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

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

Eligibility

Age range

0+ years

Sex

All

Healthy volunteers

No

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

Location