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

2019-07-11 09:17:52 | BioPortfolio

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

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

Conditions

Inherited Metabolic Disorders (IMD)

Intervention

Safety and efficacy assessments

Location

University of Minnesota
Minneapolis
Minnesota
United States
55455

Status

Enrolling by invitation

Source

Magenta Therapeutics, Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2019-07-11T09:17:52-0400

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Medical and Biotech [MESH] Definitions

Hereditary disorders of pyruvate metabolism. They are difficult to diagnose and describe because pyruvate is a key intermediate in glycolysis, gluconeogenesis, and the tricarboxylic acid cycle. Some inherited metabolic disorders may alter pyruvate metabolism indirectly. Disorders in pyruvate metabolism appear to lead to deficiencies in neurotransmitter synthesis and, consequently, to nervous system disorders.

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Disorders affecting amino acid metabolism. The majority of these disorders are inherited and present in the neonatal period with metabolic disturbances (e.g., ACIDOSIS) and neurologic manifestations. They are present at birth, although they may not become symptomatic until later in life.

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