rVA576 for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
rVA576, a small protein complement C5 inhibitor which prevents the cleavage of C5 by C5 convertase into C5a and C5b, will be used in an open label, non-comparative clinical trial in patients with PNH. Patients will be treated with rVA576 by daily subcutaneous injection in order to determine the safety and efficacy of the drug in these circumstances. If satisfactory control of the PNH is achieved, and at the discretion of the Principal Investigator (PI), patients will have the option of remaining on rVA576 and being entered into the long term follow-up study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
9
6 months of treatment, rVA576 plus SOC. Followed by a further 3 months of rVA576 plus SOC. In total, 9 months on rVA576 plus SOC.
6 months on SOC followed by 3 months of treatment with rVA576 plus SOC. In total, 3 months on rVA576 plus SOC.
Almaty City Hospital No.7
Almaty, Microdistrict Kalkaman, Kazakhstan
Vilnius University Hospital Santaros Klinikos , Santariškių St. 2, LT-08661,
Vilnius, Lithuania
University of Kelaniya, Faculty of Medicine, Thalagolla Road
Colombo, Sri Lanka
HB (Haemoglobin) stabilisation rate and the avoidance of packed red blood cells (PRBC) transfusions
Haemoglobin stabilisation rate defined as haemoglobin greater than the set point for each patient during the pre-study randomisation period and the avoidance of PRBC transfusions during the treatment period.
Time frame: 9 months
Number of units of packed red blood cells (PRBC) transfused
Number of units of packed red blood cells (PRBC) transfused from Baseline Day 1 to Day 180
Time frame: Day 1 to Day 180
Percentage of patients who achieve transfusion avoidance
Percentage of patients who achieve transfusion avoidance
Time frame: Day 1 to Day 180
Change in (QOl) Quality of Life score
Change in Quality of Life score
Time frame: Day 1 to Day 180
AUC (LDH)
AUC (Area under the curve) (LDH) Lactate Dehydrogenase
Time frame: Day 1 to Day 180
CH50
CH50 (Classical haemolytic 50% lysis)
Time frame: Day 1 to Day 180
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