Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Paroxysmal Nocturnal Hemoglobinuria
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring PNH
Eligibility Criteria
Inclusion Criteria:
- Male or female ≥18 years of age
- Treated with eculizumab for PNH for at least 3 months prior to Day 1
- LDH level ≤1.5 × upper limit of normal (ULN) at screening
- PNH diagnosis confirmed by documented high-sensitivity flow cytometry.
- Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
- Body weight ≥40 to <100 kilogram (kg)
- Female participants of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
- Willing and able to give written informed consent and comply with study visit schedule.
Exclusion Criteria:
- LDH level ≤ 2 × upper limit of normal (ULN) within 3 months prior to Day 1
- History of bone marrow transplantation.
- History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the Investigator or Sponsor, would preclude participation.
- Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH).
- Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1.
- Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.
Sites / Locations
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Arms of the Study
Arm 1
Arm 2
Experimental
Active Comparator
Ravulizumab SC Treatment Group
Ravulizumab IV Treatment Group
In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by SC maintenance doses of ravulizumab administered via the ravulizumab OBDS on Day 15 and every week (qw) thereafter for a total of 10 weeks of study treatment. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.
In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by IV maintenance doses of ravulizumab on Day 15. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.