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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

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Trial Profile

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

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 10 Mar 2023

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At a glance

  • Drugs Sesiclenegene cosaparvovec (Primary)
  • Indications Neuronal ceroid lipofuscinosis
  • Focus Adverse reactions; First in man
  • Sponsors Amicus Therapeutics

Most Recent Events

  • 07 Mar 2023 Primary and secondary endpoints amended. Study frame changed for primary endpoint from 24 months to Screening (Day -30 up to -2) up to Month 24.
  • 21 Jan 2022 Status changed from active, no longer recruiting to completed.
  • 10 May 2021 According to an Amicus Therapeutics media release, the company plans to advance manufacturing activities and regulatory discussions for the CLN6 Batten disease gene therapy program to enable dosing of additional patients with GMP clinical grade material.

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