ClinicalTrials.gov record NCT07377058

RCT of Tocilizumab for Anti-MDA5+DM

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Randomized, Double-Blind, Placebo-Controlled Clinical Study on the Efficacy and Safety of Tocilizumab in the Treatment of Anti-MDA5 Antibody-Positive Adult Dermatomyositis

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Age18 years old to 65 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This randomized, double-blind, placebo-controlled trial compares tocilizumab with a placebo in adults (18–65) who have anti‑MDA5 antibody–positive dermatomyositis. Participants receive tocilizumab or a placebo every two weeks for two months and attend clinic visits every two weeks for checks and tests. The study measures clinical improvement and, for those with lung involvement, changes in oxygenation at Week 16 and collects safety data.

What it is studying. Whether tocilizumab, given every two weeks, changes clinical symptoms and lung-related measures in adults with anti‑MDA5+ dermatomyositis, and what safety issues occur while on the drug compared with placebo.

What the researchers hope to learn. Measure the proportion of participants who reach a minimal improvement in Total Improvement Score (TIS≥20) at Week 16; assess change in PaO2/FiO2 from baseline at Week 16 for participants with interstitial lung disease (ILD); and collect safety and adverse event data while on tocilizumab.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The goal of this clinical trial is to learn if tocilizumab works to treat anti-MDA5+ dermatomyositis (anti-MDA5+DM) in adults. It will also learn about the safety of tocilizumab. The main questions it aims to answer are: Does tocilizumab improve patients' clinical symptoms? Does tocilizumab improve patients' respiratory failure? What medical problems do participants have when taking tocilizumab? Researchers will compare tocilizumab to a placebo (a look-alike substance that contains no drug) to see if tocilizumab works to treat patients with anti-MDA5+ DM. Participants will: Take tocilizumab or a placebo every two weeks for 2 months Visit the clinic once every 2 weeks for checkups and tests

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Standard medical treatment
Biological
  • Tocilizumab

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • All inclusion criteria must be met for eligibility:
  • Age ≥ 18 years and < 65 years; no gender restriction; total body weight ≥ 45 kg;
  • Diagnostic Criteria for Anti-MDA5-DM: Refer to the "2023 Chinese Expert Consensus on Diagnosis and Treatment of Anti-Melanoma Differentiation-Related Gene 5 Antibody-Positive Dermatomyositis in China." Dermatomyositis patients exhibiting one of the following manifestations-Gottron's papules, Gottron's rash, or erythema ab igne-along with positive serum anti-MDA5 antibodies, may be diagnosed with anti-MDA5-DM;
  • If patients have concomitant ILD, the following conditions must be met: i) Pulse oxygen saturation (SpO₂) ≥ 90% or PaO₂ ≥ 60 mmHg; ii) Pulmonary function tests showing forced vital capacity percentage of predicted (FVC%) ≥ 60% and carbon monoxide diffusion capacity percentage of predicted (DLco%) ≥ 40%; iii) High-resolution chest CT demonstrating pulmonary interstitial lesions involving < 50% of lung fields;
  • Patients must have received oral prednisone (< 1 mg/kg/day, or equivalent dose of other glucocorticoids) for ≥ 4 weeks prior to randomization;
  • Patients must have received a stable dose of a calcineurin inhibitor (CNI, such as cyclosporine or tacrolimus) for ≥4 weeks prior to randomization; if immunosuppressive therapy was discontinued prior to the screening visit, a washout period of at least 4 weeks is required;
  • Patients must receive prophylactic treatment with trimethoprim-sulfamethoxazole (TMP-SMZ, 400mg trimethoprim/80mg sulfamethoxazole) 1-2 tablets daily during treatment;
  • Women of childbearing potential must have a negative pregnancy test at study entry. If sexually active, they must agree to use effective contraception throughout the study period and must not intend to become pregnant during the study.
  • Patients voluntarily participate in this study and sign an informed consent form.

Exclusion criteria

  • Polymyositis, anti-synthetase syndrome, immune-mediated necrotizing myositis, or overlap myositis with other connective tissue diseases; 2. Patients with life-threatening complications, including but not limited to acute coronary syndrome (e.g., myocardial infarction, unstable angina) within 24 weeks prior to screening or any history of significant cerebrovascular disease; 3. Any of the following laboratory abnormalities at screening: white blood cell count <3.0×10⁹/L, neutrophil count <1.0×10⁹/L, lymphocyte count <0.5×10⁹/L, hemoglobin <90 g/L, platelet count <50×10⁹/L; severe hepatic impairment (ALT or AST ≥3 times ULN, total bilirubin ≥1.5 times ULN, excluding serum ALT or AST elevation due to dermatomyositis); severe renal impairment (creatinine clearance ≤45 mL/min); 4. Patients hospitalized for severe infection within 60 days prior to screening, or who received intravenous antibiotics (patients who used intravenous antibiotics must complete a five-half-life washout period and confirm absence of active infection before enrollment), but may receive empirical oral antibiotics or topical antibiotics; 5. Active tuberculosis infection that is untreated or inadequately treated; Latent tuberculosis infection (LTBI) requires at least 2 weeks of preventive antituberculosis therapy (including at least 2 antituberculosis drugs) prior to randomization, continuing through study completion. LTBI is defined as: Positive IGRA result (acceptable IGRA assays include: QFT-GIT, QFT-G, and T-spot® TB test); 6. Active viral hepatitis at screening: HBsAg-positive, HBeAg-positive, or HBV-DNA >10³ copies/L (HBV-DNA testing required if HBcAb-positive); HCVAb-positive; 7. Documented HIV infection, evidenced by positive serological test results or positive HIV serological test results at screening; 8. If the patient develops ILD-related clinical manifestations or progressive radiographic worsening within 4 weeks, RP-ILD should be considered. RP-ILD is defined as the presence of any one of the following four conditions within 1 month after the onset of respiratory symptoms: ① Acute and progressive worsening of dyspnea requiring hospitalization or supplemental oxygen; ② Decline in pulmonary function, manifested as a decrease in FVC% >10% with or without a decrease in DLco% >15%; ③ Increased interstitial abnormalities on chest HRCT scan >20%; ④ Decrease in arterial blood gas or partial pressure of oxygen >10 mmHg, indicating respiratory failure; and PaO₂/FiO₂≥200 mmHg.
  • Allergy to the active ingredient tocilizumab or any of its excipients; 10. Patients with sulfonamide allergy; 11. Patients unable to complete pulmonary function testing at baseline; 12. Patients receiving prednisone at a dose exceeding 2 mg/kg/day prior to screening; 13. Patients receiving intravenous immunoglobulin (IVIG) prior to screening must discontinue treatment for at least 30 days; 14. Patients who used one or more of the following medications within the specified time window prior to screening:
  • Rituximab within 6 months prior to screening;
  • JAK inhibitors within 2 weeks prior to screening;
  • Use of other biologics (including but not limited to anakinra, adalimumab, infliximab) or other immunosuppressive agents (including but not limited to methotrexate, azathioprine, mycophenolate mofetil) within 4 weeks prior to screening; 15. Patients with prior use of the study drug, other IL-6 inhibitors, or analogues; 16. Pregnant or lactating women, or women planning to become pregnant or initiate lactation; 17. History of malignant tumors within the past 5 years (excluding adequately treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or cervical carcinoma in situ with no evidence of recurrence within the preceding 5 years); 18. Other conditions deemed ineligible for study inclusion by the investigator.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

You would be randomly assigned (double‑blind) to receive tocilizumab or a matching placebo every two weeks for two months and attend clinic visits every two weeks for checks and tests. Visits include safety blood tests and respiratory assessments; people with ILD will have pulmonary function testing and oxygen/blood‑gas measurements, and HRCT was used for eligibility. Women of childbearing potential will have pregnancy testing and must use contraception; you must take daily trimethoprim‑sulfamethoxazole during treatment. The study includes baseline and follow‑up labs and imaging, with key outcomes assessed around Week 16.

How the study is structured. Adults are randomly assigned to one of two parallel groups: tocilizumab or placebo, in a double-blind (neither participants nor study staff know assignments) setup. Treatment is given every two weeks for two months, with clinic visits and tests every two weeks; key outcomes are assessed at Week 16.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on dermatomyositis

This trial is also listed on this site under polymyositis.

Clinical trials59 trials listed for dermatomyositisEvery trial the registry lists for dermatomyositis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in dermatomyositis researchLatest monthly update: September 2026. Includes: Upadacitinib case reports. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Nov 1, 2025
Primary completion
October 2026 (estimated)
Study completion
October 2026 (estimated)
Target enrollment
110 participants (estimated)
Last updated on registry
Jan 29, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Peking Union Medical College Hospital
Principal investigator
Wang Qian, Prof
Peking Union Medical College Hospital

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
inclusion criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
forced vital capacity
A breathing test that measures the total amount of air a person can blow out after a full breath in. It is often given as a percentage of what is expected for a person's age and size.
CT
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
prednisone
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
randomization
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
stable dose
A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
immunosuppressive
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
screening visit
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
washout period
A period during which a participant stops a previous treatment before starting the study treatment, so the earlier treatment does not affect the results.
Women of childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
neutrophil count
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
ULN
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
half-life
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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