Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditions
Brief summary
Safety and tolerability as assessed by adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory tests.
Detailed description
Efficacy as measured by: proportion of patients achieving Hb ≥ 12.0 g/dL assessed at 6-month intervals; proportion of patients maintaining an increase in Hb ≥ 2 g/dL, achieved in the prior study; proportion of patients who are transfusion free at Weeks 48 and 96; proportion of patients experiencing clinical BTH at Weeks 48 and 96; mean LDH and absolute reticulocyte changes from from baseline, start of the long-term extension, at Weeks 48 and 96; zaltenibart PK concentrations and PD parameters
Interventions
None listed
Sponsors
Omeros Corp.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability as assessed by adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory tests. | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy as measured by: proportion of patients achieving Hb ≥ 12.0 g/dL assessed at 6-month intervals; proportion of patients maintaining an increase in Hb ≥ 2 g/dL, achieved in the prior study; proportion of patients who are transfusion free at Weeks 48 and 96; proportion of patients experiencing clinical BTH at Weeks 48 and 96; mean LDH and absolute reticulocyte changes from from baseline, start of the long-term extension, at Weeks 48 and 96; zaltenibart PK concentrations and PD parameters | — |
Countries
Germany
Outcome results
None listed