A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults with Ocular Myasthenia Gravis (ADAPT oculus)

argenx

Status and phase: Enrolling; Phase 3

Conditions: Myasthenia Gravis, Ocular

Treatments: Combination Product: Efgartigimod PH20 SC; Other: Placebo PH20 SC

Study type: Interventional

Funder types: Industry

Identifiers

NCT06558279

2024-514133-38-00 (EU Trial (CTIS) Number)

ARGX-113-2315

Details and patient eligibility

About

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.

Enrollment: 124 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

124 participants in 2 patient groups

Efgartigimod PH20 SC in part A+B

Experimental group

Description:

Participants receiving efgartigimod PH20 SC during part A and part B

Treatment:

Combination Product: Efgartigimod PH20 SC

Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B

Experimental group

Description:

Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B

Treatment:

Other: Placebo PH20 SC

Combination Product: Efgartigimod PH20 SC