NCT03946748

Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

An Open-Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor-Naive or Have Not Recently Received Complement Inhibitor Therapy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to demonstrate a reduction in intravascular hemolysis by REGN3918 over 26 weeks of treatment in patients with active PNH who are treatment-naive to complement inhibitor therapy or have not recently received complement inhibitor therapy. The secondary objectives of the study are: * To evaluate the safety and tolerability of REGN3918. * To evaluate the effect of REGN3918 on parameters of intravascular hemolysis * To assess the concentrations of total REGN3918 in serum. * To evaluate the incidence of treatment-emergent anti-drug antibodies to REGN3918 over time * To evaluate the effect of REGN3918 on patient-reported outcomes (PROs) measuring fatigue and health-related quality of life

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

Type	Name	Description
DRUG	REGN3918	Single intravenous (IV) dose, then a subcutaneous (SC) dose once weekly (QW).

Timeline

• Start date: 2019-05-16
• Primary completion: 2021-06-09
• Completion: 2021-06-10
• First posted: 2019-05-13
• Last updated: 2023-06-26
• Results posted: 2023-06-26

Locations

12 sites across 5 countries: Hong Kong, Hungary, Malaysia, South Korea, United Kingdom