Description of Trial: This is a Phase 2 Study designed to evaluate the safety and efficacy of a pharmacological agent to produce rapid and sustained engraftment for patients undergoing a Hematopoietic Stem Cell Transplant (HSCT) with an Inherited Metabolic Disorder (IMD).
Patient Inclusion Criteria:
- Diagnosed with one of the following:
- Must have adequate organ function
- Must have availability of eligible donor material
Patient Exclusion Criteria:
- Availability of a matched-related donor who is not a carrier of the same genetic defect
- Active infection at screening
- Prior myeloablative conditioning
- History of human immunodeficiency virus (HIV) infection
- Choosing to participate in a clinical trial is a personal decision and one that should be discussed thoroughly with your specific care team.
- For more information on this specific clinical trial, please contact Magenta or Dr. Paul Orchard. KrabbeConnect does not have any additional insight or information.
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