Status:
TERMINATED
Reduced Intensity AlloTransplant For Osteopetrosis
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Osteopetrosis
Eligibility:
All Genders
Up to 45 years
Phase:
PHASE2
Brief Summary
We believe that hematopoietic stem cell transplantation (HSCT) will help subjects with Osteopetrosis generate functioning osteoclasts, and by so doing assist in the resolution of the abnormal bone arc...
Detailed Description
This transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol, 2) the mortality associated with transplant by day 100, 3) patient outcomes...
Eligibility Criteria
Inclusion
- Patients eligible for transplantation under this protocol will be \<45 years of age, and will be diagnosed with severe osteopetrosis. This will be defined as having the following manifestations of the disease.
- Bones that are uniformly markedly dense based on skeletal survey
- No history that would suggest autosomal dominant inheritance
- 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
- Patients \>45 years of age
- Evidence of hepatic failure
- pulmonary dysfunction sufficient to substantially 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
Key Trial Info
Start Date :
September 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2008
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00638820
Start Date
September 1 2007
End Date
May 1 2008
Last Update
December 28 2017
Active Locations (1)
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1
University of MInnesota, Fairview
Minneapolis, Minnesota, United States, 55455