MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03406962
Recruitment Status : Recruiting
First Posted : January 23, 2018
Last Update Posted : April 6, 2018
Information provided by (Responsible Party):
Magenta Therapeutics, Inc.

Brief Summary:
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.

Condition or disease Intervention/treatment Phase
Inherited Metabolic Disorders (IMD) Drug: MGTA-456 Phase 2

Detailed Description:
This phase 2 study is designed to evaluate the safety and efficacy of MGTA-456 after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment with replacement of the specific protein product missing or defective in the patient with an IMD. The study aims to enhance the efficacy of umbilical cord blood transplantation (UCBT), to preserve neurodevelopment in patients with selected IMDs. Since MGTA-456 offers increased numbers of HSCs over standard UCB, it is expected to reduce the risks of prolonged neutropenia and thrombocytopenia and graft failure, and potentially transplant-related mortality (TRM). Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) (also referred to as Krabbe disease) could be eligible for this study.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : February 9, 2018
Estimated Primary Completion Date : May 2019
Estimated Study Completion Date : April 2020

Arm Intervention/treatment
Experimental: MGTA-456
MGTA-456 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation.
Drug: MGTA-456
Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.
Other Name: HSC835

Primary Outcome Measures :
  1. Incidence of engraftment [ Time Frame: 42 days ]
    Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.

Secondary Outcome Measures :
  1. Incidence of infusion toxicities [ Time Frame: 48 hours ]
    Incidence of MGTA-456-related adverse events (AEs) that limit MGTA-456 administration

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosed with Hurler syndrome, metachromatic leukodystrophy (MLD), globoid cell leukodystrophy (GLD or Krabbe) or cerebral adrenoleukodystrophy (cALD)
  • Adequate organ function
  • Availability of eligible donor material

Exclusion Criteria:

  • Availability of a matched-related donor who is not a carrier of the same genetic defect
  • Active infection at screening
  • Prior myeloablative conditioning
  • History of human immunodeficiency virus (HIV) infection

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03406962

Contact: Magenta Clinical Trials 857-242-0170

United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Paul J Orchard, MD    612-626-2313   
Sponsors and Collaborators
Magenta Therapeutics, Inc.
Study Director: Magenta Study Director Magenta Therapeutics

Responsible Party: Magenta Therapeutics, Inc. Identifier: NCT03406962     History of Changes
Other Study ID Numbers: IMD-001
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: April 6, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Magenta Therapeutics, Inc.:
inherited metabolic disorders
cerebral adrenoleukodystrophy
Hurler syndrome
globoid cell leukodystrophy
Krabbe disease
metachromatic leukodystrophy
hematopoietic stem cells
hematopoietic stem cell transplant
umbilical cord blood
umbilical cord blood transplant
myeloablative conditioning regimen
bone marrow transplant

Additional relevant MeSH terms:
Metabolic Diseases
Pathologic Processes