Working… Menu
Trial record 1 of 1 for:    NCT03406962
Previous Study | Return to List | Next Study

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. Read our disclaimer for details. Identifier: NCT03406962
Recruitment Status : Active, not recruiting
First Posted : January 23, 2018
Last Update Posted : June 24, 2020
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 in patients with IMD after receiving myeloablative conditioning and HSCT. MGTA-456 is an expanded CD34+ cell therapy product candidate given to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein and preserve neurodevelopment. Since MGTA-456 offers increased numbers of HSCs over standard umbilical cord blood, 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) are eligible for this study.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
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
Actual Primary Completion Date : February 10, 2020
Estimated Study Completion Date : January 2021

Arm Intervention/treatment
Experimental: MGTA-456
MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age < 2.5 years with Hurler syndrome, age 2-17 years with cerebral adrenoleukodystrophy (cALD), age < 16 years with metachromatic leukodystrophy (MLD) and age ≤ 10 years with globoid cell leukodystrophy (GLD) (also referred to as Krabbe)
  • Cord blood grafts require genetic testing and/or demonstration of enzyme activity for patients with Hurler syndrome, MLD or GLD and are tested for very long chain fatty acids (VLCFA) to confirm there is no evidence of VLCFA consistent with ALD
  • 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

Layout table for location information
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Magenta Therapeutics, Inc.
Layout table for investigator information
Study Director: Magenta Study Coordinator Magenta Therapeutics
Layout table for additonal information
Responsible Party: Magenta Therapeutics, Inc. Identifier: NCT03406962    
Other Study ID Numbers: IMD-001
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: June 24, 2020
Last Verified: June 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
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:
Layout table for MeSH terms
Metabolic Diseases
Pathologic Processes