Treatment and Prevention of Progression of Interstitial Lung Disease in Systemic Sclerosis

Prevention and Treatment of Interstitial Lung Disease in Systemic Sclerosis

Systemic sclerosis (SSc) is an orphan, multiorgan disease affecting the connective tissue of the skin and all internal organs. Interstitial lung disease is a frequent morbidity and mortality-driving manifestation in systemic sclerosis.

This observational trial (OT) is part of the collaborative project "DeSScipher", one out of five OTs to decipher the optimal management of systemic sclerosis. Aim of this observational try is to identify:

• The state of clinical practice in Europe for prevention and treatment of interstitial lung disease and its impact on lung function and disease progression
• The potential predictors and confounders for response to therapy

Study Overview

• Status: Unknown
• Conditions: Systemic Sclerosis; Interstitial Lung Diseases

Detailed Description

Patients are routinely evaluated every 3 months over a 12-months period by medical history, physical examination, pulmonary function tests, VAS lung score and SF-36, SHAQ. Also, their medication and possible medication changes will be recorded.

• Study Type: Observational
• Enrollment (Anticipated): 1372

Contacts and Locations

Study Locations

• France
  • Paris, France, 75014
    • Recruiting
    • Université Paris Descartes, Hôpital Cochin, Service de Rhumatologie A & INSERM 1016
    • Principal Investigator: Yannick Allanore, Prof.
• Germany
  • Bad Nauheim, Germany, 61231
    • Recruiting
    • Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology
    • Principal Investigator: Ulf Müller-Ladner, Prof.
    • Sub-Investigator: Ingo H. Tarner, Dr.
    • Sub-Investigator: Marc Frerix, Dr.
  • Berlin, Germany, 10117
    • Recruiting
    • Charité Universitätsmedizin Berlin, Charité Centrum 12 für Innere Medizin und Dermatologie, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie
    • Principal Investigator: Gabriela Riemekasten, Prof.
  • Hamburg, Germany, 22081
    • Recruiting
    • Centre for Pediatric Rheumatology, Klinikum Eilbek
    • Principal Investigator: Ivan Foeldvari, Dr.
• Hungary
  • Pecs, Hungary, H-7622
    • Recruiting
    • Pecsi Tudomanyegyetem - University of Pecs
    • Principal Investigator: Laszlo Czirjak, Prof.
• Italy
  • Firenze, Italy, 50139
    • Recruiting
    • University of Florence, Denothe Centre, Division of Rheumatology AOUC, Department of Biomedicine
    • Principal Investigator: Marco Matucci-Cerinic, Prof.
  • Napoli-Italia, Italy, 5-80131
    • Recruiting
    • Policlinico, Via Pansini
    • Principal Investigator: Gabriele Valentini, Prof
• Switzerland
  • Basel, Switzerland, CH 4012 Basel
    • Recruiting
    • Felix-Platter Spital, University of Basel
    • Principal Investigator: Ulrich Walker, Prof.
  • Zurich, Switzerland, 8006
    • Recruiting
    • University of Zurich, Department of Rheumatology
    • Principal Investigator: Oliver Distler, Prof.
• United Kingdom
  • Leeds, United Kingdom, LS9 7TF
    • Recruiting
    • The Universitiy of Leeds, Division of Rheumatic and Musculoskeletal Disease, St James's University Hospital
    • Principal Investigator: Francesco Del Galdo, Dr.
  • London, United Kingdom, NW3 2QG
    • Recruiting
    • Royal Free Hospital, University College London
    • Principal Investigator: Christopher Denton, Prof.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

The study population are adult and juvenile SSc patients from the EUSTAR cohort (MEDSonline database) and the jSScWG cohort

Description

Inclusion Criteria:

• Diagnosis fo SSc according to the ACR/EULAR criteria for adult or the PRES/ACR/EULAR criteria for juvenile SSc patients
• SSc patients with proven ILD (by X-ray or CT scan)
• Treatment with standard dosages according to current practice with (i) cyclophosphamide, (ii) azathioprine, (iii) mycophenolate mofetil, (iv) methotrexate, or (v) no therapy

Exclusion Criterion:

• Patients with previous exposure to silica or asbestos

Study Plan

How is the study designed?

Number of groups / cohorts

5

Cohorts and Interventions

Group / Cohort
cyclophosphamide; Patients receiving cyclophosphamide
azathioprine; Patients receiving azathioprine
mycophenolate mofetil; Patients receiving mycophenolate mofetil
methotrexate; Patients receiving methotrexate
no therapy; Patients receiving no immunosuppressive therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients with 10% decline in FVC	The proportion of patients with ILD progression as defined by a 10% decline in FVC within 1 year of therapy	1 year

Secondary Outcome Measures

Outcome Measure	Time Frame
The time to a 15% decline in DLCO or a drop <55% of predicted lung function	1 year
The mortality due to lung fibrosis	1 year
The need for oxygen support	1 year

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Identification of confounders	Parameters with impact on ILD progression independent of therapies, such as SSc subgroups, presence of antibodies, presence of vasculopathy, time to therapy initiation, degree of lung fibrosis defined by FVC values, age at disease onset and at treatment initiation, gender, co-morbidities and other confounders.	1 year
Evaluation of the incidence of drug-related adverse events		1 year
Evaluation of the incidence of withdrawal from treatment due to drug-related adverse events		1 year

Collaborators and Investigators

• Sponsor: Gabriela Riemekasten
• Collaborators: University of Pecs; Charite University, Berlin, Germany; University of Zurich; University College, London; University of Leeds; University of Campania "Luigi Vanvitelli"; University of Basel; University of Giessen; University of Florence; European Union; University of Paris 5 - Rene Descartes; Schoen Klinik Hamburg Eilbek
• Investigators: Study Chair: Ulf Müller-Ladner, Prof., Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology; Principal Investigator: Gabriela Riemekasten, Prof., Charité Universitätsmedizin Berlin, Charité Centrum 12 für Innere Medizin und Dermatologie, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie; Principal Investigator: Christopher Denton, Prof., Royal Free Hospital, University College London London

Study record dates

Study Major Dates

• Study Start: May 1, 2013
• Primary Completion (Anticipated): June 1, 2016
• Study Completion (Anticipated): June 1, 2016

Study Registration Dates

• First Submitted: May 14, 2013
• First Submitted That Met QC Criteria: May 16, 2013
• First Posted (Estimate): May 21, 2013

Study Record Updates

• Last Update Posted (Estimate): August 13, 2014
• Last Update Submitted That Met QC Criteria: August 12, 2014
• Last Verified: August 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Skin Diseases; Respiratory Tract Diseases; Connective Tissue Diseases; Sclerosis; Lung Diseases; Scleroderma, Systemic; Scleroderma, Diffuse; Lung Diseases, Interstitial
• Other Study ID Numbers: HEALTH-F5-2012-305495-OT3