Gene Therapy Trial for CLN6 Batten Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes).
- Who it may be relevant to
- Registry conditions: CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6. Basic parameters: from 4 months · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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
Overview
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
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
Primary outcome measures
- Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0 [Time frame: From informed consent through Month 24]
Secondary outcome measures (2)
- 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]
- 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]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- University of California, San Diego - Rady Children's — La Jolla
Identifiers
NCT: NCT07582484 · CGGF-CLN6-001