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Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9

Trial Profile

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
  • Most Recent Events

    • 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
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