NCT03406507

A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria

A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ravulizumab in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH).

Detailed description

The study consists of a 4-week Screening Period, a 26-week Primary Evaluation Period, and an Extension Period of up to 4 years (with the exception of any country-specific mandates), whichever occurs first. Efficacy and safety data are reported for the 26-week Primary Evaluation Period only. Analyses were conducted separately for complement inhibitor treatment-naïve participants and eculizumab-experienced participants.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
BIOLOGICAL	Ravulizumab	Single intravenous (IV) loading dose on Day 1, followed by regular IV maintenance dosing beginning on Day 15, based on weight.

Timeline

• Start date: 2018-02-22
• Primary completion: 2020-03-25
• Completion: 2022-08-25
• First posted: 2018-01-23
• Last updated: 2023-03-27
• Results posted: 2022-05-09

Locations

9 sites across 6 countries: United States, France, Netherlands, Norway, Russia, United Kingdom

Regulatory

• FDA-regulated drug study