Clinical trials help us learn more about leukodystrophies and determine whether new treatments are safe and effective.
Gene Therapy: A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (cALD)
This trial will assess the efficacy and safety of autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of cerebral adrenoleukodystrophy (cALD). A patient's blood stem cells will be collected and modified using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification with the Lenti-D lentiviral vector, the cells will be transplanted back into the patient following myeloablative conditioning.
The goal of this single institution study is to evaluate boys with adrenoleukodystrophy (ALD) diagnosed early in life and to prospectively monitor them to determine parameters that will facilitate earlier detection of the childhood cerebral form of the disease. These at-risk patients will be assessed yearly through travel to the University of Minnesota, where plasma and cerebral spinal fluid (CSF) biomarker studies, MRI based imaging, and neuropsychological assessments will be performed at the University of Minnesota Masonic Children's Hospital and Clinics. The MRI and lumbar puncture to obtain CSF will be obtained under sedation. In addition, at intervening 6 months intervals information will be obtained remotely, including surveys and MRI's in their home location. Also at that time, blood samples will be obtained locally and shipped to the University of Minnesota. There is no therapeutic intent in this study.
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.
ALD-103 is a multi-site, global, data collection study that is designed to evaluate safety and efficacy of allogenic hematopoietic stem cell transplantation (allo-HSCT) in patients with cALD aged 17 or younger.