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
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Inherited Metabolic Disorders (IMD)
- Sponsor
- Magenta Therapeutics, Inc.
- Enrollment
- 3
- Locations
- 1
- Primary Endpoint
- Incidence of related adverse events
- Status
- Terminated
- Last Updated
- 5 years ago
Overview
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
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.
Investigators
Eligibility Criteria
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- •Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.
Outcomes
Primary Outcomes
Incidence of related adverse events
Time Frame: 2 years
Incidence of chronic graft versus host disease
Time Frame: 2 years
Event-free survival
Time Frame: 2 years
Very long chain fatty acid blood level (ug/mL) in cALD patients
Time Frame: 2 years
Incidence of late hematological graft failure
Time Frame: 2 years
Proportion of subjects without gadolinium enhancement on MRI over time
Time Frame: 2 years
Alpha-iduronidase blood enzyme level (nmol/hr/mg) in Hurler patients
Time Frame: 2 years
Incidence of serious adverse events
Time Frame: 2 years
Overall survival
Time Frame: 2 years
Change in cALD Neurologic Function Score over time
Time Frame: 2 years