Table of contents

• Trial overview
• Study design and phases
• Who can participate
• What the study measures
• Trial groups and comparators
• Patient-friendly terms to know

Trial overview

The available trial of IM-101 is a multicenter study in adults with generalized myasthenia gravis and ocular myasthenia gravis^[1]. It is authorised and plans to enroll 135 participants^[1].

The study is designed to look at safety, tolerability, and early signs of benefit in these patient groups^[1].

Study design and phases

This is a Phase 1b/2 study, and the trial record also lists it as Phase 1^[1]. It is an interventional study, which means the research team gives a study treatment and then measures what happens^[1].

The study is randomized, double-blind, and placebo-controlled^[1]. Randomized means people are placed into groups by chance, double-blind means the participant and study team do not know who gets which treatment, and placebo-controlled means one group receives a look-alike treatment without IM-101^[1].

The trial has two main parts: Part A includes multiple ascending dose cohorts, and Part B includes expansion cohorts^[1].

Who can participate

Part A focuses on participants with AChR antibody-positive generalized myasthenia gravis^[1]. AChR antibody-positive means a blood test found antibodies against the acetylcholine receptor, which is a marker used to define a subgroup of the disease^[1].

Part B includes people with AChR antibody-positive generalized myasthenia gravis, AChR antibody-negative generalized myasthenia gravis, and ocular myasthenia gravis^[1]. AChR antibody-negative means that blood test marker was not found^[1].

What the study measures

The main safety measures include treatment-emergent adverse events, serious adverse events, and adverse events that lead to stopping study treatment early^[1]. Treatment-emergent means the event starts after the study treatment begins, and adverse event means any unwanted medical problem seen during the study^[1].

The study also tracks clinically significant changes in physical examinations and ECGs, which are tests that record the heart’s electrical activity^[1].

For efficacy, meaning whether the treatment may help symptoms, the study measures change from baseline to Week 16 in the MG-ADL total score for generalized myasthenia gravis and the MGII ocular score for ocular myasthenia gravis^[1]. Baseline means the starting point before treatment, and a score change helps show whether symptoms get better or worse over time^[1].

Trial groups and comparators

In the expansion cohorts, IM-101 is compared with placebo in participants with AChR antibody-positive generalized myasthenia gravis, AChR antibody-negative generalized myasthenia gravis, and ocular myasthenia gravis^[1].

The study also includes several vaccine-related interventions in the trial record, but the main research focus for this record is the evaluation of IM-101 in the listed myasthenia gravis groups^[1].

Patient-friendly terms to know

Myasthenia gravis is a disease that causes muscle weakness because the immune system affects how muscles work^[1]. In generalized disease, weakness can affect several body areas, while in ocular disease it mainly affects the eyes^[1].

Safety means the study is checking for unwanted medical problems, and efficacy means it is checking whether the treatment may improve the disease^[1].

Expansion cohorts are added study groups used to collect more information after the first part of a trial^[1]. In this study, they help researchers learn more about both safety and possible benefit in different myasthenia gravis groups^[1].