Project description
Novel biomarkers for stratifying patients with primary Sjögren’s syndrome
Primary Sjögren’s syndrome (pSS) is a systemic autoimmune disease (AID) presenting with heterogeneous clinical manifestations and biological disturbances. It primarily affects exocrine glands leading to dryness of the eyes and the mouth, but it can also cause kidney or lung disease. The multi-organ impact of pSS has hampered drug discovery with only one licensed drug in the last 20 years. Scientists of the EU-funded NECESSITY project will assist in drug-discovery efforts by determining sensitive clinical endpoints, for use in future clinical trials involving patients with pSS. Through a clinical trial, they will evaluate these endpoints alongside discriminative biomarkers for the stratification of patients with pSS.
Objective
Primary Sjögren’s syndrome (pSS) is a systemic autoimmune disease (AID) involving 0.5 to 3/1000 persons. The disease affects exocrine glands leading to dryness of the eyes and the mouth and is associated with fatigue and limb pain. In 30% to 50% of the patients, systemic and extra-glandular manifestations may develop. The spectrum of extra-glandular manifestations in pSS is broad and includes vasculitis, peripheral neuropathy, synovitis, kidney involvement and interstitial lung disease. Moreover, pSS patients have a 10 to 20-fold higher risk of developing B cell lymphomas, conferring shorter lifetime expectancy to these patients.
Whereas 10 new targeted-immunomodulatory treatments have been marketed for rheumatoid arthritis in the past 20 years, only one drug has been licensed for other systemic AIDs, such as pSS and systemic erythematous lupus in the same period. There are several factors that may hamper the development of successful drugs for AID. Being multi-organ, these AIDs are considerably heterogeneous among individuals both in terms of clinical manifestations and biological disturbances, with, as a consequence, a great difficulty to set-up accurate composite clinical end-points sensitive to change and usable in clinical trials. In this project, our objectives are:
• To develop and assess sensitive clinical endpoints, for use in future clinical trials, able to evaluate response to drug treatments in patients with pSS with high disease burden and/or systemic involvement,
• To identify and evaluate discriminative biomarkers for stratification of pSS patients predictive of organ involvement and disease progression and thus available for inclusion in clinical trials,
• To set-up and perform an original multi-arm multi-stage clinical trial to validate the newly defined pSS endpoints and the identified biomarkers, by maximizing the chance of finding a difference between the placebo arm and the treated arm.
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RIA - Research and Innovation actionCoordinator
75654 Paris
France
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Participants (28)
Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
91190 Gif-Sur-Yvette
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B15 2TT Birmingham
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17177 Stockholm
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NE1 7RU Newcastle Upon Tyne
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67091 Strasbourg Cedex
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3584 CX Utrecht
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29238 BREST
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Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
29609 Brest
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41092 Sevilla
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10561 Athina
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75006 Paris
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75012 Paris
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75014 Paris
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Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
T12 YN60 Cork
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Participation ended
5020 Bergen
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53100 Siena
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The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
75001 PARIS 1er
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E1 4NS London
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33100 Udine
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75724 Paris
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4011 Stavanger
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9713 GZ Groningen
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08036 Barcelona
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Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.
08036 Barcelona
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4056 Basel
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TW89GS Brentford
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10154 New York
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92284 Courbevoie
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