Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.
Coversin, 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 and proven resistance to eculizumab due to C5 polymorphisms. Patients will be treated with Coversin by daily subcutaneous injection for 6 months 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 Coversin and being entered into the long term follow-up study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
1
Coversin - 22.5mg followed by 45mg for 6 months.
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Measurement of Serum Lactate Dehydrogenase (LDH)
Measurement of serum lactate dehydrogenase (LDH)
Time frame: Baseline to Day 186
Percentage Change in Haemoglobin (Hb) Compared to Screening
Percentage change in the haemoglobin (Hb) value at each study visit compared to screening
Time frame: Baseline to Day 186
Number of Packed Red Blood Cells (PRBC) Transfusions
Number of units of packed red blood cells (PRBC) transfusions
Time frame: Baseline to Day 180
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