Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9
Active, no longer recruiting
Phase of Trial: Phase I/II
Latest Information Update: 28 Jan 2019
At a glance
- Drugs ScAVV9 CB CLN6 (Primary)
- Indications Neuronal ceroid lipofuscinosis
- Focus Adverse reactions; First in man; Pharmacogenomic
- 07 Jan 2019 According to an Amicus Therapeutics media release, the company expects to report 2-year data from the study in mid-2019.
- 07 Jan 2019 Status changed from recruiting to active, no longer recruiting, according to an Amicus Therapeutics media release.
- 27 Sep 2018 New trial record