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

• Status: Completed
• Trial end date: 2021-01-15
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Magenta Therapeutics, Inc.

Criteria

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