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

March 10, 2021 updated by: Magenta Therapeutics, Inc.

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

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 16 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Subjects with an inherited metabolic disorder (IMD) that were treated with MGTA-456

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Magenta Therapeutics, Inc.

Investigators

  • Principal Investigator: Paul J Orchard, MD, University of Minnesota

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 21, 2019

Primary Completion (Actual)

October 8, 2020

Study Completion (Actual)

October 8, 2020

Study Registration Dates

First Submitted

May 23, 2019

First Submitted That Met QC Criteria

July 3, 2019

First Posted (Actual)

July 5, 2019

Study Record Updates

Last Update Posted (Actual)

March 15, 2021

Last Update Submitted That Met QC Criteria

March 10, 2021

Last Verified

March 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IMD-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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