NCT04966884

The Efficacy and Safety of JAK Inhibitor in the Treatment of Anti-MDA5 Antibody-positive Dermatomyositis Patients

Status unknown Phase 4 Last updated 19 July 2021

What this trial tests

Phase 4 trial testing JAK Inhibitor in Dermatomyositis, Adult Type in 20 participants. Status unknown.

Timeline

2 April 2020

Primary endpoint
30 December 2021

30 December 2021

Quick facts

Lead sponsor	First Affiliated Hospital Xi'an Jiaotong University
Phase	Phase 4
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	20
Start date	2 April 2020
Primary completion	30 December 2021
Estimated completion	30 December 2021
Sites	1 location across China

Drugs / interventions tested

• JAK Inhibitor — full drug profile →

Conditions studied

• Dermatomyositis, Adult Type — all drugs for Dermatomyositis, Adult Type →

Sponsor

First Affiliated Hospital Xi'an Jiaotong University

Who can join

Adults 18 to 70, any sex, with Dermatomyositis, Adult Type. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Anti-melanoma differentiation-associated gene5 (Anti-MDA5) antibody positive Dermatomyositis (DM) is a subtype of DM that is more frequent in East Asia, which is often exhibit skin lesion, clinically amyopathic and interstitial lung disease. About 42%-100% of patients with Anti-MDA5+ DM develop rapidly progressive interstitial lung disease (RPILD) and result in respiratory failure. The mortality is as high as 40% within 6 months. In addition, not every patient with Anti-MDA5+ DM respond to traditional treatment strategy and most of the patients are resistant to immunosuppressive therapy including a combination of high dose glucocorticoids (GCs) and immunosuppressants such as cyclosporine, tacrolimus, or cyclophosphamide. However, RP-ILD is still the main cause of death due to fatal respiratory failure. Therefore, treatment of Anti-MDA5+ DM patients is challenging.Blocking multiple cytokines may become a new target for the treatment of this disease.Jakinibs is a Janus kinase (JAK) inhibitor that blocks a variety of cytokines, such as type I and type II interferon. Few studies have reported a positive response to JAK inhibitor for Anti-MDA5+ DM. Kazuhiro et al. reported in 2018 that JAK inhibitor tofacitinib may be an effective treatment option for high risk amyopathic dermatomyositis (ADM) -ILD patients after failure of conventional treatment, but the number of cases is too small. And a recent paper showed that great efficacy of tofacitinib for the improvement of survival of anti-MDA5-positive early-stage ADM-ILD patients.The aim of this study is to evaluate the efficacy and safety of JAK inhibitors in the treatment of anti-MDA5+ DM patients, and to evaluate the effect of JAK inhibitors on B cells of these patients, so as to provide a new target and theoretical basis for the treatment of anti-MDA5+ DM.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Treatment approach to connective tissue disease-associated interstitial lung disease.
   Wilson TM, Solomon JJ, Demoruelle MK. · · 2022 · cited 13× · PMID 35662004 · DOI 10.1016/j.coph.2022.102245
2. Past, Present, and Future in Dermatomyositis Therapeutics.
   Chung MP, Paik JJ. · · 2022 · cited 9× · PMID 38650607 · DOI 10.1007/s40674-022-00193-6
3. Clinical pearls and promising therapies in myositis.
   Connolly CM, Paik JJ. · · 2023 · cited 8× · PMID 37158055 · DOI 10.1080/1744666x.2023.2212162
4. Pathogenesis and Treatment of T-Large Granular Lymphocytic Leukemia (T-LGLL) in the Setting of Rheumatic Disease.
   Couette N, Jarjour W, Brammer JE, Simon Meara A. · · 2022 · cited 5× · PMID 35747794 · DOI 10.3389/fonc.2022.854499
5. A glance into the future of myositis therapy.
   Chiapparoli I, Galluzzo C, Salvarani C, Pipitone N. · · 2022 · cited 4× · PMID 35634354 · DOI 10.1177/1759720x221100299