A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
- Conditions
- Inherited Metabolic Disorders (IMD)
- Interventions
- Other: Safety and efficacy assessments
- Registration Number
- NCT04008849
- Lead Sponsor
- 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
- 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 3
- 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.
•Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Subjects treated with MGTA-456 Safety and efficacy assessments MGTA-456 is an investigational expanded CD34+ cell therapy
- Primary Outcome Measures
Name Time Method Incidence of serious adverse events 2 years Event-free survival 2 years Very long chain fatty acid blood level (ug/mL) in cALD patients 2 years Incidence of related adverse events 2 years Incidence of chronic graft versus host disease 2 years Incidence of late hematological graft failure 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 Overall survival 2 years Change in cALD Neurologic Function Score over time 2 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States