Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft
N/A
45 Years
Both
Osteopetrosis
Trial Information
Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft
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, based on differential imaging and biologic evaluations prior to
transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and
5 years). Additional biologic studies will include microarray analysis, Campath levels just
prior to the administration of the graft, and establishment of mesenchymal stem cell lines.
In older patients, studies to evaluation osteoclast differentiation and function will also
be offered.
Inclusion Criteria:
- 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 Criteria:
- 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
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Number of Patients Achieving Donor Cell Engraftment
Outcome Description:
Number of patients with persistent presence of donor-derived cells at Day 100
Outcome Time Frame:
Day 100
Safety Issue:
No
Principal Investigator
Paul Orchard, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Masonic Cancer Center, University of Minnesota
Authority:
United States: Institutional Review Board
Study ID:
0704M06581
NCT ID:
NCT00638820
Start Date:
September 2007
Completion Date:
May 2008
Related Keywords:
- Osteopetrosis
- osteopetrosis
- Osteopetrosis
Name | Location |
|---|---|
| University of Minnesota, Fairview | Minneapolis, Minnesota 55455 |
