Scleroderma, SystemicPhase 3NCT02597933
A Trial to Compare Nintedanib With Placebo for Patients With Scleroderma Related Lung Fibrosis
A Phase 3 study testing Nintedanib in people with Scleroderma, Systemic, run by Boehringer Ingelheim at 195 sites in 32 countries.
Trial summary
Systemic Sclerosis (SSc) is a devastating disease of unknown etiology. Patients suffer from multiple organ fibrosis whereas lung fibrosis (interstitial lung disease, ILD) is one of the main driver for mortality. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Scleroderma, Systemic
- What is being tested?
- Nintedanib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 580 participants
- Where
- 195 sitesin 32 countries
- Study dates
- 12 Nov 2015to 28 Nov 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age >= 18 years
- 2013 American College of Rheumatology (ACR) / EULAR classification criteria for SSc fulfilled
- SSc disease onset (defined by first non-Raynaud symptom) within 7 years
- SSc related Interstitial Lung Disease confirmed by High Resolution Computer Tomography (HRCT); Extent of fibrotic disease in the lung >= 10%
- FVC >= 40% of predicted normal
- Carbon Monoxide Diffusion Capacity (DLCO) 30% to 89% of predicted normal
Who cannot join
- Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT) >1.5 x ULN
- Bilirubin >1.5 x ULN
- Creatinine clearance <30 mL/min
- Airway obstruction (pre-bronchodilator Forced Expiratory Volume in 1 second (FEV1)/FVC <0.7)
- Other clinically significant pulmonary abnormalities
- Significant Pulmonary Hypertension (PH)
Show all 17Show fewer
- Cardiovascular diseases
- More than 3 digital fingertip ulcers or a history of severe digital necrosis requiring hospitalization or severe other ulcers
- Bleeding risk (such as predisposition to bleeding, fibrinolysis, full-dose anticoagulation, high dose antiplatelet therapy, history of hemorrhagic central nervous system (CNS) event within last year
- international normalised ratio (INR) >2, prolongation of prothrombin time (PT) and partial thromboplastin time (PTT) by >1.5 x ULN)
- History of thrombotic event within last year
- Clinical signs of malabsorption or needing parenteral nutrition
- Previous treatment with nintedanib or pirfenidone
- Treatment with prednisone >10 mg/day, azathioprine, hydroxychloroquine, colchicine, D-penicillamine, sulfasalazine, cyclophosphamide, rituximab, tocilizumab, abatacept, leflunomide, tacrolimus, newer anti-arthritic treatments like tofacitinib and cyclosporine A, potassium para-aminobenzoate
- Unstable background therapy with either mycophenolate mofetil or methotrexate
- Previous or planned hematopoietic stem cell transplantation
- Patients with underlying chronic liver disease (Child Pugh A, B, C hepatic impairment)
Trial locations and contacts
195 sites in 32 countries.
195 sites in 32 countries
United States44 sites
University of Michigan Health System
Ann Arbor, Michigan, 48109
The Emory Clinic
Atlanta, Georgia, 30322
University of Colorado Denver
Aurora, Colorado, 80045
Johns Hopkins Hospital
Baltimore, Maryland, 21224
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Boston Medical Center
Boston, Massachusetts, 02118
Medical University of South Carolina
Charleston, South Carolina, 29425
The Lung Research Center, LLC
Chesterfield, Missouri, 63017
Northwestern University
Chicago, Illinois, 60611
University of Cincinnati Health
Cincinnati, Ohio, 45219
Cleveland Clinic
Cleveland, Ohio, 44195
University of South Carolina
Columbia, South Carolina, 29203
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Duke University Medical Center
Durham, North Carolina, 27710
Inova Fairfax Medical Campus
Falls Church, Virginia, 22042
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
The University Of Texas at Houston
Houston, Texas, 77030
University of Iowa
Iowa City, Iowa, 52242
University of Florida College of Medicine
Jacksonville, Florida, 32209
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California Los Angeles
Los Angeles, California, 90095
University of Louisville
Louisville, Kentucky, 40202
University of Miami
Miami, Florida, 33125
Froedtert and The Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
Vanderbilt Pulmonary Clinic
Nashville, Tennessee, 37232-5735
Yale University School of Medicine
New Haven, Connecticut, 06510
Tulane University Hospital and Clinic
New Orleans, Louisiana, 70112
Hospital for Special Surgery
New York, New York, 10021
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Columbia University Medical Center-New York Presbyterian Hospital
New York, New York, 10032
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
Temple University Hospital
Philadelphia, Pennsylvania, 19140
Mayo Clinic-Rochester
Rochester, Minnesota, 55905
University of California Davis
Sacramento, California, 95817
University of Utah Health Sciences Center
Salt Lake City, Utah, 84108
University of California San Francisco
San Francisco, California, 94143
Virginia Mason Medical Center
Seattle, Washington, 98101
University of Washington
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
Stanford University Medical Center
Stanford, California, 94305-5236
University of Toledo
Toledo, Ohio, 43614
Georgetown University
Washington D.C., District of Columbia, 20007
Japan20 sites
Tosei General Hospital
Aichi, Seto, 489-8642
Kurume University Hospital
Fukuoka, Kurume, 830-0011
Sapporo Medical University Hospital
Hokkaido, Sapporo, 060-8543
National Hospital Organization Himeji Medical Center
Hyogo, Himeji, 670-8520
Iwate Medical University Hospital
Iwate, Morioka, 020-8505
St. Marianna University School of Medicine Hospital
Kanagawa, Kawasaki, 216-8511
Kitasato University Hospital
Kanagawa, Sagamihara, 252-0375
Kanagawa Cardiovascular and Respiratory Center
Kanagawa, Yokohama, 236-0051
Kyoto University Hospital
Kyoto, Kyoto, 606-8507
Nagasaki University Hospital
Nagasaki, Nagasaki, 852-8501
Kindai University Hospital
Osaka, Osakasayama, 589-8511
National Hospital Organization Kinki-Chuo Chest Medical Center
Osaka, Sakai, 591-8555
Osaka Medical College Hospital
Osaka, Takatsuki, 569-8686
Saitama Medical University Hospital
Saitama, Iruma-gun, 350-0495
Hamamatsu University Hospital
Shizuoka, Hamamatsu, 431-3192
Tokushima University Hospital
Tokushima, Tokushima, 770-8503
Juntendo University Hospital
Tokyo, Bunkyo-Ku, 113-8431
Nippon Medical School Hospital
Tokyo, Bunkyo-Ku, 113-8603
Toho University Omori Medical Center
Tokyo, Ota-ku, 143-8541
Institute of Rheumatology Tokyo Women's Medical University
Tokyo, Shinjyuku-ku, 162-0054
India15 sites
St John's Medical College
Bangalore, 560 034
Ramaiah Medical College and Hospitals
Bangalore, 560054
Mazumdar Shaw Medical centre
Bangalore, 560099
Postgraduate Institute of Medical Education And Research
Chandigarh, 160012
Care Hospital
Hyderabad, 500034
Nizam's Institute of Medical Sciences
Hyderabad, 500082
Asthma Bhawan
Jaipur, 302039
P.D. Hinduja National Hospital
Mumbai, 400016
Getwell Hospital & Research Institute
Nagpur, 440012
All India Institute of Medical Science
New Delhi, 110029
Sir Gangaram Hospital
New Delhi, 110060
Jehangir Clinical Development Centre Pvt. Ltd.
Pune, 411 001
B.J. Medical College and Sasoon General Hospital
Pune, 411001
Inamdar Multispeciality Hospital
Pune, 411040
Christian Medical College
Vellore, 632 004
France13 sites
HOP Avicenne
Bobigny, 93009
HOP Louis Pradel
Bron, 69677
HOP Calmette
Lille, 59037
HOP Claude Huriez
Lille, 59037
HOP Arnaud de Villeneuve
Montpellier, 34295
HOP Hôtel-Dieu
Nantes, 44000
HOP Pasteur
Nice, 06001
HOP Cochin
Paris, 75014
HOP Bichat
Paris, 75018
HOP Pontchaillou
Rennes, 35033
HOP Charles Nicolle
Rouen, 76000
HOP Larrey
Toulouse, 31059
HOP Bretonneau
Tours, 37044
Germany13 sites
Kerckhoff-Klinik, Bad Nauheim
Bad Nauheim, 61231
Universitätsklinikum Köln (AöR)
Cologne, 50937
Klinik Donaustauf
Donaustauf, 93093
Universitätsklinikum Carl Gustav Carus Dresden
Dresden, 01307
Universitätsklinikum Erlangen
Erlangen, 91054
Universitätsmedizin Greifswald
Greifswald, 17475
Asklepios Klinik Altona
Hamburg, 22763
Medizinische Hochschule Hannover
Hanover, 30625
Thoraxklinik-Heidelberg gGmbH am Universitätsklinikum Heidelberg
Heidelberg, 69126
Universitätsklinikum Schleswig-Holstein, Campus Kiel
Kiel, 24105
Klinikum der Universität München - Campus Großhadern
München, 80336
Universitätsklinikum Münster
Münster, 48149
Universitätsklinikum Tübingen
Tübingen, 72076
China9 sites
Peking Union Medical College Hospital
Beijing, 100032
Beijing Chao-Yang Hospital
Beijing
Beijing Hospital
Beijing
First Hospital of Jilin University
Changchun
West China Hospital
Chengdu, 610041
The First Affiliated Hospital of Anhui Medical University
Hefei, 230022
Huashan Hospital, Fudan University
Shanghai, 200040
The First Hospital of Chinese Medical University
Shenyang
Zhuzhou Central Hospital
Zhuzhou, 412007
Spain8 sites
Hospital Vall d'Hebron
Barcelona, 08035
Hospital Santa Creu i Sant Pau
Barcelona, 08041
Hospital General Universitario Gregorio Marañón
Madrid, 28007
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitario Marqués de Valdecilla
Santander, 39008
Hospital Dr. Peset
Valencia, 46017
Hospital Politècnic La Fe
Valencia, 46026
Hospital Álvaro Cunqueiro
Vigo, 36312
Italy6 sites
Az. Ospedaliere Umberto I di Ancona
Ancona, 60126
Università degli Studi di Genova
Genova, 16132
A.O. San Gerardo di Monza
Monza, 20900
A.O Universitaria - Università degli Studi della Campania Luigi Vanvitelli
Naples, 80138
Università degli Studi Padova
Padova, 35128
Azienda Universitaria-Universita' La Sapienza
Roma, 00161
Portugal6 sites
Hospital Garcia de Orta, EPE
Almada, 2801-951
Hospital Fernando Fonseca, EPE
Amadora, 2720-276
CHUC - Centro Hospitalar e Universitário de Coimbra, EPE
Coimbra, 3000-075
ULSAM, EPE - Hospital Conde de Bertiandos
Ponte de Lima, 4990-041
Centro Hospitalar São João,EPE
Porto, 4200-319
Centro Hospitalar de Vila Nova de Gaia
Vila Nova de Gaia, 4434-502
Belgium5 sites
ULB Hopital Erasme
Brussels, 1070
Brussels - UNIV Saint-Luc
Brussels, 1200
UNIV UZ Gent
Ghent, 9000
UZ Leuven
Leuven, 3000
Centre Hospitalier Universitaire de Liège
Liège, 4000
United Kingdom5 sites
Glasgow Royal Infirmary
Glasgow, G4 0SF
Royal Free Hospital
London, NW3 2QG
Guy's Hospital
London, SE1 9RT
Royal Brompton Hospital
London, SW3 6NP
Salford Royal Hospital
Salford, M6 8HD
Australia4 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050
St Vincent's Hospital Melbourne
Fitzroy, Victoria, 3065
Liverpool Hospital
Sydney, New South Wales, 2170
Israel4 sites
Bnei Zion Medical Center, Haifa
Haifa, 31048
Rambam Medical Center
Haifa, 3109601
Rabin Medical Center Beilinson
Petah Tikva, 49100
Sourasky Medical Center
Tel Aviv, 64239
Malaysia4 sites
University Malaya Medical Centre
Kuala Lumpur, 59100
Hospital Selayang
Kuala Selangor, 68100
Hospital Pulau Pinang
Pulau Pinang, 10990
Hospital Tuanku Ja'afar
Seremban, 70300
Netherlands4 sites
VU Medisch Centrum
Amsterdam, 1081HV
Leids Universitair Medisch Centrum (LUMC)
Leiden, 2333 ZA
Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA
Erasmus Medisch Centrum
Rotterdam, 3015 CE
Poland4 sites
Dr.Biziel UnivHosp#2,Rheumat&Connec.Tissue Disease,Bydgoszcz
Bydgoszcz, 85168
Dobry Lekarz,Spec.Med.Clinics,Private Prac,Krakow
Krakow, 31011
EMED, Center of Medical Services,Private Prac,Rzeszow
Rzeszów, 35205
Indep.Pblic Clin.Hosp#1,Dermatol,Venereol&Allerg.dep,Wroclaw
Wroclaw, 50 368
Argentina3 sites
Hospital Militar Central Cirujano Mayor Dr. Cosme Argerich
Buenos Aires, C1426BOR
APRILLUS-Asistencia e Investigación
Ciudad Autonoma Buenos Aires, C1046AAQ
CEMER-Centro Medico De Enfermedades Respiratorias
Florida, B1602DQD
Canada3 sites
Saint Joseph's Healthcare
Hamilton, Ontario, L8N 4A6
HSCM
Montreal, Quebec, H4J 1C5
Mount Sinai Hospital
Toronto, Ontario, M5T 3L9
Thailand3 sites
Songklanagarind Hospital
Hat Yai, 90110
Srinagarind Hospital
Muang, 40002
Ramathibodi Hospital
Ratchathewi, 10400
Austria2 sites
LKH-Univ. Hospital Graz
Graz, 8036
Medical University of Innsbruck
Innsbruck, 6020
Chile2 sites
Hospital Clínico Reg. de Concepción "Dr. G. Grant Benavente"
Concepción, 4070038
Centro de Investigación del Maule
Talca, 3465586
Czechia2 sites
Institute of Rheumathology Prague
Prague, 12850
Thomayer Hospital
Prague, 14059
Denmark2 sites
Aarhus Universitets Hospital
Aarhus, 8000
Odense Universitetshospital
Odense, 5000 C
Finland2 sites
HYKS Keuhkosairauksien
Helsinki, 00290
TYKS, Keuhkosairauksien klinikka, Turku
Turku, 20520
Greece2 sites
General Hospital of Athens "Laiko"
Athens, 115 27
General Hospital of Athens "Laiko"
Athens, 11527
Ireland2 sites
Cork University Hospital
Cork
Mater Misericordiae University Hospital
Dublin
Norway2 sites
Oslo Universitetssykehus HF, Rikshospitalet
Oslo, N-0372
Universitetssykehuset Nord-Norge, Tromsø
Tromsø, N-9038
Switzerland2 sites
Kantonspital St. Gallen, Rheumatologie Department
Sankt Gallen, 9007
Universitätsspital Zürich
Zurich, 8091
Mexico1 site
Instituto Nacional de Enfermedades Respiratorias Ismael Cosío Villegas
Mexico City, 14080
Sweden1 site
Clinical Rheumatology Research Center Sahlgrenska
Gothenburg, 413 45
About this study
Official title: A Double Blind, Randomised, Placebo-controlled Trial Evaluating Efficacy and Safety of Oral Nintedanib Treatment for at Least 52 Weeks in Patients With Systemic Sclerosis Associated Interstitial Lung Disease (SSc-ILD)
Systemic Sclerosis (SSc) is a devastating disease of unknown etiology. Patients suffer from multiple organ fibrosis whereas lung fibrosis (interstitial lung disease, ILD) is one of the main driver for mortality. There is preclinical evidence for efficacy of nintedanib in SSc and associated ILD (SSc-ILD) and the anti-fibrotic efficacy of nintedanib was proven in idiopathic pulmonary fibrosis patients, who are presenting a similar pattern regarding lung fibrosis. Hence it is the purpose of the trial to confirm the efficacy and safety of nintedanib 150 mg bid in treating patients with SSc-ILD, compared with placebo. The trial will be conducted as a double blind, randomised, placebo-controlled trial with primary efficacy evaluation at week 52 and placebo-controlled treatment until last patient out (up to a maximum of 100 weeks). Respiratory function is globally accepted for assessment of treatment effects in patients with lung fibrosis. The chosen endpoint (Forced Vital Capacity (FVC) decline) is easy to obtain and is part of the usual examinations done in patients with SSc-ILD.
- Conditions
- Scleroderma, Systemic
- Being tested
- Nintedanib, Placebo
- Medical subject headings
- Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NintedanibDrug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Nintedanib | Experimental | patient receives capsules containing nintedanib twice a day Receives: Drug: Nintedanib |
| Placebo | Placebo comparator | patient receives capsules identical to those containing active drug Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual Rate of Decline in Forced Vital Capacity (FVC) Over 52 Weeks
Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test.
For this endpoint reported means represent the adjusted rate.
Measured: up to week (wk) 52 after the start of administration
Secondary outcomes (11)
Absolute Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52
This is the first key secondary endpoint.
The modified Rodnan Skin Score (mRSS) is an evaluation of the patient's skin thickness rated by clinical palpation using a 0 to 3 scale. The scale differentiates between 0 = normal skin, 1 = mild thickness, 2 = moderate thickness, and 3 = severe thickness with inability to pinch the skin into a fold.
The palpation is done for each of the 17 surface anatomic areas of the body: face, anterior chest, abdomen, fingers (right and left separately), forearms, upper arms, thighs, lower legs, dorsum of hands and feet. The sum of these individual values is defined as the total skin score. The mRSS has a range from 0 (no thickening) to 51 (severe thickening in all 17 areas). A high score corresponds to worse skin thickness.
Least square mean is actually the adjusted mean. Adjusted mean was based on all analysed patients in the model (not only patients with a baseline and measurement at Week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Saint George's Respiratory Questionnaire (SGRQ) Total Score at Week 52.
This is the second key secondary endpoint.
The Saint George's Respiratory Questionnaire measures the health status in patients with chronic airflow limitation. It consists of 2 parts that cover 3 domains: symptoms, activities, and impacts. The symptom domain relates to the effect, frequency and severity of respiratory symptoms. The activity domain relates to activities that cause or are limited by breathlessness. The impact domain evaluates a range of aspects concerned with social functioning and psychological disturbances resulting from airways disease. The scores of these domains range from 0 (no impairment) to 100 (worst possible). The calculated total score summarises the impact of the disease on overall health status. A high score corresponds to worse health.
Least square mean is actually the adjusted mean. Adjusted mean was based on all analysed patients in the model (not only patients with a baseline and measurement at Week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Annual Rate of Decline in FVC in Percentage (%) Predicted Over 52 Weeks
Annual rate of decline in FVC in percentage (%) predicted over 52 weeks.
For this endpoint reported means represent the adjusted rate.
Measured: up to 52 weeks after the start of administration
Absolute Change From Baseline in FVC in mL at Week 52
Absolute change from baseline in FVC in mL at Week 52. Least square mean is actually the adjusted mean. Adjusted mean was based on all analysed patients in the model (not only patients with a baseline and measurement at Week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Relative Change From Baseline [%] of mRSS at Week 52
Relative change from baseline [%] of mRSS at Week 52.
The modified Rodnan Skin Score (mRSS) is an evaluation of the patient's skin thickness rated by clinical palpation using a 0 to 3 scale. The scale differentiates between 0 = normal skin, 1 = mild thickness, 2 = moderate thickness, and 3 = severe thickness with inability to pinch the skin into a fold.
The palpation is done for each of the 17 surface anatomic areas of the body: face, anterior chest, abdomen, fingers (right and left separately), forearms, upper arms, thighs, lower legs, dorsum of hands and feet. The sum of these individual values is defined as the total skin score. The mRSS has a range from 0 (no thickening) to 51 (severe thickening in all 17 areas). A high score corresponds to worse skin thickness.
Least square mean is actually the adjusted mean. Adjusted mean was based on all analysed patients in the model (not only patients with a baseline and measurement at Week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Time to Death
Time to event analysis of patients with death. The number of observed patients with death are reported.
Measured: From date of first trial drug intake up to date of death or last contact date (ie., up to 100 weeks)
The Percentage (%) of Responder Based on Combined Response Index in Systemic Sclerosis (CRISS) at Week 52
The percentage (%) of responder based on Combined Response Index in Systemic Sclerosis (CRISS) at Week 52.
This is a composite endpoint, based on the mRSS, FVC percent predicted, HAQ-DI, patient's global impression of overall health Visual Analogue Scale (VAS) and physician's global impression of patient's overall health VAS, as well as the absence of significant worsening of interstitial lung disease, a new scleroderma renal crisis, left ventricular failure or pulmonary arterial hypertension.
The CRISS index score represents a probability of improvement and ranges between 0 and 1.
This is a 2 stage process to predict probability of improvement:
Step 1 - absence of major organ progression (SRC etc.) - score "0" Step 2 - predicted probability of improvement - (score "0 - 1")
Measured: Week 52
Absolute Change From Baseline in Carbon Monoxide Diffusion Capacity (DLco) in % Predicted at Week 52
Absolute change from baseline in Carbon Monoxide Diffusion Capacity (DLco) in % predicted at Week 52.
Least square mean is actually the adjusted mean. Adjusted mean is based on all analysed patients in the model (not only patients with a baseline and measurement at week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Digital Ulcer Net Burden at Week 52
Absolute change from baseline in digital ulcer net burden (defined as the number of new digital ulcers (DUs) plus the number of DUs that have been verified at any earlier assessment during the trial) at Week 52.
It is calculated at a visit by counting the total number of fingertips with ulcers (i.e. number of fingers with presence of digital ulcer ticked "Yes") at the corresponding visit
Least square mean is actually the adjusted mean. Adjusted mean is based on all analysed patients in the model (not only patients with a baseline and measurement at week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Health Assessment Questionnaire-Disability Index (HAQ-DI) Score at Week 52
Absolute change from baseline in Health Assessment Questionnaire-Disability Index (HAQ-DI) score at Week 52.
The HAQ-DI score is calculated as follows:
Each question is scored 0-3 (where 0= "without difficulty" \& 3= "unable to do"). There are 8 categories (Dressing \& Grooming, Arising, Eating, Walking, Hygiene, Reach, Grip, Activities), each including 2 or 3 questions. The score for each category corresponds to maximum question score within each category.
Finally, HAQ-DI score corresponds to sum of the sub-scores of all 8 categories divided by number of categories completed. Please note that if there are fewer than 6 categories with responses, then a score cannot be calculated.
The HAQ-DI score scale has 25 possible values (i.e., 0, 0.125, 0.250, 0.375 … 3). A high score corresponds to worse impairment.
Least square mean is actually the adjusted mean. Adjusted mean is based on all analysed patients in the model (not only patients with a baseline and measurement at week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Dyspnoea Score at Week 52
Absolute change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT) dyspnoea score at Week 52.
FACIT-Dyspnoea (Dyspnoea) 10 Item Short Form include a 4-point rating scale (no shortness of breath=0; mildly short of breath=1; moderately short of breath = 2; severely short of breath =3; or I did not do this in the past 7 days =4).
A raw score is calculated as: Sum individual item scores * 10 / number of items answered. Raw scores are then converted to scale scores using the table included in the FACIT Dyspnoea Scale Short Form Scoring Guideline. FACIT dyspnea scale score ranges between 0 and 75.9.
The FACIT-Dyspnea short forms are scored such that a high score represents high levels of dyspnea.
Least square mean is actually the adjusted mean. Adjusted mean is based on all analysed patients in the model (not only patients with a baseline and measurement at week 52).
Measured: Baseline and up to 52 weeks after the start of administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2015 (estimated)
- Study start12 Nov 2015
- Primary completion31 Oct 2018
- Study completion28 Nov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (16)Papers the sponsor has linked to this study.
- Other trials for Scleroderma, SystemicStudies of the same condition, open and under way.
- More trials run by Boehringer IngelheimThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 580 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boehringer Ingelheim
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Boehringer Ingelheim, Study chair, Boehringer Ingelheim
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02597933
- Sponsor's protocol ID
- 1199.214
- Other IDs
- 2015-000392-28, 2015
- Status last verified
- Nov 2019
- First submitted
- 8 Oct 2015
- Last updated
- 13 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Smith V, Denton CP, Herrick AL, Ittrich C, Alves M, Cutolo M. Nailfold capillaroscopy in patients with systemic sclerosis-associated interstitial lung disease: a substudy of the SENSCIS trial. RMD Open. 2025 Oct 17;11(4):e005704. doi: 10.1136/rmdopen-2025-005704.
PubMed 41107055Volkmann ER, Assassi S, Denton CP, Simonovska R, Sambevski S, Alves M, Bernstein EJ. Outcomes in Systemic Sclerosis-Associated Interstitial Lung Disease Based on Serological Profiles With a Focus on Anticentromere and Anti-RNA Polymerase III Antibodies. J Rheumatol. 2025 Sep 1;52(9):914-918. doi: 10.3899/jrheum.2024-1063.
PubMed 40233987Volkmann ER, McMahan ZH, Smith V, Jouneau S, Miede C, Alves M, Herrick AL; SENSCIS Trial Investigators. Risk of Malnutrition in Patients With Systemic Sclerosis-Associated Interstitial Lung Disease Treated With Nintedanib in the Randomized, Placebo-Controlled SENSCIS Trial. Arthritis Care Res (Hoboken). 2023 Dec;75(12):2501-2507. doi: 10.1002/acr.25176. Epub 2023 Aug 7.
PubMed 37357024Allanore Y, Khanna D, Smith V, Aringer M, Hoffmann-Vold AM, Kuwana M, Merkel PA, Stock C, Sambevski S, Denton CP; SENSCIS Trial Investigators. Effects of nintedanib in patients with limited cutaneous systemic sclerosis and interstitial lung disease. Rheumatology (Oxford). 2024 Mar 1;63(3):639-647. doi: 10.1093/rheumatology/kead280.
PubMed 37294870Denton CP, Goh NS, Humphries SM, Maher TM, Spiera R, Devaraj A, Ho L, Stock C, Erhardt E, Alves M, Wells AU. Extent of fibrosis and lung function decline in patients with systemic sclerosis and interstitial lung disease: data from the SENSCIS trial. Rheumatology (Oxford). 2023 May 2;62(5):1870-1876. doi: 10.1093/rheumatology/keac535.
PubMed 36111858Maher TM, Bourdin A, Volkmann ER, Vettori S, Distler JHW, Alves M, Stock C, Distler O. Decline in forced vital capacity in subjects with systemic sclerosis-associated interstitial lung disease in the SENSCIS trial compared with healthy reference subjects. Respir Res. 2022 Jul 5;23(1):178. doi: 10.1186/s12931-022-02095-6.
PubMed 35790961Kreuter M, Hoffmann-Vold AM, Matucci-Cerinic M, Saketkoo LA, Highland KB, Wilson H, Alves M, Erhardt E, Schoof N, Maher TM. Impact of lung function and baseline clinical characteristics on patient-reported outcome measures in systemic sclerosis-associated interstitial lung disease. Rheumatology (Oxford). 2023 Feb 6;62(SI):SI43-SI53. doi: 10.1093/rheumatology/keac325.
PubMed 35640959Volkmann ER, Kreuter M, Hoffmann-Vold AM, Wijsenbeek M, Smith V, Khanna D, Denton CP, Wuyts WA, Miede C, Alves M, Sambevski S, Allanore Y. Dyspnoea and cough in patients with systemic sclerosis-associated interstitial lung disease in the SENSCIS trial. Rheumatology (Oxford). 2022 Nov 2;61(11):4397-4408. doi: 10.1093/rheumatology/keac091.
PubMed 35150246Kreuter M, Del Galdo F, Miede C, Khanna D, Wuyts WA, Hummers LK, Alves M, Schoof N, Stock C, Allanore Y. Impact of lung function decline on time to hospitalisation events in systemic sclerosis-associated interstitial lung disease (SSc-ILD): a joint model analysis. Arthritis Res Ther. 2022 Jan 10;24(1):19. doi: 10.1186/s13075-021-02710-9.
PubMed 35012623Highland KB, Distler O, Kuwana M, Allanore Y, Assassi S, Azuma A, Bourdin A, Denton CP, Distler JHW, Hoffmann-Vold AM, Khanna D, Mayes MD, Raghu G, Vonk MC, Gahlemann M, Clerisme-Beaty E, Girard M, Stowasser S, Zoz D, Maher TM; SENSCIS trial investigators. Efficacy and safety of nintedanib in patients with systemic sclerosis-associated interstitial lung disease treated with mycophenolate: a subgroup analysis of the SENSCIS trial. Lancet Respir Med. 2021 Jan;9(1):96-106. doi: 10.1016/S2213-2600(20)30330-1.
PubMed 33412120Roennow A, Sauve M, Welling J, Riggs RJ, Kennedy AT, Galetti I, Brown E, Leite C, Gonzalez A, Portales Guiraud AP, Houyez F, Camp R, Gilbert A, Gahlemann M, Moros L, Luna Flores JL, Schmidt F, Sauter W, Finnern H. Collaboration between patient organisations and a clinical research sponsor in a rare disease condition: learnings from a community advisory board and best practice for future collaborations. BMJ Open. 2020 Dec 16;10(12):e039473. doi: 10.1136/bmjopen-2020-039473.
PubMed 33328257Azuma A, Chung L, Behera D, Chung M, Kondoh Y, Ogura T, Okamoto M, Swarnakar R, Zeng X, Zou H, Meng X, Gahlemann M, Alves M, Kuwana M; SENSCIS trial investigators. Efficacy and safety of nintedanib in Asian patients with systemic sclerosis-associated interstitial lung disease: Subgroup analysis of the SENSCIS trial. Respir Investig. 2021 Mar;59(2):252-259. doi: 10.1016/j.resinv.2020.10.005. Epub 2020 Nov 19.
PubMed 33223487Maher TM, Mayes MD, Kreuter M, Volkmann ER, Aringer M, Castellvi I, Cutolo M, Stock C, Schoof N, Alves M, Raghu G; SENSCIS Trial Investigators. Effect of Nintedanib on Lung Function in Patients With Systemic Sclerosis-Associated Interstitial Lung Disease: Further Analyses of a Randomized, Double-Blind, Placebo-Controlled Trial. Arthritis Rheumatol. 2021 Apr;73(4):671-676. doi: 10.1002/art.41576. Epub 2021 Mar 8.
PubMed 33142016Kuwana M, Ogura T, Makino S, Homma S, Kondoh Y, Saito A, Ugai H, Gahlemann M, Takehara K, Azuma A. Nintedanib in patients with systemic sclerosis-associated interstitial lung disease: A Japanese population analysis of the SENSCIS trial. Mod Rheumatol. 2021 Jan;31(1):141-150. doi: 10.1080/14397595.2020.1751402. Epub 2020 Apr 23.
PubMed 32243207Distler O, Highland KB, Gahlemann M, Azuma A, Fischer A, Mayes MD, Raghu G, Sauter W, Girard M, Alves M, Clerisme-Beaty E, Stowasser S, Tetzlaff K, Kuwana M, Maher TM; SENSCIS Trial Investigators. Nintedanib for Systemic Sclerosis-Associated Interstitial Lung Disease. N Engl J Med. 2019 Jun 27;380(26):2518-2528. doi: 10.1056/NEJMoa1903076. Epub 2019 May 20.
PubMed 31112379
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double Blind, Randomised, Placebo-controlled Trial Evaluating Efficacy and Safety of Oral Nintedanib Treatment for at Least 52 Weeks in Patients With Systemic Sclerosis Associated Interstitial Lung Disease (SSc-ILD)
- ClinicalTrials.gov ID
- NCT02597933
- Acronym / sponsor ID
- 1199.214
- Conditions
- Scleroderma, Systemic
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 580 participants (actual)
- Lead sponsor
- Boehringer IngelheimIndustry (pharma, biotech, devices)
- Study start
- 12 Nov 2015 (actual)
- Primary completion
- 31 Oct 2018 (actual)
- Study completion
- 28 Nov 2018 (actual)
- First posted
- 5 Nov 2015 (estimated)
- Results
- Posted 13 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Systemic Sclerosis (SSc) is a devastating disease of unknown etiology. Patients suffer from multiple organ fibrosis whereas lung fibrosis (interstitial lung disease, ILD) is one of the main driver for mortality. There is preclinical evidence for efficacy of nintedanib in SSc and associated ILD (SSc-ILD) and the anti-fibrotic efficacy of nintedanib was proven in idiopathic pulmonary fibrosis patients, who are presenting a similar pattern regarding lung fibrosis. Hence it is the purpose of the trial to confirm the efficacy and safety of nintedanib 150 mg bid in treating patients with SSc-ILD, compared with placebo. The trial will be conducted as a double blind, randomised, placebo-controlled trial with primary efficacy evaluation at week 52 and placebo-controlled treatment until last patient out (up to a maximum of 100 weeks). Respiratory function is globally accepted for assessment of treatment effects in patients with lung fibrosis. The chosen endpoint (Forced Vital Capacity (FVC) decline) is easy to obtain and is part of the usual examinations done in patients with SSc-ILD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Nintedanib | Experimental | Drug: Nintedanibpatient receives capsules containing nintedanib twice a day |
| Placebo | Placebo comparator | Drug: Placebopatient receives capsules identical to those containing active drug |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age >= 18 years
- 2013 American College of Rheumatology (ACR) / EULAR classification criteria for SSc fulfilled
- SSc disease onset (defined by first non-Raynaud symptom) within 7 years
- SSc related Interstitial Lung Disease confirmed by High Resolution Computer Tomography (HRCT); Extent of fibrotic disease in the lung >= 10%
- FVC >= 40% of predicted normal
Key exclusion criteria (17 listed)
- Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT) >1.5 x ULN
- Bilirubin >1.5 x ULN
- Creatinine clearance <30 mL/min
- Airway obstruction (pre-bronchodilator Forced Expiratory Volume in 1 second (FEV1)/FVC <0.7)
- Other clinically significant pulmonary abnormalities
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nintedanib | Drug | Nintedanib |
| Placebo | Drug | Placebo |
Primary outcome
Annual Rate of Decline in Forced Vital Capacity (FVC) Over 52 Weeks
Time frame: up to week (wk) 52 after the start of administration
Forced vital capacity (FVC) is the total amount of air exhaled during the lung function test. For this endpoint reported means represent the adjusted rate.
11 secondary outcomes
Absolute Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52
Time frame: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Saint George's Respiratory Questionnaire (SGRQ) Total Score at Week 52.
Time frame: Baseline and up to 52 weeks after the start of administration
Annual Rate of Decline in FVC in Percentage (%) Predicted Over 52 Weeks
Time frame: up to 52 weeks after the start of administration
Absolute Change From Baseline in FVC in mL at Week 52
Time frame: Baseline and up to 52 weeks after the start of administration
Relative Change From Baseline [%] of mRSS at Week 52
Time frame: Baseline and up to 52 weeks after the start of administration
Time to Death
Time frame: From date of first trial drug intake up to date of death or last contact date (ie., up to 100 weeks)
The Percentage (%) of Responder Based on Combined Response Index in Systemic Sclerosis (CRISS) at Week 52
Time frame: Week 52
Absolute Change From Baseline in Carbon Monoxide Diffusion Capacity (DLco) in % Predicted at Week 52
Time frame: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Digital Ulcer Net Burden at Week 52
Time frame: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Health Assessment Questionnaire-Disability Index (HAQ-DI) Score at Week 52
Time frame: Baseline and up to 52 weeks after the start of administration
Absolute Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Dyspnoea Score at Week 52
Time frame: Baseline and up to 52 weeks after the start of administration
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41107055Smith V, Denton CP, Herrick AL, Ittrich C, Alves M, Cutolo M. Nailfold capillaroscopy in patients with systemic sclerosis-associated interstitial lung disease: a substudy of the SENSCIS trial. RMD Open. 2025 Oct 17;11(4):e005704. doi: 10.1136/rmdopen-2025-005704.
- Derived from this study: PubMed 40233987Volkmann ER, Assassi S, Denton CP, Simonovska R, Sambevski S, Alves M, Bernstein EJ. Outcomes in Systemic Sclerosis-Associated Interstitial Lung Disease Based on Serological Profiles With a Focus on Anticentromere and Anti-RNA Polymerase III Antibodies. J Rheumatol. 2025 Sep 1;52(9):914-918. doi: 10.3899/jrheum.2024-1063.
- Derived from this study: PubMed 37357024Volkmann ER, McMahan ZH, Smith V, Jouneau S, Miede C, Alves M, Herrick AL; SENSCIS Trial Investigators. Risk of Malnutrition in Patients With Systemic Sclerosis-Associated Interstitial Lung Disease Treated With Nintedanib in the Randomized, Placebo-Controlled SENSCIS Trial. Arthritis Care Res (Hoboken). 2023 Dec;75(12):2501-2507. doi: 10.1002/acr.25176. Epub 2023 Aug 7.
- Derived from this study: PubMed 37294870Allanore Y, Khanna D, Smith V, Aringer M, Hoffmann-Vold AM, Kuwana M, Merkel PA, Stock C, Sambevski S, Denton CP; SENSCIS Trial Investigators. Effects of nintedanib in patients with limited cutaneous systemic sclerosis and interstitial lung disease. Rheumatology (Oxford). 2024 Mar 1;63(3):639-647. doi: 10.1093/rheumatology/kead280.
- Derived from this study: PubMed 36111858Denton CP, Goh NS, Humphries SM, Maher TM, Spiera R, Devaraj A, Ho L, Stock C, Erhardt E, Alves M, Wells AU. Extent of fibrosis and lung function decline in patients with systemic sclerosis and interstitial lung disease: data from the SENSCIS trial. Rheumatology (Oxford). 2023 May 2;62(5):1870-1876. doi: 10.1093/rheumatology/keac535.
- Derived from this study: PubMed 35790961Maher TM, Bourdin A, Volkmann ER, Vettori S, Distler JHW, Alves M, Stock C, Distler O. Decline in forced vital capacity in subjects with systemic sclerosis-associated interstitial lung disease in the SENSCIS trial compared with healthy reference subjects. Respir Res. 2022 Jul 5;23(1):178. doi: 10.1186/s12931-022-02095-6.
- Derived from this study: PubMed 35640959Kreuter M, Hoffmann-Vold AM, Matucci-Cerinic M, Saketkoo LA, Highland KB, Wilson H, Alves M, Erhardt E, Schoof N, Maher TM. Impact of lung function and baseline clinical characteristics on patient-reported outcome measures in systemic sclerosis-associated interstitial lung disease. Rheumatology (Oxford). 2023 Feb 6;62(SI):SI43-SI53. doi: 10.1093/rheumatology/keac325.
- Derived from this study: PubMed 35150246Volkmann ER, Kreuter M, Hoffmann-Vold AM, Wijsenbeek M, Smith V, Khanna D, Denton CP, Wuyts WA, Miede C, Alves M, Sambevski S, Allanore Y. Dyspnoea and cough in patients with systemic sclerosis-associated interstitial lung disease in the SENSCIS trial. Rheumatology (Oxford). 2022 Nov 2;61(11):4397-4408. doi: 10.1093/rheumatology/keac091.
- Derived from this study: PubMed 35012623Kreuter M, Del Galdo F, Miede C, Khanna D, Wuyts WA, Hummers LK, Alves M, Schoof N, Stock C, Allanore Y. Impact of lung function decline on time to hospitalisation events in systemic sclerosis-associated interstitial lung disease (SSc-ILD): a joint model analysis. Arthritis Res Ther. 2022 Jan 10;24(1):19. doi: 10.1186/s13075-021-02710-9.
- Derived from this study: PubMed 33412120Highland KB, Distler O, Kuwana M, Allanore Y, Assassi S, Azuma A, Bourdin A, Denton CP, Distler JHW, Hoffmann-Vold AM, Khanna D, Mayes MD, Raghu G, Vonk MC, Gahlemann M, Clerisme-Beaty E, Girard M, Stowasser S, Zoz D, Maher TM; SENSCIS trial investigators. Efficacy and safety of nintedanib in patients with systemic sclerosis-associated interstitial lung disease treated with mycophenolate: a subgroup analysis of the SENSCIS trial. Lancet Respir Med. 2021 Jan;9(1):96-106. doi: 10.1016/S2213-2600(20)30330-1.
- Derived from this study: PubMed 33328257Roennow A, Sauve M, Welling J, Riggs RJ, Kennedy AT, Galetti I, Brown E, Leite C, Gonzalez A, Portales Guiraud AP, Houyez F, Camp R, Gilbert A, Gahlemann M, Moros L, Luna Flores JL, Schmidt F, Sauter W, Finnern H. Collaboration between patient organisations and a clinical research sponsor in a rare disease condition: learnings from a community advisory board and best practice for future collaborations. BMJ Open. 2020 Dec 16;10(12):e039473. doi: 10.1136/bmjopen-2020-039473.
- Derived from this study: PubMed 33223487Azuma A, Chung L, Behera D, Chung M, Kondoh Y, Ogura T, Okamoto M, Swarnakar R, Zeng X, Zou H, Meng X, Gahlemann M, Alves M, Kuwana M; SENSCIS trial investigators. Efficacy and safety of nintedanib in Asian patients with systemic sclerosis-associated interstitial lung disease: Subgroup analysis of the SENSCIS trial. Respir Investig. 2021 Mar;59(2):252-259. doi: 10.1016/j.resinv.2020.10.005. Epub 2020 Nov 19.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Boehringer Ingelheim study chair
Boehringer Ingelheim
195 sites in 32 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Nintedanib in people with Scleroderma, Systemic, run by Boehringer Ingelheim at 195 sites in 32 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 195 sites in United States, Japan, India, France, Germany and 27 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Dec 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annual Rate of Decline in Forced Vital Capacity (FVC) Over 52 Weeks (time frame: up to week (wk) 52 after the start of administration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02597933, which also lists 16 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Dec 2019.