Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9
Phase of Trial: Phase I/II
Latest Information Update: 07 Oct 2019
Price : $35 *
At a glance
- Drugs ABO 201 (Primary)
- Indications Neuronal ceroid lipofuscinosis
- Focus Adverse reactions; First in man; Therapeutic Use
- Sponsors Amicus Therapeutics
- 02 Oct 2019 Planned End Date changed from 1 Dec 2022 to 1 Sep 2023.
- 02 Oct 2019 Planned primary completion date changed from 1 Dec 2022 to 1 Jun 2023.
- 28 Feb 2019 According to an Amicus Therapeutics media release, further patient dosing is expected in the coming months.