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Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft

Phase 2
45 Years
Not Enrolling

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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

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:


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:


Principal Investigator

Paul Orchard, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Masonic Cancer Center, University of Minnesota


United States: Institutional Review Board

Study ID:




Start Date:

September 2007

Completion Date:

May 2008

Related Keywords:

  • Osteopetrosis
  • osteopetrosis
  • Osteopetrosis



University of Minnesota, FairviewMinneapolis, Minnesota  55455