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Clinical trial

GAIN-CTNNB1: A Phase I/II Open-Label Trial To Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intracerebroventricular Administration of an AAV9 Based Gene Replacement Therapy in Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome

NCT07270549 is registered in ClinicalTrials.gov with CTNNB1 Foundation as lead sponsor.

NCT NCT07270549
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Registry record

As filed in ClinicalTrials.gov.

Title
GAIN-CTNNB1: A Phase I/II Open-Label Trial To Evaluate the Safety, Tolerability, and Preliminary Efficacy of Intracerebroventricular Administration of an AAV9 Based Gene Replacement Therapy in Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome
Lead sponsor
CTNNB1 Foundation
Phase
Phase 1/2
Overall status
Recruiting
Start date
November 1, 2025
Completion date
December 31, 2032
Enrollment
12 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

ctnnb1 neurodevelopmental syndrome