Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Age 1. Patients must be at least 18 years of age at the time of signing the informed consent. Patient and Disease Characteristics 2. Treated with eculizumab according to the labeled dosing recommendation for PNH (900 mg every 14 days ± 2 days) for at least 3 months prior to study entry with no missed doses within 2 months prior to study entry and no more than 2 doses outside of the visit window. 3. Lactate dehydrogenase levels = 1.5 × ULN (upper limit of normal), according to central laboratory, at Screening. Sample must be obtained within 24 hours of or immediately prior to a scheduled eculizumab dose administration (ie, at trough eculizumab level). 4. Documented diagnosis of PNH confirmed by high-sensitivity flow cytometry evaluation (Borowitz, Craig et al. 2010). 5. Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis). Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 95 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: Medical Conditions 1. More than 1 LDH value > 2 × ULN within the 3 months prior to study entry. 2. Major adverse vascular event (MAVE) in the 6 months prior to study entry. 3. Platelet count < 30,000/mm3 (30 × 109/L) at Screening. 4. Absolute neutrophil count < 500/µL (0.5 × 109/L) at Screening. 5. History of bone marrow transplantation. 6. History of N meningitidis infection.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate PK noninferiority of ravulizumab SC versus ravulizumab IV in adult patients with PNH;Secondary Objective: - To characterize PK of ravulizumab SC - To characterize PD of ravulizumab SC - To characterize immunogenicity of ravulizumab SC - To evaluate HRQoL and treatment satisfaction on ravulizumab SC - To evaluate safety of ravulizumab SC and ravulizumab OBDS - To evaluate efficacy of ravulizumab SC - To assess performance of ravulizumab OBDS;Primary end point(s): Day 71 serum ravulizumab Ctrough;Timepoint(s) of evaluation of this end point: Day 71 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): PK Endpoint: Ctrough over time PD Endpoint: Free serum C5 concentrations over time Immunogenicity HRQoL and Treatment Satisfaction Endpoints Change in FACIT-Fatigue Scale, Version 4, from Baseline to Day 183 - Change in EORTC QLQ-C30 Version 3.0, from Baseline to Day 183 - Reported treatment administration satisfaction as measured by the TASQ score at Baseline and Day 183 - Reported patient preference as measured by the PPQ-SC score at Days 1093 Safety Endpoints Change in physical examinations, vital signs, electrocardiograms, laboratory assessments over time - Incidence of adverse events and serious adverse events - Incidence of adverse device effects and serious adverse device effects Efficacy Endpoints - Change over time in LDH - Incidence of breakthrough hemolysis - Achievement of transfusion avoidance - Achievement of stabilized hemoglobin - Change in clinical manifestations of PNH over time - Change in reticulocyte count over time - Change in eGFR over time - Change in PNH RBC clone size over time Performance Endpoint - Reported outcome of attempted full dose administration (including device failure/malfunction);Timepoint(s) of evaluation of this end point: throughout the study | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, Czechia, Czech Republic, Finland, France, Germany, Italy, Korea, Republic of, Netherlands, Russian Federation, Spain, Sweden, Turkey, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed