NCT02605993

Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.

Detailed description

The study consisted of a screening period of up to 30 days and a Treatment Period of up to 253 days for Cohorts 1-3 and 281 days for Cohort 4. After completion of the Treatment Period, all participants had the opportunity to enter the Extension Period, wherein participants continue to receive ravulizumab for up to 5 years. The first dose in the Extension Period occurred on Day 253 for Cohorts 1-3 and on Day 281 for Cohort 4. The data presented includes the Primary Completion date of the study for the Treatment Period. The results for the Extension Period will be reported after study completion.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria
• PNH

Interventions

Type	Name	Description
BIOLOGICAL	Ravulizumab	All treatments were given as IV infusions.

Timeline

• Start date: 2016-01-04
• Primary completion: 2017-02-23
• Completion: 2022-01-12
• First posted: 2015-11-17
• Last updated: 2023-01-04
• Results posted: 2019-02-18

Locations

16 sites across 7 countries: Canada, France, Germany, South Korea, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study