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NCT Number: NCT07582484

Gene Therapy Trial for CLN6 Batten Disease

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

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Key information

Conditions

CLN6 Batten Disease Batten's Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuronal Ceroid Lipofuscinosis Neuronal Ceroid Lipofuscinosis CLN6 Neuronal Ceroid-Lipofuscinoses Nutritional and Metabolic Diseases

Age range

4 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of California, San Diego - Rady Children's

La Jolla, California, 92093, United States

Location contact

Olivia Kim-McManus, MD

CONTACT

[email protected]

858-576-1700 ext. x224550

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of CLN6
  • At least 4 months old

Exclusion criteria

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

Treatment and study plan

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

Primary outcomes

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

    Time frame: From informed consent through Month 24

    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.

Secondary outcomes

  1. Change From Baseline in Hamburg Rating Scale Score

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

    The Hamburg Rating Scale will be used to assess disease status and function in participants with CLN6 disease. Change from baseline in Hamburg Rating Scale score will be summarized at scheduled post-baseline visits.

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

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

    The Weill-Cornell Late Infantile Neuronal Ceroid Lipofuscinosis Scale will be used to assess disease status and function in participants with CLN6 disease. Change from baseline in Weill-Cornell Scale score will be summarized at scheduled post-baseline visits.

Study contacts

Contact information is provided by the study sponsor or research team.

Tiffany M Sepp

CONTACT

[email protected]

617-710-0770

Sponsors and collaborators

Lead sponsor

The Charlotte and Gwenyth Gray Foundation

Other

Collaborators

  • University of California, San Diego

Registry information

Official study title

Phase 1/2b Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease), Delivering the CLN6 Gene by Self-Complementary AAV9

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
May 13, 2026
Registry last updated
May 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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