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Study on Upadacitinib for Patients with Idiopathic Inflammatory Myopathies After Stopping IVIG

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a group of diseases known as idiopathic inflammatory myopathies. These diseases include polymyositis, dermatomyositis, antisynthetase syndrome, overlap myositis, and immune-mediated necrotizing myopathy. These conditions are characterized by inflammation of the muscles, which can lead to muscle weakness and other symptoms. The trial will evaluate the effectiveness and safety of a medication called Upadacitinib, also known by its code name ABT-494. This medication is taken orally in the form of prolonged-release tablets, which means it is designed to release the active ingredient slowly over time.

The purpose of the study is to assess whether patients can maintain stable disease activity without the need for intravenous immunoglobulins (IVIG), a common treatment for these conditions. Participants in the study will be randomly assigned to receive either Upadacitinib or a placebo, which is a substance with no active medication. The study will last for a period of 20 weeks, during which the participants' health and disease activity will be closely monitored. The main goal is to see if patients can remain stable without IVIG by the 16th week of the study.

Throughout the trial, various aspects of the participants' health will be evaluated, including muscle strength, quality of life, and any side effects experienced. The study will also compare the time it takes for symptoms to worsen between those taking Upadacitinib and those taking the placebo. This research aims to provide valuable insights into the potential of Upadacitinib as a treatment option for individuals with idiopathic inflammatory myopathies, potentially reducing the need for IVIG and improving overall management of these conditions.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Randomization
Upon joining the study, participants are randomly assigned to one of two groups. One group receives Upadacitinib 30 mg, and the other group receives a placebo. The placebo consists of gelatin capsules filled with maltodextrin.
The assignment is double-blind, meaning neither the participants nor the researchers know who is receiving the actual medication or the placebo.
Step 2
Medication administration
Participants take the assigned medication orally once daily. The medication is in the form of a prolonged-release tablet, which means it releases the active substance slowly over time.
The duration of this phase is up to 16 weeks, during which the participants continue their regular background treatment for idiopathic inflammatory myopathies, including any immunosuppressive drugs or antimalarials they were already taking.
Step 3
Monitoring and assessment
Throughout the trial, participants are monitored for any changes in their condition. This includes regular assessments of muscle strength, disease activity, and overall health.
The primary goal is to determine if participants can maintain stable disease activity without the need for intravenous immunoglobulins (IVIG) by week 16.
Step 4
Evaluation of outcomes
At week 16, the main outcome is assessed: the proportion of participants who achieve stable disease activity without IVIG.
Secondary outcomes are also evaluated, including differences in muscle strength, patient-reported outcomes, and any adverse events experienced by participants.
Step 5
Follow-up
Participants continue to be monitored until week 20 to assess the long-term effects of the treatment and any differences between the Upadacitinib and placebo groups.
The study aims to gather comprehensive data on the safety and efficacy of the treatment over this extended period.

Who can join the trial?

8 criteria

Provide written informed consent, which means you agree to participate in the study after understanding all the details.
Be a female or male aged between 18 and 65 years at the time of signing the consent form.
Have a clinical diagnosis of **idiopathic inflammatory myopathies**. This includes conditions like **Polymyositis**, **Dermatomyositis**, **Antisynthetase Syndrome**, **overlap Myositis**, and **Immune-mediating necrotising myopathy**. These are types of muscle diseases that cause inflammation and weakness.
Be receiving **IVIG** (intravenous immunoglobulin) at a stable dose and schedule for at least 12 weeks before the study starts. IVIG is a treatment made from donated blood that helps people with immune system problems.
Have stable disease activity, meaning your condition has not changed much, according to your doctor's judgment, for at least 3 months.
Be on a stable dose and schedule of allowed background treatments for your condition, such as **immunosuppressive drugs** (medications that reduce the activity of your immune system), **antimalarials** (drugs used to prevent or treat malaria), or **corticosteroids** (a type of steroid medication) up to 10 mg per day of prednisone equivalent, for at least 12 weeks before joining the study.
Be willing and able to understand and follow the study procedures.
If you are a female participant, agree to use effective birth control methods unless you are not able to have children. This includes women who are infertile due to surgical procedures like hysterectomy (removal of the uterus), bilateral oophorectomy (removal of both ovaries), or tubal ligation (blocking of the fallopian tubes), women aged 55 or older who are not on hormone therapy and have had at least 6 months of no menstrual periods, women aged 55 or older diagnosed with menopause, or women who have not had a menstrual period for more than 12 months.

Who cannot join the trial?

4 criteria

Patients with certain muscle diseases like Polymyositis, Dermatomyositis, Antisynthetase Syndrome, overlap Myositis, and Immune-mediating necrotising myopathy cannot participate. These are conditions where the immune system attacks the muscles, causing weakness and inflammation.
Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
Both male and female patients are eligible, but those who do not meet other specific criteria may be excluded.
Patients who are considered part of a vulnerable population, which means they might need special protection or care, are not included in this study.

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Medical University Of Vienna
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Vienna, Austria

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Investigated drugs

Upadacitinib is a medication being studied for its effectiveness and safety in patients with idiopathic inflammatory myopathies. It is being tested to see if it can help maintain stable disease activity in patients who have stopped using intravenous immunoglobulins (IVIG). The trial aims to determine if patients can remain stable without IVIG when taking Upadacitinib.

Upadacitinib

What is already known about the treatment

Upadacitinib – Upadacitinib is administered orally in the form of tablets. It is currently being studied in clinical trials for its effectiveness and safety in treating idiopathic inflammatory myopathies, such as polymyositis and dermatomyositis. The medication works by inhibiting Janus kinase (JAK) enzymes, which play a role in the inflammatory process, thereby reducing inflammation and immune response. Upadacitinib is classified as a JAK inhibitor and is being evaluated for its potential to maintain stable disease activity without the need for intravenous immunoglobulins.

Investigated diseases

Polymyositis
Polymyositis is an inflammatory disease that causes muscle weakness, primarily affecting the muscles closest to the trunk of the body. It progresses gradually, leading to difficulties in climbing stairs, lifting objects, or reaching overhead. The condition can also affect the muscles involved in swallowing and breathing. It is more common in adults and can occur alongside other autoimmune diseases.
Dermatomyositis
Dermatomyositis is characterized by muscle weakness and a distinctive skin rash. The rash often appears on the face, knuckles, and other areas exposed to sunlight. Muscle weakness typically affects the muscles closest to the trunk and can develop over weeks or months. This condition can also involve joint pain, lung problems, and difficulty swallowing.
Antisynthetase Syndrome
Antisynthetase Syndrome is an autoimmune condition that involves muscle inflammation, lung disease, and arthritis. It is associated with the presence of specific autoantibodies in the blood. The disease can cause muscle weakness, joint pain, and interstitial lung disease, which affects breathing. Symptoms may vary widely among individuals.
Overlap Myositis
Overlap Myositis is a condition where features of myositis occur alongside other autoimmune diseases, such as lupus or scleroderma. It involves muscle inflammation and weakness, often affecting the muscles closest to the trunk. The progression can vary, with some individuals experiencing more severe symptoms than others. It may also involve skin rashes and joint pain.
Immune-Mediated Necrotizing Myopathy
Immune-Mediated Necrotizing Myopathy is a rare condition characterized by severe muscle weakness and muscle cell death. It progresses rapidly and can lead to significant muscle damage. The condition is associated with specific autoantibodies and can occur in conjunction with other autoimmune diseases. It primarily affects the muscles closest to the trunk, making everyday activities challenging.