Trial Profile

Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 19 Jun 2026

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At a glance

Drugs AAV9 CLN3 (Primary)
Indications Neuronal ceroid lipofuscinosis
Focus Adverse reactions; First in man; Therapeutic Use
Sponsors Alcyone Therapeutics; Amicus Therapeutics; Neela Therapeutics

Most Recent Events

07 Aug 2025 Planned End Date changed from 30 Sep 2024 to 30 Sep 2029.
07 Aug 2025 Planned primary completion date changed from 30 Sep 2024 to 30 Sep 2029.
25 Aug 2023 Planned End Date changed from 1 Sep 2023 to 30 Sep 2024.

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