A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia Gravis

argenx102 sites in 10 countries141 target enrollmentSeptember 18, 2024

ConditionsMyasthenia Gravis, Ocular

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Myasthenia Gravis, Ocular
Sponsor: argenx
Enrollment: 141
Locations: 102
Primary Endpoint: MGII (PRO) ocular score change from baseline to day 29 in part A
Status: Active, not recruiting
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.

Registry

clinicaltrials.gov

Start Date

September 18, 2024

End Date

December 1, 2027

Last Updated

4 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

argenx

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Is at least 18 years of age and the local legal age of consent for clinical studies
•Has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab; or abnormal neuromuscular transmission demonstrated by abnormal neurophysiology testing and history on positive edrophonium chloride testing or demonstrated improvement on MG therapy"
•Is MGFA Class I (any ocular muscle weakness)
•Has a screening and baseline MGII (PRO) ocular score of at least 6 with at least 2 ocular items with a score of at least 2

Exclusion Criteria

•Other diseases that lead to eyelid drooping, peripheral muscle weakness, or diplopia
•Known autoimmune disease or any medical condition other than indication under study that would interfere with an accurate assessment of clinical symptoms of ocular myasthenia gravis or puts the participant at undue risk

Outcomes

Primary Outcomes

MGII (PRO) ocular score change from baseline to day 29 in part A

Time Frame: Up to 29 days

The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease.

Secondary Outcomes

MGII (PRO+PE) ocular score change from baseline to day 29 in part A(Up to 29 days)
MG-ADL ocular domain score change from baseline to day 29 in part A(Up to 29 days)
MGII total score change from baseline to day 29 in part A(Up to 29 days)
MGII (PE) ocular score change from baseline to day 29 in part A(Up to 29 days)
MG-ADL total score change from baseline to day 29 in part A(Up to 29 days)
MGII ocular scores changes (PRO) from baseline in part A+B(Up to 111 weeks)
Incidence of (serious) adverse events(Up to 111 weeks)
MG-QoL15r total score changes from baseline in part A+B(Up to 111 weeks)
NEI VFQ-25 score changes from baseline in part A+B(Up to 111 weeks)
Percent change from baseline in total IgG levels over time in part A+B(Up to 111 weeks)
Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B(Up to 111 weeks)
Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B(Up to 111 weeks)
Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B(Up to 111 weeks)