A Phase II, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of OTI-010 in Subjects Who Receive HLA-Identical Sibling Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies
- Compare the safety and efficacy of OTI-010 vs placebo as graft-versus-host disease
prophylaxis in patients with hematologic malignancies undergoing HLA-identical sibling
matched peripheral blood stem cell transplantation.
OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients
are stratified according to age (18 to 34 vs 35 to 55) and donor/recipient gender (female
donor/male recipient vs female donor/female recipient vs male donor/female recipient vs male
- Conditioning regimen: Patients receive cyclophosphamide IV once daily on days -5 and -4
and undergo total body irradiation twice daily on days -3 to -1 OR busulfan IV over 2
hours every 6 hours on days -7 to -4 and cyclophosphamide IV once daily on days -3 and
- Graft-versus-host disease prophylaxis: Patients receive methotrexate IV on days 1, 3,
6, and 11. Patients also receive cyclosporine orally or IV (over 1-4 hours) twice daily
beginning on day -1 and continuing for at least 6 months followed by a taper until 1
year after transplantation.
- OTI-010 therapy: Patients are randomized to 1 of 3 treatment arms.
- Arm I: Patients receive placebo IV 4 hours before peripheral blood stem cell
transplantation (PBSCT) on day 0.
- Arm II: Patients receive OTI-010 IV 4 hours before PBSCT on day 0.
- Arm III: Patients receive a higher dose of OTI-010 IV 4 hours before PBSCT on day
- Allogeneic stem cell transplantation: Patients undergo allogeneic PBSCT on day 0.
Patients are followed at 18 weeks, at 6, 9, and 12 months, every 6 months for 1 year, and
then annually for 3 years.
PROJECTED ACCRUAL: A total of 99 patients (33 per treatment arm) will be accrued for this
study within 5 months.
Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Supportive Care
Mary C. Territo, MD
Jonsson Comprehensive Cancer Center
United States: Federal Government
|Jonsson Comprehensive Cancer Center, UCLA||Los Angeles, California 90095-1781|