Gene Therapy Trial for CLN6 Batten Disease
Public ClinicalTrials.gov record NCT07582484. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Study identification
- NCT ID
- NCT07582484
- Recruitment status
- Not yet recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- The Charlotte and Gwenyth Gray Foundation
- Other
- Enrollment
- 12 participants
Conditions and interventions
Conditions
Interventions
- scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) Drug
Drug
Eligibility (public fields only)
- Age range
- 4 Months and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 31, 2026
- Primary completion
- Jul 31, 2027
- Completion
- Jul 31, 2028
- Last update posted
- May 13, 2026
2026 – 2028
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, San Diego - Rady Children's | La Jolla | California | 92093 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07582484, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 13, 2026 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07582484 live on ClinicalTrials.gov.