Clinical Study of MMF in Treatment of IIM-ILD and Its Effect on Peripheral Blood Treg Cells

NCT05129410 | Unknown Phase 4

• 1 other identifier: XJTU1AF2020LSK-194
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Interstitial lung disease (ILD) is a common pulmonary manifestation of idiopathic inflammatory myopathy (IIM). The overall 5-year mortality is 50%. The prognosis is poor and the treatment is challenging.At present, according to the consensus of IIM-ILD experts, glucocorticoids as first-line treatment are often used in high doses and have a variety of adverse reactions. Previous studies have shown that cyclophosphamide (CYC) is effective for IIM-ILD and tends to be used in rapidly progressive interstitial lung disease（RP-ILD）or refractory ILD. However, CYC is an alkylating agent with many toxic and side effects. It is prone to gonadal inhibition, infection, tumor, hemorrhagic cystitis and other risks. At present, Mycophenolate mofetil (MMF) has been widly used in the treatment of IIM, systemic lupus erythematosus (SLE), ANCA associated vasculitis (AAV). The observational research on MMF in the treatment of IIM-ILD shows that it can delay the progress of pulmonary fibrosis and can be used as the first-line treatment of IIM-ILD. Moreover, immune tolerance caused by defects in the number and/or quality of regulatory T cells (Treg) is considered to be a key source of autoimmune diseases. However, it is unclear whether MMF can improve the immune status of IIM-ILD by increasing Treg cells. The aim of this study was to evaluate the effect of MMF for IIM-ILD and its effcts on Treg through a prospective open single arm study, and provide a theoretical basis for the individualized treatment of IIM-ILD, which has important clinical significance.

Conditions

Idiopathic Inflammatory Myopathy; Interstitial Lung Disease

Keywords

Idiopathic inflammatory myopathy; interstitial lung disease; Mycophenolate Mofetil; Treg; Th17

Outcome Measures

Primary Outcomes (1)

• FVC % predicted

  Percentage of predicted FVC

  12 months

Secondary Outcomes (6)

• DLCO % predicted

  12 months

• Lung high resolution CT score

  12 months

• TDI

  12 months

• TIS

  12 months

• Overall survival rate

  12 months

Study Arms (1)

A single-arm open-label pilot observational study

EXPERIMENTAL

Patients were received prednisone(0.5mg-1mg/kg/day) and a combination with MMF (1.5g-2.0g/d).

Drug: Mycophenolate Mofetil

Interventions

Mycophenolate Mofetil DRUG

1. Prednisone 0.5-1.0 mg/kg/d, the dose was gradually reduced, and after 4 weeks, the dose was reduced by 5mg every two weeks, and then reduced to 10mg/d for 4-6 months after oral administration for 3 months, and then reduced to 7.5mg/d for maintenance therapy until 12 months; 2. MMF1.5g-2.0g/d

A single-arm open-label pilot observational study

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• meet the PM/DM diagnostic criteria according to Bohan-Peter
• consistent with ILD diagnosis
• predicted forced vital capacity (FVC) of at least 50%
• patients who did not use immunosuppress agents (including but not limited to cyclophosphamide, cyclosporine, leflunomide, azathioprine, tacrolimus, etc.) at the time of screening, or who had stopped taking drugs for ≥3 months.

You may not qualify if:

• Anti MDA5 antibody positive DM and necrotizing myopathy
• patients if they had other connective tissue diseases, an underlying cancer, a concomitant active infection.

Sponsors & Collaborators

• First Affiliated Hospital Xi'an Jiaotong University: lead

Study Sites (1)

Department of Rheumatology,the First Affiliated Hospital of Xi'an Jiaotong University

Xi'an, 710061, China

RECRUITING

Related Publications (4)

• Long K, Danoff SK. Interstitial Lung Disease in Polymyositis and Dermatomyositis. Clin Chest Med. 2019 Sep;40(3):561-572. doi: 10.1016/j.ccm.2019.05.004.

  PMID: 31376891 RESULT

• Nihtyanova SI, Brough GM, Black CM, Denton CP. Mycophenolate mofetil in diffuse cutaneous systemic sclerosis--a retrospective analysis. Rheumatology (Oxford). 2007 Mar;46(3):442-5. doi: 10.1093/rheumatology/kel244. Epub 2006 Aug 9.

  PMID: 16899504 RESULT

• Edge JC, Outland JD, Dempsey JR, Callen JP. Mycophenolate mofetil as an effective corticosteroid-sparing therapy for recalcitrant dermatomyositis. Arch Dermatol. 2006 Jan;142(1):65-9. doi: 10.1001/archderm.142.1.65.

  PMID: 16415388 RESULT

• Antiga E, Kretz CC, Klembt R, Massi D, Ruland V, Stumpf C, Baroni G, Hartmann M, Hartschuh W, Volpi W, Del Bianco E, Enk A, Fabbri P, Krammer PH, Caproni M, Kuhn A. Characterization of regulatory T cells in patients with dermatomyositis. J Autoimmun. 2010 Dec;35(4):342-50. doi: 10.1016/j.jaut.2010.07.006. Epub 2010 Sep 16.

  PMID: 20843660 RESULT

MeSH Terms

Conditions

Myositis; Lung Diseases, Interstitial

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Lan He

  First Affiliated Hospital Xi'an Jiaotong University

  STUDY CHAIR

Central Study Contacts

Lan He

CONTACT

086-13809156236; Xajdhl87@mail.xjtu.edu.cn

Yanhua Wang

CONTACT

086-13571490573; 1367677985@qq.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: A single-arm open-label pilot observational study

Study Record Dates

• First Submitted: November 10, 2021
• First Posted: November 22, 2021
• Study Start: December 1, 2020
• Primary Completion: November 30, 2022
• Study Completion: November 30, 2023
• Last Updated: November 22, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)