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

Magenta Therapeutics, Inc.8 enrolled

Overview

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.

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

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Inherited Metabolic Disorders (IMD)

Interventions

MGTA-456DRUG

Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.

Eligibility

Sex: ALLMin age: 0 YearsMax age: 17 Years

Medical Language ↔ Plain English

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

Locations (2)

University of Minnesota

Minneapolis, Minnesota, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

Number of Participants With Engraftment

Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.

Time frame: 42 days

Secondary Outcomes

Number of Participants With Infusion Toxicities

Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration

Time frame: 48 hours

Data from ClinicalTrials.gov

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