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

Inherited Metabolic Disorders (IMD) Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03406962
• Other study ID #: IMD-001
• Status: Completed
• Phase: Phase 2
• Start date: February 9, 2018
• Est. completion date: January 15, 2021

Study information

• Verified date: June 2020
• Source: Magenta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: January 15, 2021
• Est. primary completion date: February 10, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Disease
• Inherited Metabolic Disorders (IMD)
• Metabolic Diseases

Intervention

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

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	University of Minnesota	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Magenta Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Engraftment	Engraftment is defined as achieving an absolute neutrophil count (ANC) =0.5 × 10?/L for 3 consecutive days.	42 days
Secondary	Number of Participants With Infusion Toxicities	Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration	48 hours