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
Diagnosis of CLN6
At least 4 months old
Presence of another inherited neurologic disease
Prior stem cell transplantation
Prior gene transfer, gene editing, or viral vector therapy
Single group
scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
Drugself-complementary adeno-associated viral vector, serotype 9 (scAAV9), which contains the human CLN6 gene under the control of a hybrid CMV/CB promoter
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.
Change From Baseline in Hamburg Rating Scale Score
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.
Change From Baseline in Weill-Cornell Late Infantile Neuronal Ceroid Lipofuscinosis Scale Score
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.
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The Charlotte and Gwenyth Gray Foundation
Lead sponsor
University of California, San Diego
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
