Inclusion Body Myositis – Clinical Trials

Clinical trials for inclusion body myositis are research studies that investigate the effectiveness of new treatments, medical tests, or devices. Trials are carefully designed and follow strict procedures to help provide reliable results.

Clinical Trials – General Considerations

  • Phase I: Initial studies of new drugs in humans are small, short studies designed primarily to see if the medication is safe.
  • Phase II: If a medication seems safe, a larger and longer study will be done to begin to learn if the drug actually works, and to obtain more safety data. Sometimes studies are designated as either Phase IIa or Phase IIb. Phase IIa studies are more exploratory in nature. Although they are designed to reveal information about the efficacy of the drug, the primary endpoint may be to determine the optimal frequency of dosing, the type of patient most suitable for future testing, or other aspects of safety and efficacy. Phase IIb studies are more advanced, and are generally designed with efficacy as the primary endpoint.
  • Phase III: The final phase involves research studies of large groups of patients.

Diagram of test tube, DNA, and patientsEach trial has eligibility criteria that specify which patients are allowed to participate. This can be frustrating if you are a patient who does not meet the criteria, especially for diseases like inclusion body myositis that do not have currently approved treatments. The purpose of the criteria is to ensure that all the patients in the trial have the same disease, and that they can participate in all aspects of the treatment and testing. This increases the likelihood that a successful treatment will be identified. If the treatment eventually does receive FDA approval, the treatment might then be given to patients who did not meet the eligibility criteria for the study.

Clinical trials for Rare Diseases are Challenging:

  • It can be difficult to recruit adequate numbers of patients.
  • Clinical trials are expensive, and funding can be hard to obtain.
  • The natural history of the disease may be poorly known.

Natural History Studies

The natural history of a chronic disease refers to how the disease affects people, from the time the disease begins until the patient dies, without treatment. Accurate knowledge of how the disease progresses is crucial to the design and implementation of clinical trials. How do you test if a drug works if you don’t know what happens in the disease without the drug? Cure IBM encourages all inclusion body myositis patients to participate in natural history studies.

All eligible people with inclusion body myositis are urged to participate in the INSPIRE-IBM study, a large natural history study that is currently recruiting new participants. Study sites are located throughout the United States. Please visit the webpage for more information.

The online registry is an online registry of clinical trials, and is the place to go if you would like to search for clinical trials for inclusion body myositis. The site is managed by the United States National Library of Medicine and the National Institutes of Health. Although it is a US government website, some studies offered outside the United States are listed on the website. After entering inclusion body myositis as the name of the condition/disease, clicking the “Search all studies” button will bring up a list not only of active studies that are currently recruiting new patients, but also recently concluded studies and studies which are about to begin.

Inclusion Body Myositis Clinical Trials of Current Interest


A Phase 2/3 clinical trial of ABC008, made by Abcuro, is currently underway. ABC008 is an antibody designed to attack highly cytotoxic T cells, a type of white blood cell that is present in the muscles of people with inclusion body myositis. These highly cytotoxic T cells may be responsible for much of the muscle damage in IBM. The treatment is selective, destroying most of the highly cytotoxic T cells without causing significant harm to other types of T cells or other white blood cells.

ABC008 clinical trial information at



Sirolimus, also known as rapamycin, is an established, FDA-approved drug that has been used for many years to prevent kidney transplant rejection. A Phase IIb clinical trial of sirolimus in inclusion body myositis was completed in France, and results were reported in 2017. The results were promising enough to spur interest in further study. A Phase III trial is now underway in Australia and the United States.

Cure IBM Blog article on Rapamycin and Inclusion Body Myositis.

Sirolimus clinical trial information at



The clinical trial of Arimoclomol, a drug which helps with protein folding and clumping, did not demonstrate a benefit in inclusion body myositis.

A Randomized, Double-Blind, Placebo-Controlled Study of Arimoclomol in Patients with Inclusion Body Myositis



by Kevin Dooley, MD

Revised July 7, 2023

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