The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.
This is a multicenter, open-label, single arm study. The primary objective is to assess the long-term safety and tolerability of OMS906 in patients with PNH. Secondary objectives of this study include assessment of the long-term efficacy of OMS906 in patients with PNH. Patients will receive OMS906 5 mg/kg administered as intravenous (IV) injections at 8-week intervals.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
OMS906 study drug repeat-dose 5mg/kg IV administration at 8-week intervals
Omeros Investigational Site
Aachen, Germany
Omeros Investigational Site
Ulm, Germany
Omeros Investigational Site
Lausanne, Switzerland
Omeros Investigational Site
Kyiv, Ukraine
Omeros Investigational Site
To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.
Treatment-emergent adverse events, including clinically significant clinical laboratory tests, 12-lead electrocardiograms, vital signs, and physical examinations recorded as an adverse event.
Time frame: 104 weeks
To assess efficacy measured by hemoglobin (Hgb).
Measured by patients achieving Hb ≥ 12.0 g/dL and by proportion of patients maintaining an increase in Hb ≥ 2 g/dL, achieved in the prior study, through the duration of the long-term extension.
Time frame: 6 month intervals
To assess efficacy by transfusion requirements.
Measure proportion of patients who are transfusion free and mean change from baseline in transfusion frequency from the start of the long-term extension.
Time frame: Weeks 48 and 96
To assess efficacy by measurement of lactate dehydrogenase (LDH).
Measure mean LDH change from baseline.
Time frame: Weeks 48 and 96
To assess efficacy by measurement of reticulocyte count.
Measure mean change in reticulocyte count from baseline.
Time frame: Weeks 48 and 96
To assess efficacy by measurement of clinical breakthrough hemolysis.
Measure proportion of patients experiencing clinical breakthrough hemolysis.
Time frame: Weeks 48 and 96
To assess population PK Cmax of OMS906.
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter maximum concentration (Cmax).
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Leeds, United Kingdom
Time frame: Weeks 48 and 96
To assess population PK AUC of OMS906.
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter area under the time-concentration curve (AUC).
Time frame: Weeks 48 and 96
To assess population PK terminal half life of OMS906.
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using terminal half-life parameter.
Time frame: Weeks 48 and 96
To assess PD of OMS906
PD parameters include change from baseline in mature complement factor D (FD).
Time frame: Weeks 48 and 96
OMS906 anti-drug antibodies (ADA).
Presence of ADA in serum will be measured.
Time frame: Weeks 24, 48, 72, and 96
Assess the change in Functional Assessment of Chronic Illness Therapy (FACIT) fatigue score.
To assess the effect of OMS906 on Quality of Life using the FACIT fatigue scale.
Time frame: Weeks 24, 48, 72, and 96