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Completed

NCT Number: NCT07662265

Tocilizumab in Refractory ASS-ILD

Refractory anti-synthetase syndrome-associated interstitial lung disease (ASyS-ILD) lacks targeted therapy. Interleukin-6 drives both inflammation and fibrosis. We evaluated the real-world efficacy and safety of tocilizumab, an IL-6 receptor antagonist.

This single-center retrospective cohort study included patients with refractory ASyS-ILD treated between January 2020 and July 2025. Tocilizumab recipients were compared with those receiving standard of care (SOC) immunosuppressants. Overlap weighting based on propensity scores was used to balance 11 baseline variables. The primary outcome was improvement in symptoms and HRCT findings at ≥3 months. Secondary outcomes included changes in biomarkers and pulmonary function, progression-free survival (PFS), and adverse events. Cox regression identified independent predictors of symptom progression. Generalized additive models (GAM) explored nonlinear relationships, and XGBoost-SHAP machine learning assessed variable importance.

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Key information

Conditions

Antisynthetase Syndrome Tocilizumab

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

China-Japan Friendship Hospital

Beijing, Beijing Municipality, 100000, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • age ≥18 years;
  • fulfillment of the 2017 EULAR/ACR classification criteria for ASyS;
  • HRCT-confirmed ILD;
  • refractory disease, defined as active disease despite prior treatment with glucocorticoids and at least one first-line immunosuppressant (including methotrexate, mycophenolate mofetil, azathioprine, cyclosporine, cyclophosphamide, baricitinib, or upadacitinib);
  • availability of baseline and follow-up clinical data at ≥3 months.

Exclusion criteria

  • pregnancy or lactation;
  • pre-existing psychiatric disorders that would preclude study participation;
  • prior lung transplantation;
  • missing key outcome data;
  • Patients with Anti-MDA5 dermatomyositis.

Treatment and study plan

Tociliuzumab

Drug

Tocilizumab, a humanized monoclonal antibody against the IL-6 receptor, is approved for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, and giant cell arteritis. In patients with rheumatoid arthritis-associated ILD, tocilizumab has been shown to reduce KL-6 levels, a biomarker of alveolar epithelial injury. However, evidence for tocilizumab in ASyS-ILD is limited to isolated case reports, and no systematic evaluation of its efficacy and safety in this specific population has been published.

Primary outcomes

  1. Disease progression

    Time frame: From the start of tocilizumab treatment or second-line treatment to disease progression, the longest possible duration is until October 31, 2025.

    Disease progression is classified data, which included the number of patients with symptoms progression（Symptom progression was defined as worsening of dyspnea, cough or rash requiring escalation of therapy or hospitalization） or with the worsening of pattern demonstrated on CT scans (defined as an increase in the extent of reticulation, traction bronchiectasis, or honeycombing on follow-up HRCT ) or the number of the patients with FVC% decline >10% or with DLCO% decline >15%.

Secondary outcomes

  1. serum test of CK

    Time frame: From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

    The level of CK (U/L) in patients' serum

  2. Serum test of ferritin

    Time frame: From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

    The value of the ferritin (ng/ml) with serum test.

  3. Serum test of IL6

    Time frame: From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

    The value of the IL6 (pg/ml) with serum test.

  4. Serum test of LDH

    Time frame: From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

    The value of the LDH (U/L) with serum test.

  5. Serum test of CRP

    Time frame: From the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

    The value of the CRP (mg/L) with serum test.

  6. PFS

    Time frame: The time from the start of tocilizumab treatment or the first disease progression after second-line treatment, up to October 31, 2025.

    progression-free survival (PFS), defined as the time (months) from second-line treatment initiation to symptom progression, CT progression, lung function decline, or death from any cause, whichever occurred first;

  7. DLCO% predicted

    Time frame: Before the treatment with tocilizumab or the second-line treatment, and from the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

    The DLCO% predicted of thre patients

  8. FVC % predicted

    Time frame: Before the treatment with tocilizumab or the second-line treatment, and from the start of tocilizumab treatment or the start of second-line treatment, within 3 months (within 2 weeks before or after), up to October 31, 2025.

    FVC % predicted of the patient

Sponsors and collaborators

Lead sponsor

Hu Yinan

Other

Registry information

Official study title

Tocilizumab for Refractory Anti-Synthetase Syndrome-Associated Interstitial Lung Disease: A Real-World Retrospective Cohort Study

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Jun 23, 2026
Registry last updated
Jun 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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