NCT03748823

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

A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 139 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The study will consist of up to a 30-day Screening Period, a 10-week Randomized Treatment Period, and an Extension Period of up to 172 weeks.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
COMBINATION_PRODUCT	Ravulizumab OBDS	The ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector.
BIOLOGICAL	Ravulizumab	Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight.

Timeline

• Start date: 2019-02-19
• Primary completion: 2021-02-02
• Completion: 2023-08-31
• First posted: 2018-11-21
• Last updated: 2024-09-19
• Results posted: 2022-10-20

Locations

51 sites across 14 countries: United States, Australia, Austria, Belgium, Brazil, Canada, Finland, France, Italy, Netherlands, Russia, Spain, Sweden, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study
• FDA-regulated device study