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A study testing brepocitinib compared to placebo for adults with dermatomyositis to see if it works and is safe

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What is this trial about?

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

This study is looking at a disease called dermatomyositis, which is a condition that causes muscle weakness and skin rashes. The study will test a medicine called brepocitinib, which is also known by its code name PVT-2201. This medicine works by blocking certain enzymes in the body that can cause inflammation. Some people in the study will receive brepocitinib while others will receive placebo. The purpose of the study is to find out if brepocitinib can help improve the symptoms of dermatomyositis compared to placebo after 52 weeks of treatment.

People taking part in this study must be adults between 18 and 75 years old who have been diagnosed with dermatomyositis and have active muscle and skin disease. Participants should have received or be receiving treatment with corticosteroids, hydroxychloroquine, or other medicines that affect the immune system. The study will measure improvement using a scoring system that looks at different aspects of the disease, including muscle strength, skin condition, overall health as rated by both the doctor and the patient, ability to perform daily activities, and levels of certain enzymes in the blood that indicate muscle damage.

During the study, participants will take their assigned medicine by mouth in tablet form for up to 114 days. The study will track various measures of improvement at different time points, with the main assessment happening at week 52. These measures include changes in skin disease activity scores, the proportion of people who show moderate or major improvement, how quickly improvement occurs, and changes in the ability to carry out daily activities. The study will also look at whether participants can reduce their dose of prednisone or similar corticosteroid medicines while still showing improvement.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Treatment period begins

    At the start of the trial, you will be randomly assigned to receive either brepocitinib or placebo. A placebo is an inactive substance that looks like the actual medication but contains no active ingredient.

    Neither you nor your doctor will know which treatment you are receiving during this period. This is called a double-blind study design.

    You will receive the assigned treatment in the form of tablets that are taken by mouth.

  2. Step 2

    Taking the study medication

    You will take the study medication orally as tablets throughout the trial period.

    The medication contains brepocitinib tosilate as the active ingredient, or it may be a placebo tablet if you are assigned to the placebo group.

    Your doctor will provide specific instructions on how often to take the tablets and at what times of day.

  3. Step 3

    Regular clinic visits during the first month

    At Week 4, you will have a clinic visit where your doctor will assess changes in your skin condition.

    Your skin disease activity will be measured using a scoring system called CDASI Activity Score, which evaluates the severity of skin symptoms related to dermatomyositis.

  4. Step 4

    Ongoing treatment and monitoring

    You will continue taking the study medication as prescribed throughout the trial.

    During this period, you may continue receiving other medications you were taking before the trial, such as corticosteroids, hydroxychloroquine, or other medications that affect the immune system.

    Regular visits will be scheduled to monitor your condition and any effects of the treatment.

  5. Step 5

    Assessment at Week 48

    At Week 48, your doctor will evaluate your overall improvement and check the dose of any corticosteroid medication you are taking.

    If you are taking oral prednisone or an equivalent corticosteroid, the dose will be recorded to determine if it has been reduced to 2.5 milligrams per day or less.

  6. Step 6

    Final assessment at Week 52

    At Week 52, which marks the end of the main treatment period, you will undergo comprehensive assessments.

    Your overall improvement will be measured using the Total Improvement Score, which combines six different measures including assessments by your doctor, your own assessment of your condition, muscle strength testing, ability to perform daily activities, muscle enzyme levels in your blood, and muscle disease activity.

    Your skin condition will be assessed again using the CDASI Activity Score to measure changes from the beginning of the trial.

    Another combined measure called DMOMS will be calculated, which includes muscle strength, skin condition, and assessments from both you and your doctor.

    Your ability to perform daily activities will be evaluated using the HAQ Disability Index, which measures how easily you can complete everyday tasks.

    If you are taking corticosteroids, the dose will be checked again to see if it remains at 2.5 milligrams per day or less.

  7. Step 7

    Completion of the trial

    After the Week 52 visit, the main part of the trial will be complete.

    Your doctor will discuss the next steps in your care and any changes to your treatment plan.

Who can join the trial?

7 criteria

  • You must be between 18 and 75 years old when you sign the consent form to join the study
  • You must have a confirmed diagnosis of dermatomyositis, which is a condition that causes muscle weakness and skin rash, according to specific medical classification guidelines from 2017
  • You must have active muscle and skin disease at the time of screening and at the start of the study, meaning your symptoms are currently present
  • You must be currently taking or have previously taken corticosteroids (medications that reduce inflammation), hydroxychloroquine (a medication often used for inflammatory conditions), or at least one other medication that suppresses the immune system
  • Your body weight must be more than 40 kilograms but less than 130 kilograms
  • Your body mass index, which is a measure of body fat based on your height and weight, must be less than 40
  • Both men and women can participate in this study

Who cannot join the trial?

3 criteria

  • The source data does not provide specific exclusion criteria (reasons why a patient cannot participate in this study)
  • Without detailed exclusion criteria information, it is not possible to list the specific conditions or situations that would prevent participation in this clinical trial (a research study testing a new treatment)
  • Typically, exclusion criteria may include things like other serious health conditions, certain medications being taken, pregnancy or breastfeeding, recent vaccinations, or abnormal laboratory test results, but these specific details are not available in the provided information
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Investigated drugs

  • Brepocitinib

    is an oral medication being tested in this study to treat dermatomyositis, which is a rare disease that causes muscle weakness and skin rash. This medicine is taken by mouth and works by affecting the immune system to help reduce inflammation in the muscles and skin.

  • Placebo

    is an inactive substance that looks like the real medication but contains no active medicine. It is used in this study to compare whether brepocitinib works better than no treatment at all.

What is already known about the treatment

Brepocitinib – Brepocitinib is an oral medication currently being studied in clinical trials for the treatment of dermatomyositis, which is a rare condition that causes muscle weakness and skin rash. This drug works by blocking specific proteins in the body called tyrosine kinase 2 and Janus kinases, which are involved in causing inflammation and immune system overactivity. It belongs to a class of medications known as JAK inhibitors, which help reduce inflammation by interfering with the signals that trigger the immune system to attack healthy tissues. The medication is being tested in a phase 3 clinical trial to determine if it can safely and effectively improve symptoms in adults with dermatomyositis when taken by mouth.

Investigated diseases

Dermatomyositis – Dermatomyositis is a rare inflammatory disease that affects both the muscles and the skin. The condition causes muscle weakness, particularly in the muscles closest to the trunk of the body, such as those in the hips, thighs, shoulders, and neck. A distinctive skin rash typically appears, often affecting the face, eyelids, knuckles, elbows, knees, chest, and back. The muscle weakness tends to develop gradually over weeks to months, making it difficult to perform everyday activities like climbing stairs or lifting objects. The skin changes may appear before, after, or at the same time as the muscle symptoms. The disease progresses through periods of increased activity and remission, with symptoms varying in severity from person to person.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-500367-12-00Protocol codePVT-2201-301Estimated enrolment235 patientsSponsorPriovant Therapeutics Inc.

sourced from the EU Clinical Trials Register and site verification

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