Status:
COMPLETED
Allogeneic Transplantation For Severe Osteopetrosis
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Severe Osteopetrosis
Eligibility:
All Genders
1-45 years
Phase:
PHASE2
PHASE3
Brief 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 radiat...
Detailed Description
This revised transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol and a second infusion of stem cells on day 42, 2) the mortality asso...
Eligibility Criteria
Inclusion
- 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.
- 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 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
Key Trial Info
Start Date :
August 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2013
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT00775931
Start Date
August 1 2008
End Date
June 1 2013
Last Update
July 31 2019
Active Locations (1)
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1
University of MInnesota, Fairview
Minneapolis, Minnesota, United States, 55455