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Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA

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Trial Profile

Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA

Status: Active, no longer recruiting
Phase of Trial: Phase II/III

Latest Information Update: 13 Sep 2023

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At a glance

  • Drugs Olenasufligene relduparvovec (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Proof of concept; Registrational; Therapeutic Use
  • Acronyms AAVance
  • Sponsors LYSOGENE

    • Most Recent Events

    • 11 Sep 2023 According to European Clinical Trials Database, study has been suspended in Germany
    • 29 Nov 2022 According to a Lysogene media release, the company has initiated fruitful discussions with regulatory authorities in the European Union in late Q3 2022.
    • 29 Nov 2022 According to a Lysogene media release, the company will complete full analysis and evaluation of the data from the main cohort of AAVance and work with investigators on the future presentation and publication of the results.
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