NCT03056040

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 202 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who were clinically stable after having been treated with eculizumab for at least 6 months.

Detailed description

The study consisted of a 4-week Screening Period and a 26-week Randomized Treatment Period (Primary Evaluation Period). After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive ravulizumab for up to 4 years.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

Type	Name	Description
BIOLOGICAL	Ravulizumab	All treatments were given as intravenous (IV) infusions. For participants weighing ≥40 to \<60 kilograms (kg): 2400 mg was given as a single loading dose, followed by 3000 mg as maintenance dose. For participants weighing ≥60 to \<100 kg: 2700 mg was given as a loading dose, followed by 3300 mg as maintenance dose. For participants weighing ≥100 kg: 3000 mg was given as a loading dose, followed by 3600 mg as maintenance dose.
BIOLOGICAL	Eculizumab	All treatments were given as IV infusions. Participants received 900 mg of eculizumab q2w.

Timeline

• Start date: 2017-05-17
• Primary completion: 2022-02-21
• Completion: 2022-02-21
• First posted: 2017-02-16
• Last updated: 2025-03-12
• Results posted: 2019-03-21

Locations

51 sites across 11 countries: United States, Australia, Canada, France, Germany, Italy, Japan, Netherlands, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study