Clinical Trial: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional




Official Title: Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation

Brief Summary: The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).