Treatment Response in Immune-mediated Myositis Associated Rapidly-progressing Interstitial Lung Disease

NCT07775235 | Recruiting FDA Drug

• 1 other identifier: 2024/2307
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

Idiopathic inflammatory myopathies (IIM) are a group of autoimmune conditions characterized by inflammation of muscles with possible extra-muscular manifestations which can include skin and interstitial lung disease (ILD). IIM-associated ILD carries poor prognosis. Particular subtypes of IIM such as anti-melanoma differentiation-associated protein 5 positive (anti-MDA5+) dermatomyositis with ILD are most commonly associated with rapidly progressive-interstitial lung disease (RP-ILD). RP-ILD is defined as worsening dyspnoea on exertion, hypoxaemia, and presence of newly emerging or expanding ground glass opacities on radiographic or computed topography of chest imaging excluding drug or infectious cause. Particularly, patients with anti-MDA5+ dermatomyositis often have RP-ILD with high mortality of over 60% in the first six months of diagnosis. The mainstay of treatment is immunosuppression though there has been no highly efficacious therapy proven to date. Therefore, the overall goal is to improve patient outcomes in IIM-associated RP-ILD including those with anti-MDA5+ dermatomyositis through the development of better treatment regimens. The objective of this research study is to evaluate the efficacy and safety of a combined immunosuppressive regime in patients with IIM-associated RP-ILD. The investigators hypothesize that the simultaneous inhibition of particular targets in the innate and adaptive immune system will improve efficacy and patient survival. The approach involves a combination of four immunosuppressive medications targeting different pathways implicated in IIM associated ILD. If successful, this study could contribute significantly to improving clinical outcomes for patients with IIM-associated RP-ILD.

Conditions

Myositis; Interstitial Lung Disease

Outcome Measures

Primary Outcomes (1)

• All-cause mortality at 6 months from treatment initiation

  Proportion of total participants who demise from all cause till 6 months from treatment initiation (percentage of all participants)

  6 months

Secondary Outcomes (11)

• Change in clinical status through Modified Medical Research Council Dyspnea Scale

  6 months

• Change in clinical status through requirement of supplemental oxygen at 6 months

  6 months

• Safety of treatment regimen in participants

  6 months

• Safety of treatment regimen in participants

  6 months

• Safety of treatment regimen in participants

  6 months

Study Arms (1)

Treatment group

Patients with immune-mediated myositis-associated rapidly progressive interstitial lung disease undergoing treatment with combined immunosuppressive treatment

Drug: Immunosuppressant Regimen

Interventions

Immunosuppressant Regimen DRUG

Combination treatment regimen lasting 6 months with A) Steroids: Starting with Intravenous methylprednisolone (500mg once daily for three days) followed by tapering dose of prednisolone B) Rituximab (1000mg given at the start of treatment and 1000mg 2 weeks after the first dose) C) Tacrolimus D) Tofacitinib

Also known as: Tofacitinib, Rituximab, Tacrolimus, Prednisolone

Treatment group

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients currently undergoing treatment with combined immunosuppressive treatment for myositis-associated rapidly progressive interstitial lung disease

You may qualify if:

• Age of 21 years or above;
• Diagnosis of myositis associated rapidly progressive interstitial lung disease

You may not qualify if:

• Age of less than 21 year old;

Sponsors & Collaborators

• Singapore General Hospital: lead

Study Sites (1)

Singapore General Hospital

Singapore, Singapore

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Peripheral blood mononuclear cells will be collected from participants which will be used for analysis of immune cell profiles and transcription patterns and changes as participants undergo treatment. Serum and plasma samples will be collected from participants to analyze cytokine patterns and changes as participants undergo treatment.

MeSH Terms

Conditions

Lung Diseases, Interstitial; Myositis

Interventions

tofacitinib; Rituximab; Tacrolimus; Prednisolone

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Macrolides; Lactones; Organic Chemicals; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Central Study Contacts

Mohamad Fadhli Bin Masri, MD PhD

CONTACT

6576 2609; mohamad.fadhli@mohh.com.sg

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 1 Year
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 23, 2026
• First Posted: August 20, 2026
• Study Start: December 26, 2024
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: August 20, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

SG (1)