ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod

Phase 2RecruitingNCT07284420

argenx70 enrolled

Overview

This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life. The aim of this ISA1 is to evaluate the safety and therapeutic relevance of empasiprubart as add-on therapy to efgartigimod in participants with AChR-Ab seropositive generalized myasthenia gravis. The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170

Once the master protocol and ISA1 screening periods are completed, eligible participants can enroll in the run-in period (part A) where they will receive efgartigimod IV. Eligible participants can then continue to the add-on period (part B) where they will receive both efgartigimod IV and empasiprubart IV. Participants who are not eligible for part B will continue directly to the safety follow-up period (part C) where they will receive efgartigimod IV only. The study duration for each participant is approximately up to 54 weeks.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

AChR-Ab Seropositive Generalized Myasthenia Gravis Myasthenia Gravis

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Is seropositive for anti-acetylcholine receptor antibodies (AChR-Ab) * Has confirmed diagnosis of gMG and is Myasthenia Gravis Foundation of America (MGFA) Class II, III, IVa, or IVb * Has documented immunization against encapsulated bacterial pathogens (Neisseria meningitidis and Streptococcus pneumoniae) within 5 years of ISA screening or is willing to receive immunization at least 14 days before the first study drug administration Exclusion Criteria: * Clinical diagnosis of systemic lupus erythematosus (SLE) * Any known complement deficiency * Current administration of a complement inhibitor or received zilucoplan or eculizumab \<2 months or ravulizumab \<6 months before the first study drug administration * Patients proven to be refractory to efgartigimod (ie, not achieving a clinically meaningful improvement in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score defined as an improvement of ≥2 points)

Locations (5)

Profound Research LLC - Carlsbad

Carlsbad, California, United States

RECRUITING

Visionary Investigators Network

Miami, Florida, United States

RECRUITING

Dent Neurologic Institute - Amherst

Amherst, New York, United States

RECRUITING

Erlanger Health System

Chattanooga, Tennessee, United States

RECRUITING

National Neuromuscular Research Institute

Austin, Texas, United States

RECRUITING

Outcomes

Primary Outcomes

Incidence of adverse events and serious adverse events in parts A and B

Time frame: Up to 21 weeks

Secondary Outcomes

MG-ADL total score change from baseline at week 18 in part B (cycle 2 day 29) compared with MG ADL total score change from baseline at week 4 in part A

The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms)

Time frame: Up to 18 weeks

Proportion of participants reaching MSE at any point in part B cycles 1 and 2, and part B cycles 1 or 2

MSE: Minimal symptom expression

Time frame: Up to 21 weeks

MG-ADL total score changes from baseline over time in part B compared with part A

Time frame: Up to 21 weeks

QMG total score change from baseline at week 18 in part B (cycle 2 day 29) compared with QMG total score change from baseline at week 4 in part A

The Quantitative myasthenia gravis (QMG) includes 13 items that measure endurance or fatigability and accounts for fluctuations in disease state. The total score ranges from 0 (no disease severity) to 39 (highest disease severity)

Time frame: Up to 18 weeks

QMG total score changes from baseline over time in part B compared with part A

Time frame: Up to 21 weeks

Proportion of participants who have a 50% MG ADL total score improvement in part B cycles 1 and 2

The Myasthenia Gravis Activities of Daily Living (MG ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms)

Time frame: Up to 21 weeks

Proportion of participants who have positive PASS in part B cycles 1 and 2, and part B cycles 1 or 2

PASS: Patient acceptable symptom state

Time frame: Up to 21 weeks

ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod

Overview

Conditions

Interventions

Eligibility

Locations (5)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts