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

NCT ID: NCT03406962

Last Updated: 2021-11-03

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-09

Study Completion Date

2021-01-15

Brief Summary

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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

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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.

Conditions

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Inherited Metabolic Disorders (IMD)

Keywords

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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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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MGTA-456

MGTA-456 is an expanded CD34+ cell therapy investigational product used in replacement of single umbilical cord blood transplantation.

Group Type EXPERIMENTAL

MGTA-456

Intervention Type DRUG

Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.

Interventions

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MGTA-456

Hematopoietic stem cell transplantation will be done with the cell therapy product MGTA-456.

Intervention Type DRUG

Other Intervention Names

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HSC835

Eligibility Criteria

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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
Minimum Eligible Age

0 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Magenta Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Magenta Study Coordinator

Role: STUDY_DIRECTOR

Magenta Therapeutics

Locations

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University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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IMD-001

Identifier Type: -

Identifier Source: org_study_id