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

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ClinicalTrials.gov Identifier: NCT03406962
Recruitment Status : Recruiting
First Posted : January 23, 2018
Last Update Posted : July 15, 2019
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Inherited Metabolic Disorders (IMD) Drug: MGTA-456 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : February 9, 2018
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : January 2021


Arm Intervention/treatment
Experimental: MGTA-456
MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.
Drug: MGTA-456
Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.
Other Name: HSC835




Primary Outcome Measures :
  1. Incidence of engraftment [ Time Frame: 42 days ]
    Engraftment is defined as achieving an absolute neutrophil count (ANC) ≥0.5 × 10⁹/L for 3 consecutive days.


Secondary Outcome Measures :
  1. Incidence of infusion toxicities [ Time Frame: 48 hours ]
    Incidence of MGTA-456-related adverse events (AEs) that limit MGTA-456 administration



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Ages Eligible for Study:   6 Months to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosed with Hurler syndrome, metachromatic leukodystrophy (MLD), globoid cell leukodystrophy (GLD or Krabbe) or cerebral adrenoleukodystrophy (cALD)
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03406962


Contacts
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Contact: Magenta Clinical Trials 857-242-0170 clinicaltrials@magentatx.com

Locations
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United States, Georgia
Children's Healthcare of Atlanta - Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Lakshmanan Krishnamurti, MD         
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Paul J Orchard, MD         
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27710
Contact: Vinod K Prasad, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Pooja Khandelwal, MD         
Sponsors and Collaborators
Magenta Therapeutics, Inc.
Investigators
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Study Director: Magenta Study Coordinator Magenta Therapeutics

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Responsible Party: Magenta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03406962     History of Changes
Other Study ID Numbers: IMD-001
First Posted: January 23, 2018    Key Record Dates
Last Update Posted: July 15, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Magenta Therapeutics, Inc.:
MGTA-456
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
myeloablative conditioning regimen
bone marrow transplant
mucopolysaccharidosis-1H
Additional relevant MeSH terms:
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Metabolic Diseases
Disease
Pathologic Processes