MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG


The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.

I'm interested

Male or Female
Not specified
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• up to 55 years old
• diagnosis of an Inherited Metabolic Disorders (IMD)
• see link to for complete Inclusion and Exclusion criteria
Exclusion Criteria:

• uncontrolled bacterial, fungal or viral infections including HIV
• women who are pregnant

Rare Diseases

Clinics and Surgery Center (CSC), Allogeneic Hematopoietic Cell Transplantation, IMD, Inherited metabolic disorders, osteopetrosis

Paul Orchard -
Paul Orchard
Phase 2
See this study on