Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Completed · Phase 3

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria

In brief

The primary objective of this study is to evaluate pharmacokinetics (PK) of ravulizumab administered subcutaneously via an on-body delivery system (OBDS) compared with intravenously administered ravulizumab in adult participants with PNH who are clinically stable on eculizumab for at least 3 months prior to study entry.

Key facts

Study ID

NCT03748823

Run by

Alexion Pharmaceuticals, Inc.

People needed

139

Starts

2019-02-19

Expected to finish

2023-08-31

Last updated by the study team

2024-09-19

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• More than 1 LDH value > 2 × ULN within the 3 months prior to study entry
• 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.

Where it is running

• Research Site — Liverpool, Australia
• Research Site — Parkville, Australia
• Research Site — Vienna, Austria
• Research Site — Antwerp, Belgium
• Research Site — Brussels, Belgium
• Research Site — Hasselt, Belgium
• Research Site — Leuven, Belgium
• Research Site — Botucatu, Brazil
• Research Site — Ribeirão Preto, Brazil
• Research Site — Rio de de Janeiro, Brazil
• Research Site — Salvador, Brazil
• Research Site — São Paulo, Brazil
• Research Site — São Paulo, Brazil
• Research Site — Toronto, Ontario, Canada
• Research Site — Helsinki, Finland
• Research Site — Amiens, France
• Research Site — Brest, France
• Research Site — Lille, France
• Research Site — Montpellier, France
• Research Site — Nantes, France
• Research Site — Nice, France
• Research Site — Paris, France
• Research Site — Pessac, France
• Research Site — Pierre-Bénite, France
• Research Site — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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