Batten Disease, Batten's Disease, CLN6, Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN6
Conditions
Keywords
CLN6, CLN6 Batten, Batten, Batten's, Neuronal Ceroid Lipofuscinosis
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
Interventions
DRUGscAAV9.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
Sponsors
The Charlotte and Gwenyth Gray Foundation
University of California, San Diego
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Months to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0 | 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
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Hamburg Rating Scale Score | 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. |
| 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 | 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. |
Countries
United States
Contacts
CONTACTTiffany M Sepp
Outcome results
None listed