Inicio
Proyectos y Estudios
Estudio abierto, multicéntrico, de dos cohortes y un solo grupo para evaluar la eficacia, la seguridad y la tolerabilidad, la farmacocinética y la farmacodinámica de emapalumab en niños y adultos con síndrome de activación de macrófagos (SAM) en la enfermedad de Still (incluida la artritis idiopática juvenil sistémica y la enfermedad de Still del adulto) o con SAM en el lupus eritematoso sistémico.

Estudio abierto, multicéntrico, de dos cohortes y un solo grupo para evaluar la eficacia, la seguridad y la tolerabilidad, la farmacocinética y la farmacodinámica de emapalumab en niños y adultos con síndrome de activación de macrófagos (SAM) en la enfermedad de Still (incluida la artritis idiopática juvenil sistémica y la enfermedad de Still del adulto) o con SAM en el lupus eritematoso sistémico.

Datos básicos

Protocolo:: NI-0501-14
EUDRACT:: 2021-001577-24
NCT:
Centro:: HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Año de incio:: 2021
Año de finalización:: 2022

ENSAYO CLÍNICO

Objetivos del proyecto

Objetivo Principal: Demostrar la eficacia de emapalumab en el tratamiento de los siguientes pacientes: • Cohorte 1: SAM en el contexto de la artritis idiopática juvenil sistémica (AIJs) y la enfermedad de Still del adulto (ESA). • Cohorte 2: SAM en el contexto del lupus eritematoso sistémico (LES) infantil y del adulto. Objetivos Secundarios: • Demostrar la eficacia de emapalumab con respecto a la reducción gradual de glucocorticoides (GC). • Evaluar el tiempo hasta el inicio de la respuesta al tratamiento con emapalumab. • Evaluar la eficacia de emapalumab con respecto a la respuesta global. • Evaluar la eficacia sostenida del tratamiento con emapalumab. • Evaluar la supervivencia del paciente después del tratamiento con emapalumab. • Evaluar la seguridad y tolerabilidad de emapalumab. • Evaluar los resultados notificados por el paciente en relación con el SAM en pacientes tratados con emapalumab. • Determinar el perfil FC de emapalumab. • Determinar el perfil FD de emapalumab. • Determinar la inmunogenicidad de emapalumab.

Documentos

No hay documentos

Participantes

María Pilar Calvillo Batlles

Investigador Colaborador
Berta López Montesinos

Investigador Colaborador
Francisco Menor Serrano

Investigador Colaborador
José Luis Poveda Andrés

Investigador Colaborador
María Isabel González Fernández

Investigador Colaborador
Maria Perez Fernandez

Técnico Apoyo Investigación
Joan Octavi Carreres Polo

Investigador Colaborador
Inmaculada Calvo Penadés

Investigador Principal
Beatriz Insa Albert

Investigador Colaborador
Juan Agustín Fornés Torres

Data Manager / Study Coordinator
Rosa Maria Pascual Costa

Investigador Colaborador
Miguel Martí Masanet

Investigador Colaborador
Lucia Lacruz Perez

Investigador Colaborador
Sonia Gines Cardenas

Investigador Colaborador
Africa Almendros Riaza

Investigador Colaborador
Fatima Tomas Medina

Investigador Colaborador
Oussama Bardouz Gouguasse

Investigador Colaborador
Silvia Maestro Huguet

Investigador Colaborador
Vanessa Quiles Fortuny

Investigador Colaborador
Maria Del Mar San Juan Tubilleja

Investigador Colaborador
Lorena Saiz Ferris

Investigador Colaborador

Ver todos los Investigadores

Grupos

Regeneración y Trasplante Cardíaco

Leader

Pilar Sepúlveda Sanchis
Farmacogenética

Investigador Principal

María José Herrero Cervera
Unidad de Descubrimiento de Fármacos (UDF)

Investigador Principal

Leonor Puchades Carrasco
Reumatología Pediátrica

Investigador establecido

Inmaculada Calvo Penadés
Investigación en Neuroinmunología y Esclerosis Múltiple

Leader

Bonaventura Casanova Estruch
Grupo de Investigación de Farmacia

Investigador Principal

Javier García Pellicer

Financiadores - Promotores

Swedish Orphan Biovitrum AG (Sobi AG)

Resultados del Ensayo Clínico

Publicaciones en Revistas 86

[Translated article] Pharmacokinetic-guided switching from standard half-life factor VIII to extended half-life pegylated factor VIII in haemophilia A therapy in clinical practice.

Chovi-Trull, Maria; (...); Andres, Jose Luis Poveda

Article. 10.1016/j.farma.2024.12.007. 2025

[Translated article] Real-world effectiveness and safety of nivolumab in patients with relapsed or refractory classical Hodgkin lymphoma.

Fernandez, Laura Lorente; (...); Andres, Jose Luis Poveda

Article. 10.1016/j.farma.2024.09.006. 2024

A Gadolinium(III) Complex Based on Pyridoxine Molecule with Single-Ion Magnet and Magnetic Resonance Imaging Properties.

Orts-Arroyo, Marta; (...); Martinez-Lillo, Jose

Article. 10.3390/ijms25042112. 2024

A multicriteria decision analysis (MCDA) applied to three long-term prophylactic treatments for hereditary angioedema in Spain

Zozaya, Neboa; (...); Hidalgo-Vega, Alvaro

Article. 10.33393/grhta.2022.2333. 2022

A multi-criteria decision analysis on the value of nintedanib for interstitial lung diseases.

Zozaya, Neboa; (...); Verde, Luis

Article. 10.1017/S0266462322000459. 2022

Aicardi-Goutieres syndrome type 6: report of ADAR variant and clinical outcome after ruxolitinib treatment in the neonatal period

Gabaldon-Albero, Alba; (...); Martinez, Francisco

Article. 10.1186/s12969-024-01036-5. 2024

Assessing the value of moderate-to-severe atopic dermatitis treatment using multi-criteria decision analysis (MCDA).

Pereyra-Rodríguez JJ; (...); Ortiz de Frutos FJ

Letter. 10.1111/jdv.19432. 2023

Automated Cervical Spinal Cord Segmentation in Real-World MRI of Multiple Sclerosis Patients by Optimized Hybrid Residual Attention-Aware Convolutional Neural Networks

Bueno, America; (...); Alberich-Bayarri, Angel

Article. 10.1007/s10278-022-00637-4. 2022

Bioavailability of once-daily tacrolimus formulations used in clinical practice in the management of De Novo kidney transplant recipients: the better study.

Fernandez Rivera C; (...); Hernandez D

Article. 10.1111/ctr.14550. 2022

CLINICAL VARIABILITY IN PATIENTS DIAGNOSED OF JUVENILE IDIOPATHIC ARTHRITIS WITH POSITIVE AND NEGATIVE ANTINUCLEAR ANTIBODIES (ANA)

Torrat Noves, A. M.; (...); Roman Ivorra, J. A.

Meeting Abstract. 10.1136/annrheumdis-2023-eular.5203. 2023

Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia.

Montoro-Ronsano JB; (...); Soto-Ortega I

Article. 10.1016/j.farma.2023.01.003. 2023

Consensus recommendations for the improvement of inter- and intra-centre care coordination in the management of hemophilia.

Montoro-Ronsano, Jose Bruno; (...); Soto-Ortega, Inmaculada

Article. 10.1016/j.farma.2023.04.002. 2023

Childhood-Onset Non-Infectious Uveitis in the "Biologic Era". Results From Spanish Multicenter Multidisciplinary Real-World Clinical Settings.

Mesa-Del-Castillo P; (...); Sánchez Sevila JL

Article. 10.1080/09273948.2024.2336609. 2024

Design and rationale for a real-world prospective, multicenter registry of myocardial revascularization failure and secondary revascularization: The REVASEC study.

Salinas P; (...); Escaned J

Article. 10.1016/j.carrev.2021.11.015. 2021

Design and Validation of an Expanded Disability Status Scale Model in Multiple Sclerosis.

Barreiro-Gonzalez, Antonio; (...); Casanova, Bonaventura

Article. 10.1159/000519772. 2022

Determining value in the treatment of activated PI3Kdelta syndrome in Spain: a multicriteria decision analysis from the perspective of key stakeholders.

Abad, Maria Reyes; (...); Gil, Alicia

Article. 10.33393/grhta.2024.3041. 2024

Determining what represents value in the treatment of prurigo nodularis and its key unmet needs in Spain through Multi-Criteria Decision Analysis

Silvestre, Juan Francisco; (...); Serra-Baldrich, Esther

Article. 10.1002/jvc2.275. 2024

Development of severity and mortality prediction models for covid-19 patients at emergency department including the chest x-ray

Calvillo-Batllés P; (...); Martí-Bonmatí L

Article. 10.1016/j.rx.2021.09.011. 2022

Development of severity and mortality prediction models for covid-19 patients at emergency department including the chest x-ray.

Calvillo-Batllés P; (...); Martí-Bonmatí L

Article. 10.1016/j.rxeng.2021.09.004. 2022

Dual latent tuberculosis screening with tuberculin skin tests and QuantiFERON-TB assays before TNF-alpha inhibitor initiation in children in Spain.

Calzada-Hernandez, Joan; (...); Noguera-Julian, Antoni

Article. 10.1007/s00431-022-04640-3. 2022

Elevation of serum neurofilament light-chain levels disclose possible occult progressive multifocal leukoencephalopathy and immune reconstitution syndrome in a patient receiving ozanimod: a case report.

Quintanilla-Bordás C; (...); Pérez-Miralles FC

Case Reports. 10.3389/fimmu.2024.1465678. 2024

Healthcare Resource Utilization among Patients between 60-75 Years with Secondary Acute Myeloid Leukemia Receiving Intensive Chemotherapy Induction: A Spanish Retrospective Observational Study

Solana-Altabella, Antonio; (...); Martinez-Cuadron, David

Article. 10.3390/cancers14081921. 2022

HOW TO DIAGNOSE: JUVENILE PSORIATIC ARTHRITIS. MULTICENTER PROSPECTIVE OBSERVATIONAL STUDY IN CHILDREN WITH SUSPECTED DIAGNOSIS

Palmou-Fontana, N.; (...); Collado, P.

Meeting Abstract. 10.1136/annrheumdis-2023-eular.1883. 2023

Inmune-mediated inflammatory rheumatic diseases in transgender people: A scoping review.

Salgado E; (...); Gomez-Reino JJ

Article. 10.1016/j.semarthrit.2021.10.004. 2022

INSIGHTS FROM A NOVEL MONOGENIC AUTOIMMUNE DISEASE: OVERVIEW OF A MULTICENTRIC EUROPEAN COHORT OF 27 PATIENTS WITH COPA SYNDROME

David-Gabarre, C.; (...); Fremond, M. L.

Meeting Abstract. 10.1136/annrheumdis-2023-eular.3505. 2023

Is erenumab an efficient alternative for the prevention of episodic and chronic migraine in Spain? Results of a cost-effectiveness analysis.

Pozo-Rosich P; (...); Irimia P

Article. 10.1186/s10194-024-01747-w. 2024

Isavuconazole Pharmacokinetics in Critically Ill Patients: Relationship with Clinical Effectiveness and Patient Safety.

Martin-Cerezuela, Maria; (...); Ramirez Galleymore, Paula

Article. 10.3390/antibiotics13080706. 2024

Long-term efficacy and safety of canakinumab in patients with mevalonate kinase deficiency: results from the randomised Phase 3 CLUSTER trial.

Jeyaratnam J; (...); Frenkel J

Article. 10.1093/rheumatology/keab696. 2021

Long-term efficacy and safety of subcutaneous tocilizumab in clinical trials of polyarticular or systemic juvenile idiopathic arthritis.

Brunner, Hermine I; (...); De Benedetti, Fabrizio

Article. 10.1093/rheumatology/keae180. 2024

Long-term efficacy/safety of canakinumab in patients with tumor necrosis factor receptor-associated periodic syndrome: results from a phase III trial.

Gattorno, Marco; (...); Lachmann, Helen

Article. 10.1002/art.42695. 2023

Long-Term Maintenance of Clinical Responses by Individual Patients With Polyarticular-Course Juvenile Idiopathic Arthritis Treated With Abatacept.

Brunner, Hermine I; (...); Ruperto, Nicolino

Article. 10.1002/acr.25156. 2023

Management of iodine contrast induced salivary gland swelling (sialadenitis): experiences from an observational study.

Saro-Buendía M; (...); Armengot Carceller M

Article. 10.1080/00016489.2022.2162121. 2023

Manipulación de fármacos para su administración por gastrostomía endoscópica percutánea en pacientes con esclerosis lateral amiotrófica y nutrición enteral.

Vázquez Polo A; (...); Vázquez Costa JF

Article. 10.20960/nh.03946. 2022

Observational study to evaluate discontinuation of monotherapy with cobicistat-boosted darunavir in patients with human immunodeficiency virus

Solana-Altabella, Antonio; (...); Poveda-Andres, Jose Luis

Article. 10.1097/MD.0000000000032208. 2022

ODs with a positive TPR conclusion, not subject to a conditional approval, and approved without requering a pass would be more likely to be reimbursed in Spain.

Poveda JL; (...); Badia X

Article. 10.1186/s13023-022-02610-4. 2023

Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes

Gomez-Cebrian, Nuria; (...); Puchades-Carrasco, Leonor

Review. 10.3390/biology12091159. 2023

OPTIMIZING MANAGEMENT AND OUTCOMES OF ACTIVATED PHOSPHOINOSITIDE 3-KINASE DELTA SYNDROME (APDS) IN SPAIN: BEWAYAPDS PROJECT

Flores-Moreno, S.; (...); Gil, A.

Meeting Abstract. 2024

Paracetamol vs. Ibuprofen in Preterm Infants With Hemodynamically Significant Patent Ductus Arteriosus: A Non-inferiority Randomized Clinical Trial Protocol (vol 8, 372, 2020)

Garcia-Robles, Ana; (...); Aguar Carrascosa, Marta

Correction. 10.3389/fped.2021.834454. 2022

Pharmacogenetics in Response to Biological Agents in Inflammatory Bowel Disease: A Systematic Review.

Ballesta-Lopez, Octavio; (...); Poveda-Andres, Jose Luis

Article. 10.3390/ijms26041760. 2025

Pharmacokinetic, Pharmacodynamic, and Safety Profile of Subcutaneous Belimumab in Pediatric Patients with Systemic Lupus Erythematosus: Analysis of Data from a Multicenter, Open-Label Trial

Brunner, Hermine; (...); Wilde, Paul

Meeting Abstract. 2023

Secukinumab in enthesitis-related arthritis and juvenile psoriatic arthritis: a randomised, double-blind, placebo-controlled, treatment withdrawal, phase 3 trial

Brunner, Hermine, I; (...); Ruperto, Nicolino

Article. 10.1136/ard-2022-222849. 2022

SMPTall man lettering application in medication information systems as a quality and safety strategy in hospital organization

Iglesias Gomez, Ruben; (...); Poveda Andres, Jose Luis

Article. 10.1111/jcpt.13703. 2022

The value of the reflective discussion in decision-making using multi-criteria decision analysis (MCDA): an example of determining the value contribution of tabelecleucel for the treatment of the Epstein Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD).

Badia, Xavier; (...); Valles, Joan-Antoni

Article. 10.1186/s13023-024-03324-5. 2024

Toxic-metabolic encephalopathy induced by metronidazole and disulfiram: classics never die.

Chovi-Trull, Maria; (...); Poveda-Andres, Jose Luis

Article. 10.1136/ejhpharm-2024-004184. 2024

Transient reduced diffusion on magnetic resonance imaging due to transneuronal degeneration of the striatum.

Belloch-Ripollés V, Carreres-Polo J

Case Reports. 10.33588/rn.7504.2022035. 2022

Transient restriction of diffusion in magnetic resonance imaging due to transneuronal degeneration of the corpus striatum

Belloch Ripolles, Vicente, Carreres-Polo, Joan

Editorial Material. 2022

USE OF HEALTHCARE RESOURCES AND COST ASSOCIATED RELATED TO JUVENILE IDIOPATHIC ARTHRITIS IN SPAIN: THE ITACA-OR REAL-WORLD STUDY

Peral, C.; (...); Calvo, I

Meeting Abstract. 2022

Validity and reliability of four parent/patient reported outcome measures for juvenile idiopathic arthritis remote monitoring.

van Dijkhuizen EHP; (...); Paediatric Rheumatology International Trials Organisation (PRINTO)

Article. 10.1002/acr.24855. 2022

VALUE CRITERIA FOR OUTCOMES BASED MANAGED ENTRY AGREEMENT OF CAR-T IN RELAPSED OR REFRACTORY MULTIPLE MYELOMA (RRMM) THROUGH MCDA

Poveda, J. L.; (...); Lizan, L.

Meeting Abstract. 2023

What drives the decision to optimise biological treatment in children and youngsters with juvenile idiopathic arthritis? A discrete-choice experiment

Murias, Sara; (...); Anton, Jordi

Article. 10.1016/j.reuma.2021.12.003. 2023

What drives the decision to optimise biological treatment in children and youngsters with juvenile idiopathic arthritis? A discrete-choice experiment.