TRIUMPH: A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
- Determine the safety of eculizumab in patients with transfusion-dependent hemolytic
paroxysmal nocturnal hemoglobinuria.
- Determine the efficacy of this drug, in terms of hemoglobin stabilization and the
number of packed red blood cell units transfused during the 26-week treatment period,
in these patients.
- Compare the occurrence of transfusion avoidance, hemolysis (measured by lactate
dehydrogenase [LDH] area under the curve), and the changes in fatigue during the
26-week treatment period in patients treated with this drug vs placebo.
- Compare LDH changes, quality of life changes, thrombosis, platelet activity, nitric
oxide, and free hemoglobin measures during the 26-week treatment period in patients
treated with these regimens.
OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients
are stratified according to the number of packed red blood cell (PRBC) units transfused 1
year prior to screening (< 15 units vs 15-25 units vs > 25 units). Patients are randomized
to 1 of 2 treatment arms.
- Arm I: Within 10 days after PRBC transfusion (administered during the study observation
period), patients receive placebo IV over 30 minutes once a week for 5 weeks and then
once every 2 weeks for 21 weeks.
- Arm II: Within 10 days after PRBC transfusion (administered during the study
observation period), patients receive eculizumab IV over 30 minutes once a week for 5
weeks and then once every 2 weeks for 21 weeks.
Quality of life is assessed at baseline; at weeks 0-4, 12, 20, and 26 during study
treatment; then at weeks 1, 2, 4, and 8 after completion of study treatment.
After completion of study treatment, patients are followed at weeks 1, 2, 4, and 8.
PROJECTED ACCRUAL: Approximately 75 patients (37 per treatment arm) will be accrued for this
Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Prevention
Ronald Paquette, MD
Jonsson Comprehensive Cancer Center
United States: Federal Government
|Jonsson Comprehensive Cancer Center at UCLA||Los Angeles, California 90095-1781|