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

Trial Profile

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

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 07 Jan 2019

At a glance

  • Drugs ABO 201 (Primary)
  • Indications Neuronal ceroid lipofuscinosis
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Most Recent Events

    • 07 Jan 2019 According to an Amicus Therapeutics media release, the company expects to complete enrollment in the study in 2019.
    • 03 Jan 2019 According to an Amicus Therapeutics media release, the company plans to present clinical data from this study, including interim data, at future scientific congresses and other relevant venues.
    • 03 Jan 2019 According to an Amicus Therapeutics media release, the initial patient completed a one-month observation period following dosing with no serious adverse events reported to date.
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