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

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)

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.

Study Overview

• Status: Completed
• Conditions: Inherited Metabolic Disorders (IMD)
• Intervention / Treatment: Drug: MGTA-456

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.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 15 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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 Names: HSC835

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Engraftment	Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.	42 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Infusion Toxicities	Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration	48 hours

Collaborators and Investigators

• Sponsor: Magenta Therapeutics, Inc.
• Investigators: Study Director: Magenta Study Coordinator, Magenta Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): February 9, 2018
• Primary Completion (Actual): February 10, 2020
• Study Completion (Actual): January 15, 2021

Study Registration Dates

• First Submitted: January 16, 2018
• First Submitted That Met QC Criteria: January 16, 2018
• First Posted (Actual): January 23, 2018

Study Record Updates

• Last Update Posted (Actual): November 3, 2021
• Last Update Submitted That Met QC Criteria: October 27, 2021
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Keywords: hematopoietic stem cells; umbilical cord blood; Krabbe disease; hematopoietic stem cell transplant; bone marrow transplant; metachromatic leukodystrophy; MGTA-456; globoid cell leukodystrophy; inherited metabolic disorders; cerebral adrenoleukodystrophy; Hurler syndrome; umbilical cord blood transplant; myeloablative conditioning regimen; mucopolysaccharidosis-1H
• Additional Relevant MeSH Terms: Pathologic Processes; Disease; Metabolic Diseases
• Other Study ID Numbers: IMD-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No