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A Multi-center, Randomized, Active Controlled Clinical Trial to Evaluate the Efficacy and Safety of OTL-203 in Subjects With Mucopolysaccharidosis Type I, Hurler Syndrome (MPS-IH) Compared to Standard of Care With Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)

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Trial Profile

A Multi-center, Randomized, Active Controlled Clinical Trial to Evaluate the Efficacy and Safety of OTL-203 in Subjects With Mucopolysaccharidosis Type I, Hurler Syndrome (MPS-IH) Compared to Standard of Care With Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 10 Jul 2024

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At a glance

  • Drugs OTL 203 (Primary)
  • Indications Mucopolysaccharidosis I
  • Focus Registrational; Therapeutic Use
  • Acronyms HURCULES
  • Sponsors Orchard Therapeutics
  • Most Recent Events

    • 05 Feb 2024 According to an Orchard Therapeutics media release, currently the University of Minnesota is actively recruiting patients with additional trial sites expected to begin recruitment in the coming months. Company plans to activate six sites in the United States and Europe.
    • 05 Feb 2024 According to an Orchard Therapeutics media release, announced the first patient has been randomized at the M Health Fairview Masonic Children's Hospital.
    • 02 Feb 2024 According to an Orchard Therapeutics media release, trial design of this HURCULES trial, will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.
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