Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1 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 6 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 6 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
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 Safety Endpoints Efficacy Endpoints ;Timepoint(s) of evaluation of this end point: throughout the study | — |
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 | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, 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