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

Study 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.

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

Conditions
-----------------
Inherited Metabolic Disorders (IMD)

Intervention / Treatment
-----------------
* Other: Safety and efficacy assessments


Participation Criteria
=================
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 Exclusion Criteria: •Patients enrolled in an IMD study in the MGTA-456 program who did not receive MGTA-456 or were withdrawn from the core study.

Ages Eligible for Study
-----------------
Maximum Age: 16 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Subjects treated with MGTA-456<br>MGTA-456 is an investigational expanded CD34+ cell therapy | Other: Safety and efficacy assessments<br>* Long term safety and clinical outcomes<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Inherited Metabolic Disorders, Hematopoietic Stem Cell Transplant, MGTA-456, Umbilical Cord Blood Transplant, Hurler Syndrome, Cerebral Adrenoleukodystrophy, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy