Back to resultsMGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
Primary Purpose
Inherited Metabolic Disorders (IMD)
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
MGTA-456
Sponsored by
About this trial
This is an interventional treatment trial for Inherited Metabolic Disorders (IMD) focused on measuring inherited metabolic disorders, cerebral adrenoleukodystrophy, Hurler syndrome, globoid cell leukodystrophy, Krabbe disease, metachromatic leukodystrophy, hematopoietic stem cells, hematopoietic stem cell transplant, umbilical cord blood, umbilical cord blood transplant, MGTA-456, myeloablative conditioning regimen, bone marrow transplant, mucopolysaccharidosis-1H
Eligibility 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
Sites / Locations
- University of Minnesota
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
MGTA-456
Arm Description
MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Outcomes
Primary Outcome Measures
Number of Participants With Engraftment
Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.
Secondary Outcome Measures
Number of Participants With Infusion Toxicities
Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration
Full Information
NCT ID
NCT03406962
First Posted
January 16, 2018
Last Updated
October 27, 2021
Sponsor
Magenta Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03406962
Brief Title
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
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)
Study Type
Interventional
2. Study Status
Record Verification Date
June 2020
Overall Recruitment Status
Completed
Study Start Date
February 9, 2018 (Actual)
Primary Completion Date
February 10, 2020 (Actual)
Study Completion Date
January 15, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Magenta Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Inherited Metabolic Disorders (IMD)
Keywords
inherited metabolic disorders, cerebral adrenoleukodystrophy, Hurler syndrome, globoid cell leukodystrophy, Krabbe disease, metachromatic leukodystrophy, hematopoietic stem cells, hematopoietic stem cell transplant, umbilical cord blood, umbilical cord blood transplant, MGTA-456, myeloablative conditioning regimen, bone marrow transplant, mucopolysaccharidosis-1H
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
MGTA-456
Arm Type
Experimental
Arm Description
MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Intervention Type
Drug
Intervention Name(s)
MGTA-456
Other Intervention Name(s)
HSC835
Intervention Description
Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.
Primary Outcome Measure Information:
Title
Number of Participants With Engraftment
Description
Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.
Time Frame
42 days
Secondary Outcome Measure Information:
Title
Number of Participants With Infusion Toxicities
Description
Incidence of treatment-emergent adverse events (AEs) within 48 hours after MGTA-456 administration
Time Frame
48 hours
10. Eligibility
Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Magenta Study Coordinator
Organizational Affiliation
Magenta Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
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