A Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adults With New-onset Generalized Myasthenia Gravis (gMG)

Phase 4

Recruiting

• Conditions: New Onset Generalized Myasthenia Gravis (gMG)
• Interventions: Biological: Efgartigimod PH20 SC

• Registration Number: NCT06909214
• Lead Sponsor: argenx

Brief Summary

The main purpose of this study is to measure how well adults with new-onset gMG (which means they've had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-03-27
• Study First Submitted QC: 2025-03-27
• Study First Posted: 2025-04-03
• Study Start: 2025-04-17
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-21
• Last Update Posted: 2025-05-25
• Primary Completion: 2026-08
• Study Completion: 2027-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• Is at least 18 years when signing the ICF
• Has been diagnosed with gMG of MGFA class II, III, or IV
• Is seropositive for AChR-Ab
• Is treatment-naive for gMG or has been administered AChEI for the treatment of gMG
• Had onset of generalized MG signs and/or symptoms within 12 months before screening; candidates who also had onset of ocular MG signs and/or symptoms within 24 months before screening may be enrolled in the study
• Has an MG-ADL score ≥5

Exclusion Criteria

• gMG diagnosis of MGFA class I or V
• Underwent a thymectomy prior to screening, except thymectomy for treatment of nonmalignant thymoma prior to the gMG diagnosis
• Prior or current use of any of any systemic corticosteroid therapy or nonsteroidal immunosuppressive therapy for the treatment of gMG

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Efgartigimod PH20 SC	Efgartigimod PH20 SC	Participants receiving efgartigimod PH20 SC

Primary Outcome Measures

Name	Time	Method
Proportion of participants who achieve MSE of MG at any time within the first 16 weeks of treatment with efgartigimod PH20 SC	up to 16 weeks	MSE (minimal symptom expression) is defined as an MG-ADL (Myasthenia Gravis Activities of Daily Living) total score of 0 or 1. The MG-ADL scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe).

Secondary Outcome Measures

Name	Time	Method
Changes from baseline in MG-ADL total scores over time	up to 51 weeks	The MG-ADL (Myasthenia Gravis Activities of Daily Living) scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe).
Changes from baseline in MG-QOL-15r total scores over time	up to 51 weeks	The MG-QoL15r (Myasthenia Gravis Quality of Life 15-item scale revised) questionnaire is a patient-reported instrument that measures the impact of MG symptoms on Quality of Life. The score varies between 0 and 30 (with 30 the lowest quality of life).
Proportion of participants who are administered systemic corticosteroid therapy as background add-on therapy throughout the study	up to 55 weeks
Incidence of adverse events, serious adverse events and adverse events leading to study drug discontinuation	up to 55 weeks

Trial Locations

Locations (1)

EZR Research; 🇺🇸 Boca Raton, Florida, United States