NCT04008849

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

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

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 3 (actual)
• Sponsor: Magenta Therapeutics, Inc. · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Inherited Metabolic Disorders (IMD)

Interventions

Type	Name	Description
OTHER	Safety and efficacy assessments	Long term safety and clinical outcomes

Timeline

• Start date: 2019-05-21
• Primary completion: 2020-10-08
• Completion: 2020-10-08
• First posted: 2019-07-05
• Last updated: 2021-03-15

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study