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

Phase 2

Completed

• Conditions: Inherited Metabolic Disorders (IMD)
• Interventions: Drug: MGTA-456

• Registration Number: NCT03406962
• Lead Sponsor: 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.

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 Timeline

• Study First Submitted: 2018-01-16
• Study First Submitted QC: 2018-01-16
• Study First Posted: 2018-01-23
• Study Start: 2018-02-09
• Primary Completion: 2020-02-10
• Status Verified: 2020-06
• Study Completion: 2021-01-15
• Results First Submitted: 2021-09-29
• Results First Submitted QC: 2021-09-29
• Last Update Submitted: 2021-10-27
• Results First Posted: 2021-10-28
• Last Update Posted: 2021-11-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MGTA-456	MGTA-456	MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Infusion Toxicities	48 hours	Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration

Trial Locations

Locations (2)

University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States