NCT04735432

Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis

A Phase 3, Randomized, Open-Label, Parallel-Group Study to Compare the Pharmacodynamics, Pharmacokinetics, Efficacy, Safety, Tolerability, and Immunogenicity of Multiple Subcutaneous Injections of Efgartigimod PH20 SC With Multiple Intravenous Infusions of Efgartigimod in Patients With Generalized Myasthenia Gravis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 110 (actual)
• Sponsor: argenx · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to investigate the Pharmacodynamics (PD), Pharmacokinetics (PK), safety, tolerability, immunogenicity, and clinical efficacy of efgartigimod coformulated with recombinant human hyaluronidase PH20 (rHuPH20) as compared to efgartigimod IV infused in patients with generalized myasthenia gravis (gMG). The study duration is approximately 12 weeks. After screening, patients will be randomized to receive either efgartigimod infusions or efgartigimod PH20 subcutaneously (SC)

Detailed description

Main objective of the trial: To demonstrate that the pharmacodynamic (PD) effect of injections of 1000 mg efgartigimod PH20 SC (efgartigimod co-formulated with recombinant humanhyaluronidase PH20 for subcutaneous administration), administered once weekly for 4 administrations, is NI (noninferior) to IV infusions of efgartigimod (efgartigimod formulation for intravenous infusion) at a dose of 10 mg/kg administered once weekly for 4 administrations. Secondary objectives: To compare the PD effect of efgartigimod PH20 SC and efgartigimod IV over time; To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC and efgartigimod IV; To evaluate the safety, tolerability, and immunogenicity of efgartigimod PH20 SC and efgartigimod IV; To evaluate the clinical efficacy of efgartigimod PH20 SC and efgartigimod IV.

Conditions

• Generalized Myasthenia Gravis

Interventions

Type	Name	Description
BIOLOGICAL	efgartigimod PH20 SC	Subcutaneous injection with efgartigimod PH20 SC
BIOLOGICAL	efgartigimod IV	Intravenous infusion of efgartigimod

Timeline

• Start date: 2021-02-05
• Primary completion: 2021-11-02
• Completion: 2021-12-13
• First posted: 2021-02-03
• Last updated: 2023-02-28
• Results posted: 2023-02-28

Locations

47 sites across 11 countries: United States, Belgium, Georgia, Germany, Hungary, Italy, Japan, Netherlands, Poland, Russia, Spain

Regulatory

• FDA-regulated drug study