AN ADAPTIVE PHASE I/II STUDY TO ASSESS SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7112689 IN HEALTHY VOLUNTEERS AND PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH)

1. Safety (Parts 1, 2, 3, and 4): Incidence of dose-limiting events, 2. Safety (Parts 1, 2, 3, and 4): Incidence and severity of adverse events (AEs), serious adverse events and AEs leading to withdrawal, 3. PD (Parts 1, 2, 3, and 4): Ex vivo liposome lysis in serum and ex-vivo lysis of antibody-coated erythrocytes, 4. PD (Parts 1, 2, 3, and 4): Total and target engaged C5 concentration, 5. PD (Parts 1, 2, 3, and 4): Serum Lactate Dehydrogenase (LDH)

1. Efficacy (Parts 2, 3, and 4): Change in LDH, 2. Efficacy (Parts 2, 3, and 4): Change in free-haemoglobin, 3. Efficacy (Parts 2, 3, and 4): Proportion of patients with stabilized haemoglobin levels, 4. Efficacy (Parts 2, 3, and 4): Change in fatigue as measured by the functional assessment of chronic illness therapy fatigue, 5. Efficacy (Parts 2, 3, and 4): Change in health-related quality of life as measured by the European Organization for Research and Treatment of Cancer quality of life questionnaire-core 30, 6. Efficacy (Parts 2, 3, and 4): Number of packed RBC units transfused per patient, 7. Efficacy (Parts 2, 3, and 4): Time to (1) first transfusion or (2) persistent elevation of LDH, 8. Efficacy (Parts 2, 3, and 4): Proportion of patients with LDH below upper limit of normal (ULN), 9. Efficacy (Parts 2, 3, and 4): Proportion of patients with complement suppression throughout the dosing interval, 10. PK (Parts 1, 2, 3, and 4): Pharmacokinetic profile of crovalimab Cmax, Tmax, Area under the curve (AUC), T1/2, bioavailability following SC administration

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