Status:
COMPLETED
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
Lead Sponsor:
Magenta Therapeutics, Inc.
Conditions:
Inherited Metabolic Disorders (IMD)
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE2
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 prod...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
February 9 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 15 2021
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT03406962
Start Date
February 9 2018
End Date
January 15 2021
Last Update
November 3 2021
Active Locations (2)
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1
University of Minnesota
Minneapolis, Minnesota, United States, 55455
2
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229