This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and continue to have anemia (low red blood cell count). The aim of the study is to see how tolerable ALN-CFB is compared to placebo. A placebo looks like the study drug but does not contain any drug. The study is looking at several other research questions, including: * What side effects may happen from taking ALN-CFB * How much ALN-CFB is in the blood at different times * How much Complement Factor B (CFB) protein levels in the blood are affected by ALN-CFB
The protocol will be amended to describe Part B of the study after Part A data have been analyzed.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
24
Administered as defined in the protocol
Administered as defined in the protocol
Toronto General Hospital
Toronto, Ontario, Canada
RECRUITINGSeoul National University Hospital
Seoul, South Korea
RECRUITINGSamsung Medical Center
Seoul, South Korea
RECRUITINGOccurrence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Through 365 Days
Severity of TEAEs
Time frame: Through 365 Days
Concentrations of combined ALN-CFB and major metabolites in plasma
Time frame: Through 365 Days
Concentrations of combined ALN-CFB and major metabolites in urine
Time frame: Through 24 Hours following ALN-CFB administration
Absolute change from baseline in CFB concentration
Time frame: Baseline, Through 365 Days
Percentage change from baseline in CFB concentration
Time frame: Baseline, Through 365 Days
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