Paroxysmal Nocturnal Hemoglobinuria (PNH). Paroxysmal nocturnal hemoglobinuria
Conditions
Interventions
Intervention 1: Intervention group: Infusions of 600 mg of Eculizumab(produced by CinnaGen Co) every week for 4 weeks, followed 1 week later by 900 mg of Eculizumab, and then by a maintenance dose of
Treatment - Drugs
Placebo
Intervention group: Infusions of 600 mg of Eculizumab(produced by CinnaGen Co) every week for 4 weeks, followed 1 week later by 900 mg of Eculizumab, and then by a maintenance dose of 900 mg of Eculiz
Control group: Infusions of 600 mg of Placebo every week for 4 weeks, followed 1 week later by 900 mg of Placebo, and then by a maintenance dose of 900 mg of Placebo every 2 weeks through week 26
Sponsors
CinnaGen Company
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: Patients 18 years of age or older who had received at least four transfusions during the previous 12 months Platelet counts of at least 100,000 per cubic millimeter Lactate dehydrogenase levels that were at least 1.5 times the upper limit of the normal range Eastern Cooperative Oncology Group (ECOG) performance status score of 1 or less PNH(Paroxysmal Nocturnal Haemoglobinuria) diagnosis after flow cytometry and assessment of CD55 and CD59;
Exclusion criteria
Exclusion criteria: Mean hemoglobin level greater than 10.5 g per deciliter before transfusion during the 12 months before entry into the study Receiving another investigational drug within 30 days before the first visit Patients who had a complement deficiency Active bacterial infection History of meningococcal disease Those who had undergone bone marrow transplantation Pregnant and nursing women
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The stabilization of hemoglobin levels, defined as a hemoglobin value that was maintained above the level at which the qualifying transfusion was administered, in the absence of transfusions during the 26-week treatment period. Timepoint: Weekly and then bi-weekly until week 26. Method of measurement: Complete Blood Count (CBC).;The number of units of packed red cells transfused during that period. Timepoint: Weekly and then bi-weekly until week 26. Method of measurement: Calculation. | — |
Secondary
| Measure | Time frame |
|---|---|
| Immunogenicity. Timepoint: Weeks 1, 4, 12 and 16. Method of measurement: ELISA.;Independence of Transfusion and Receiving Blood. Timepoint: From the second week of observation until the 12th week, and from the fourth week after the intervention, until the week 26, every two weeks. Method of measurement: Calculation.;Hemolysis. Timepoint: Screening visit, after the intervention, until the fourth week on a weekly basis, and then every four weeks until the 24th week and in the final visit. Method of measurement: by the lactate dehydrogenase value for the area under the curve.;Presence of thrombosis. Timepoint: During the three months of the observation period, once every two weeks and after the intervention, in the first four weeks weekly, and then until the week 26, every two weeks. Method of measurement: Doppler ultrasound from a marked organ and assessment of serum d-dimer level.;Changes in LDH level. Timepoint: Baseline and weeks 1, 2, 3, 4, 8, 12, 16, 20, 24, 26. Method of measurement: Blood LDH Test.;Adverse event. Timepoint: Weekly and then bi-weekly until week 26. Method of measurement: evaluation adverse events and adverse drug reaction. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactSomayeh Amini
Orchid Pharmed Co.
Outcome results
None listed