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A Phase 2, Single-arm, Open-label Study to Evaluate the Safety and Efficacy of MGTA-456 in Patients With Inherited Metabolic Disorders (IMD) Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Trial Profile

A Phase 2, Single-arm, Open-label Study to Evaluate the Safety and Efficacy of MGTA-456 in Patients With Inherited Metabolic Disorders (IMD) Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Status: Discontinued
Phase of Trial: Phase II

Latest Information Update: 08 Nov 2021

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At a glance

  • Drugs Spanlecortemlocel (Primary) ; Antithymocyte globulin; Busulfan; Ciclosporin; Cyclophosphamide; Fludarabine; Methylprednisolone
  • Indications Adrenoleucodystrophy; Globoid cell leukodystrophy; Metachromatic leukodystrophy; Mucopolysaccharidosis I; Transplant rejection
  • Focus Therapeutic Use
  • Sponsors Magenta Therapeutics
  • Most Recent Events

    • 22 Jun 2020 Planned end date (follow up) changed from 1 Apr 2022 to 1 Jan 2021.
    • 11 Jun 2020 According to an Magenta Therapeutics media release, the company has made a strategic decision to discontinue enrollment in this trial.This decision was the result of several factors, enrollment challenges common to rare disease populations, particularly during the COVID-19 pandemic, a growing understanding in the field of the current challenges of allogeneic stem cell transplant in patients with non-malignant diseases, such as IMDs and feedback from the FDA on endpoints and trial design.
    • 11 Jun 2020 Status changed from recruiting to discontinued, according to an Magenta Therapeutics media release.
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