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

Sponsor

Magenta Therapeutics, Inc. (Industry)

Overall Status

Terminated

CT.gov ID

NCT04008849

Collaborator

(none)

Enrollment

Location

16.6

Actual Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

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	Phase
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.

Study Design

Study Type:

Observational

Actual Enrollment :

3 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

Actual Primary Completion Date :

Oct 8, 2020

Actual Study Completion Date :

Oct 8, 2020

Arms and Interventions

Arm	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

Arm

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

Outcome Measures

Primary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 16 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.