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Trial record 2 of 3 for:    MGTA-456

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 (IMD-002)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04008849
Recruitment Status : Enrolling by invitation
First Posted : July 5, 2019
Last Update Posted : July 10, 2019
Information provided by (Responsible Party):
Magenta Therapeutics, Inc.

Brief Summary:
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456

Condition or disease Intervention/treatment
Inherited Metabolic Disorders (IMD) Other: Safety and efficacy assessments

Detailed Description:
This is a follow-up study to evaluate the long-term safety and efficacy outcomes of patients with inherited metabolic disorders (IMDs) who received MGTA-456 for HSCT in the core study. MGTA-456 is an expanded CD34+ cell therapy product candidate given after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment. In patients with selected IMDs, transplant is expected to replace defective or missing protein, and preserve neurodevelopment. Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) enrolled in the core study will be eligible to participate in this follow-up evaluation.

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Study Type : Observational
Estimated Enrollment : 12 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Follow-Up Study to Evaluate the Safety and Clinical Outcomes of Patients With Non-Malignant Disease Who Have Undergone Hematopoietic Stem Cell Transplantation With MGTA-456
Actual Study Start Date : May 21, 2019
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : May 2025

Group/Cohort Intervention/treatment
Subjects treated with MGTA-456
MGTA-456 is an investigational expanded CD34+ cell therapy
Other: Safety and efficacy assessments
Long term safety and clinical outcomes

Primary Outcome Measures :
  1. Incidence of related adverse events [ Time Frame: 5 years ]
  2. Incidence of serious adverse events [ Time Frame: 5 years ]
  3. Incidence of late hematological graft failure [ Time Frame: 5 years ]
  4. Incidence of chronic graft versus host disease [ Time Frame: 5 years ]
  5. Overall survival [ Time Frame: 5 years ]
  6. Event-free survival [ Time Frame: 5 years ]
  7. Change in cALD Neurologic Function Score over time [ Time Frame: 5 years ]
  8. Proportion of subjects without gadolinium enhancement on MRI over time [ Time Frame: 5 years ]
  9. Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients [ Time Frame: 5 years ]
  10. Very long chain fatty acid blood level (ug/mL) in cALD patients [ Time Frame: 5 years ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456

Inclusion Criteria:

  • An Institutional Review Board (IRB)/Independent Ethics Committee-approved written informed consent form must be signed and dated by the patient or legal guardian. Study assents will also be prepared for children and adolescents to review when applicable.
  • Patient completed an IMD study in the MGTA-456 program and was administered MGTA-456 for HSCT.

Exclusion Criteria:

•Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.

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 identifier (NCT number): NCT04008849

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United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Magenta Therapeutics, Inc.

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Responsible Party: Magenta Therapeutics, Inc. Identifier: NCT04008849    
Other Study ID Numbers: IMD-002
First Posted: July 5, 2019    Key Record Dates
Last Update Posted: July 10, 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.:
Inherited Metabolic Disorders
Hematopoietic Stem Cell Transplant
Umbilical Cord Blood Transplant
Hurler Syndrome
Cerebral Adrenoleukodystrophy
Globoid Cell Leukodystrophy
Metachromatic Leukodystrophy
Additional relevant MeSH terms:
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Metabolic Diseases