Mucopolysaccharidosis (MPS) Center
Clinical trials help us learn more about MPS.
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This phase 2 study is designed to evaluate the safety and efficacy of MGTA-456 after myeloablative conditioning to induce rapid and sustained hematopoietic engraftment with replacement of the specific protein product missing or defective in the patient with an IMD. The study aims to enhance the efficacy of umbilical cord blood transplantation (UCBT), to preserve neurodevelopment in patients with selected IMDs. Since MGTA-456 offers increased numbers of HSCs over standard UCB, it is expected to reduce the risks of prolonged neutropenia and thrombocytopenia and graft failure, and potentially transplant-related mortality (TRM). Patients with Hurler syndrome (also referred to as mucopolysaccharidosis-1H (MPS-1H)), cerebral adrenoleukodystrophy (cALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD) (also referred to as Krabbe disease) could be eligible for this study.
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring for patients with various inherited metabolic disorders and severe osteopetrosis.
Neurobehavioral function and quality of life are compromised in many patients with mucopolysaccharidosis (MPS) disorders. The long-term goals of this research are to: 1) more accurately inform patients/parents regarding potential neurobehavioral outcomes; 2) develop sensitive measures of disease progression and central nervous system (CNS) treatment outcome; and 3) help clinical researchers develop direct treatments for specific brain structures/functions. The investigators hypothesize that specific and localized neuroimaging and neuropsychological findings and their relationship will be distinct for each MPS disorder. It is further hypothesized that without treatment, functions will decline and structure will change over time in a predictable fashion, and will be related to locus of abnormality and stage of disease.