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: 31 Aug 2023

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At a glance

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

Most Recent Events

25 Aug 2023 Planned End Date changed from 1 Sep 2023 to 30 Sep 2024.
25 Aug 2023 Planned primary completion date changed from 1 Jun 2023 to 30 Sep 2024.
12 Feb 2021 Preliminary results (n=4, Data cut off: 11 Aug 2020) presented at 17th Annual WORLDSymposium 2021

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