In short
Clinical trials are investigating IM-101 in adults with generalized myasthenia gravis and ocular myasthenia gravis. These studies aim to assess safety, tolerability, and early signs of efficacy in different patient groups, including antibody-positive and antibody-negative participants.
Key points
- IM-101 is being studied in an early clinical trial for adults with generalized myasthenia gravis and ocular myasthenia gravis. The study is authorised and includes 135 participants. It is a Phase 1b/2 randomized, double-blind, placebo-controlled trial, which means the groups are assigned by chance and neither participants nor researchers know who gets IM-101 or placebo during the study. The trial is looking at safety, tolerability, and early signs of benefit. It includes people with AChR antibody-positive generalized myasthenia gravis, and also expansion groups for AChR antibody-negative generalized myasthenia gravis and ocular myasthenia gravis. The main outcome measures include treatment-emergent adverse events, serious adverse events, and changes in symptom scores by Week 16.
Trial overview
The available trial of IM-101 is a multicenter study in adults with generalized myasthenia gravis and ocular myasthenia gravis1. It is authorised and plans to enroll 135 participants1.
The study is designed to look at safety, tolerability, and early signs of benefit in these patient groups1.
Study design and phases
This is a Phase 1b/2 study, and the trial record also lists it as Phase 11. It is an interventional study, which means the research team gives a study treatment and then measures what happens1.
The study is randomized, double-blind, and placebo-controlled1. 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-1011.
The trial has two main parts: Part A includes multiple ascending dose cohorts, and Part B includes expansion cohorts1.
Who can participate
Part A focuses on participants with AChR antibody-positive generalized myasthenia gravis1. AChR antibody-positive means a blood test found antibodies against the acetylcholine receptor, which is a marker used to define a subgroup of the disease1.
Part B includes people with AChR antibody-positive generalized myasthenia gravis, AChR antibody-negative generalized myasthenia gravis, and ocular myasthenia gravis1. AChR antibody-negative means that blood test marker was not found1.
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 early1. Treatment-emergent means the event starts after the study treatment begins, and adverse event means any unwanted medical problem seen during the study1.
The study also tracks clinically significant changes in physical examinations and ECGs, which are tests that record the heart’s electrical activity1.
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 gravis1. Baseline means the starting point before treatment, and a score change helps show whether symptoms get better or worse over time1.
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 gravis1.
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 groups1.
Patient-friendly terms to know
Myasthenia gravis is a disease that causes muscle weakness because the immune system affects how muscles work1. In generalized disease, weakness can affect several body areas, while in ocular disease it mainly affects the eyes1.
Safety means the study is checking for unwanted medical problems, and efficacy means it is checking whether the treatment may improve the disease1.
Expansion cohorts are added study groups used to collect more information after the first part of a trial1. In this study, they help researchers learn more about both safety and possible benefit in different myasthenia gravis groups1.