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NEw Clinical Endpoints in primary Sjögren’s Syndrome: an Interventional Trial based on stratifYing patients

Descripción del proyecto

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Biomarcadores novedosos para estratificar pacientes con síndrome de Sjögren primario

El síndrome de Sjögren primario (SSp) es una enfermedad autoinmunitaria sistémica que presenta manifestaciones clínicas heterogéneas y trastornos biológicos. Afecta principalmente a las glándulas enxocrinas, por lo que provoca sequedad en ojos y boca, pero también puede causar enfermedad pulmonar o renal. El impacto multiorgánico del SSp ha obstaculizado el descubrimiento de fármacos, de manera que solo se ha autorizado un fármaco en los últimos veinte años. Los científicos del proyecto financiado con fondos europeos NECESSITY ayudarán en el descubrimiento de un fármaco determinando criterios de evaluación clínica sensibles para su uso en ensayos clínicos futuros que incluyan a pacientes con SSp. A través de un ensayo clínico, evaluarán estos criterios de evaluación junto con biomarcadores discriminativos para la estratificación de pacientes con SSp.

Objetivo

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.

Ámbito científico

Palabras clave

Programa(s)

Tema(s)

Convocatoria de propuestas

H2020-JTI-IMI2-2017-12-two-stage

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Régimen de financiación

RIA - Research and Innovation action

Coordinador

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
Aportación neta de la UEn
€ 575 416,85
Dirección
RUE DE TOLBIAC 101
75654 Paris
Francia

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Región
Ile-de-France Ile-de-France Paris
Tipo de actividad
Research Organisations
Enlaces
Coste total
€ 932 235,61

Participantes (28)

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Última actualización: 21 Mayo 2024

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Permalink: https://cordis.europa.eu/project/id/806975/es

European Union, 2024