CHP 834 Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transportation With the CliniMACs Device for T and B Cell Depletion
PRIMARY HYPOTHESIS: T cell depletion utilizing the CliniMACS device will allow more precise,
specific and controlled graft engineering of peripheral blood stem cells from unrelated and
partially matched related donors without an increase in relapse or graft rejection and grade
III or IV acute graft vs. host disease (GVHD).
SECONDARY HYPOTHESIS: Use of the CliniMACS device will allow defined levels of T cell
depletion to reflect the risk of severe GVHD in the donor/recipient pair.
Thus, patients with a relatively lower risk of severe GVHD will be assigned to Stratum 1 and
receive a graft with lesser T cell depletion and a defined level of reinfused T cells.
Patients with higher risk of severe GVHD or for whom there is no perceived clinical benefit
of GVHD will be assigned to Stratum 2 and receive a more T cell-depleted graft.
Conditioning of the patient (except immunodeficiencies) includes :
- Thiotepa 5 mg/kg days for 2 days
- Cyclophosphamide 60 mg/kg days for 2 days
- Total body irradiation 200 cGy given twice a day for 3 days
Following conditioning patient's will receive stem cells that have been processed using the
CliniMACS device. This processing is done in the stem cell laboratory at The Children's
Hospital of Philadelphia. The Stem Cell Lab is accredited by the Foundation for the
Accreditation of Cellular Therapy (FACT) and maintain complete standard operating procedures
(SOP's) and procedure records.
Processing of cells using the CliniMACS will occur in accordance with the Investigator
Brochure and Technical Manual following the laboratory SOPs and using aseptic technique. The
CHOP Stem Cell Lab has extensive prior experience with automated cell processing
technologies, including the CellPro Ceprate device and the Isolex 300i.
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Rates of success of engraftment, day 100 treatment related mortality, acute GVHD, relapse and EBV LPD. Patients who die will be considered failure for the engraftment success evaluation, and relapsed for that endpoint.
Stephan Grupp, MD, PhD
Children's Hospital of Philadelphia
United States: Food and Drug Administration
|The Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|