Allogeneic Transplantation For Severe Osteopetrosis

• Status: Completed
• Trial end date: 2013-06-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this research is to explore what we believe may be a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality. Prior multi-institutional data in past studies found that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we utilize a reduced intensity design of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Alemtuzumab; Busulfan; Cyclophosphamide; Fludarabine; Fludarabine phosphate; Vidarabine

Criteria

Inclusion Criteria:

• Patients eligible for transplantation under this protocol will be < or = 45 years of age, and will be diagnosed with severe osteopetrosis. This will be defined as having the following manifestations of the disease.

1. Bones that are uniformly markedly dense based on skeletal survey

2. No history that would suggest autosomal dominant inheritance

3. Evidence of hematologic changes that are attributed to the underlying disease, including

• the need for ongoing transfusions, OR

• the presence of progressive anemia or thrombocytopenia, OR

• a white blood cell differential with a predominance of immature forms and evidence of extramedullary hematopoiesis, OR

• persistence of serious infectious complications that are thought to be due to the abnormal architecture of the bone that are resistant to surgical and medical interventions.

Exclusion Criteria:

• Patients >45 years of age

• Evidence of hepatic failure

• Pulmonary dysfunction sufficient to significantly increase the risk of transplant.

• Renal dysfunction with glomerular filtration rate (GFR) <30% of predicted.

• Cardiac compromise sufficient to substantially increase the risk of transplantation

• Severe, stable neurologic impairment.

• Human immunodeficiency virus (HIV) positivity.

• Pregnant or lactating females