Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Interventions
Trade Name: ULTOMIRIS
Product Name: ULTOMIRIS
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: RAVULIZUMAB
Current Sponsor code: ALXN1210
Concentr
Sponsors
Alexion Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participant must be 18 years of age or older, at the time of signing the informed consent. Documented diagnosis of PNH. Received 1200 mg eculizumab every 12 to 16 days (q2w) for at least 3 months prior to Screening. Lactate dehydrogenase (LDH) = 2 x upper limit of normal (ULN). Vaccinated against meningococcal infections within 3 years prior to, initiating study drug. Body weight = 40 kg. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: History of major adverse vascular events (MAVEs) within 6 months of Day 1. History of bone marrow transplantation. N. meningitidis infection: Participants with unresolved N. meningitidis infection at treatment initiation, Participants who are not currently vaccinated against N. meningitidis unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination. History of unexplained, recurrent infection. Active systemic bacterial, viral, or fungal infection within 14 days prior to study drug administration on Day 1. Concomitant use of anticoagulants is prohibited if not on a stable regimen for at least 2 weeks prior to Day 1. More than one LDH value > 2 × ULN within the 6 months prior to Day 1. Platelet count < 30,000/mm3 (30 × 109/L) at Screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the prevalence of free complement component 5 (C5)-associated breakthrough hemolysis (BTH) in participants on high-dose eculizumab who switch to ravulizumab;Secondary Objective: To evaluate efficacy by other measures in participants on high-dose eculizumab who switch to ravulizumab; ;Primary end point(s): Free C5-associated BTH through Day 351;Timepoint(s) of evaluation of this end point: Day 351 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): BTH through Day 351 Hemolysis from baseline to Day 351 Transfusion from baseline to Day 351 Stabilized hemoglobin from baseline to Day 351;Timepoint(s) of evaluation of this end point: Throughout the study | — |
Countries
United Kingdom
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed