About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age of 21 years or above;
Diagnosis of myositis associated rapidly progressive interstitial lung disease
Disqualifiers
Age of less than 21 year old;
Trial population
Patients currently undergoing treatment with combined immunosuppressive treatment for myositis-associated rapidly progressive interstitial lung disease
Trial design
Case-only
Prospective
Treatments tested in this trial
Immunosuppressant Regimen
DrugCombination 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
Treatment groups
Trial outcomes
Primary outcomes
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)
Secondary outcomes
Change in clinical status through Modified Medical Research Council Dyspnea Scale
The Modified Medical Research Council (mMRC) Dyspnea Scale will be used to rate participants' functional status at start of treatment and at 6 months post treatment (0 to 4).
Change in clinical status through requirement of supplemental oxygen at 6 months
Proportion of participants who require oxygen supplementation at the start of treatment and 6 months post treatment (percentage of all participants).
Safety of treatment regimen in participants
Number of adverse infection events experienced by participants
Safety of treatment regimen in participants
Number of adverse haematological events leading to change of medication as per managing physician
Sponsors and contacts
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