6; CLN6 Batten; Batten; Batten's; Neuronal Ceroid Lipofuscinosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0

  Treatment-emergent adverse events, including serious adverse events, hospitalizations, deaths, clinically significant laboratory abnormalities, and other clinically significant safety findings will be recorded from informed consent through Month 24. Adverse events will be assessed for intensity using CTCAE version 5.0 and for relationship to scAAV9.CB.CLN6. The number and percentage of participants with treatment-emergent adverse events will be summarized.

  From informed consent through Month 24

Secondary Outcomes (2)

• Change From Baseline in Hamburg Rating Scale Score

  Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24

• Change From Baseline in Weill-Cornell Late Infantile Neuronal Ceroid Lipofuscinosis Scale Score

  Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24

Study Arms (1)

Treatment group

EXPERIMENTAL

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

Interventions

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

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

Treatment group

Eligibility Criteria

• Age: 4 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of CLN6
• At least 4 months old

You may not qualify if:

• Presence of another inherited neurologic disease
• Prior stem cell transplantation
• Prior gene transfer, gene editing, or viral vector therapy

Sponsors & Collaborators

• University of California, San Diego: collaborator
• The Charlotte and Gwenyth Gray Foundation: lead

Study Sites (1)

University of California, San Diego - Rady Children's

La Jolla, California, 92093, United States

Location

MeSH Terms

Conditions

Neuronal Ceroid-Lipofuscinoses

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Tiffany M Sepp

CONTACT

617-710-0770; tiffany@vanguardclinical.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 20, 2026
• First Posted: May 13, 2026
• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): August 1, 2028
• Last Updated: May 14, 2026

Record last verified: 2026-05

Locations

US (1)