NCT04320602

Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab

Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat paroxysmal nocturnal hemoglobinuria (PNH).

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
BIOLOGICAL	Eculizumab	Participants must have been prescribed and be receiving a stable dose of eculizumab 1200 milligrams (mg) every 2 weeks (q2w) for at least 3 months prior to the Screening Period. During the Screening Period, participants will continue to receive eculizumab 1200 mg q2w.
BIOLOGICAL	Ravulizumab	During the Treatment Period, participants will receive a loading dose of ravulizumab on Day 1, followed by maintenance doses on Day 15 and every 8 weeks, administered by intravenous infusion. Ravulizumab loading and maintenance doses will be based on participants' body weight per approved dose regimen.

Timeline

• Start date: 2021-04-14
• Primary completion: 2022-12-20
• Completion: 2022-12-20
• First posted: 2020-03-25
• Last updated: 2024-09-05
• Results posted: 2024-08-12

Locations

4 sites across 1 country: United Kingdom