A multicentre pivotal clinical trial to investigate LYS-SAF302 for the treatment of Sanfilippo Type A

Trial Profile

A multicentre pivotal clinical trial to investigate LYS-SAF302 for the treatment of Sanfilippo Type A

Planning
Phase of Trial: Phase II/III

Latest Information Update: 16 Apr 2018

At a glance

  • Drugs SAF 302 (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Registrational; Therapeutic Use
  • Sponsors LYSOGENE
  • Most Recent Events

    • 16 Apr 2018 According to a Lysogene media release, this trial is expected to start in the second half of 2018.
    • 06 Feb 2018 According to a LYSOGENE media release, company plans submission of Marketing Authorization Application following completion of this trial.
    • 06 Feb 2018 According to a Lysogene media release, Paediatric Committee (PDCO) of the European Medicines Agency (EMA) adopted a positive opinion regarding the Paediatric Investigation Plan (PIP) for LYS-SAF302 in patients with mucopolysaccharidosis type IIIA.
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