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MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
Status: Recruiting
To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.
Age: Not specified
Healthy Volunteers:
Inclusion Criteria:
• up to 55 years old
• diagnosis of an Inherited Metabolic Disorders (IMD)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
Exclusion Criteria:
• uncontrolled bacterial, fungal or viral infections including HIV
• women who are pregnant
Conditions:
Rare Diseases
Keywords:
Clinics and Surgery Center (CSC), Allogeneic Hematopoietic Cell Transplantation, IMD, Inherited metabolic disorders, osteopetrosis
Contact(s): Paul Orchard - orcha001@umn.edu
Principal Investigator: Paul Orchard
Phase: PHASE2
IRB Number: 1406M51542
System ID: 16279
See this study on ClinicalTrials.gov