Ensayo abierto no controlado para evaluar la farmacocinética, farmacodinámica, seguridad y actividad de efgartigimod en niños de 2 a menos de 18 años con miastenia grave generalizada.

Dades bàsiques

Protocol:: ARGX-113-2006
EURDRACT:: 2020-005841-18
NCT:
Centre:: HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Any inici:: 2021
Any de finalització:: 2022

ENSAYO CLÍNICO

Objectius del projecte

Objetivo Principal: Confirmar una dosis óptima, ajustada respecto a la edad, de efgartigimod IV y proporcionar pruebas (según la predicción de un modelo) de una respuesta al tratamiento. Objetivos Secundarios: -Evaluar la seguridad y la tolerabilidad de efgartigimod IV. -Determinar la FC y FD de efgartigimod IV -Determinar la inmunogenicidad de efgartigimod IV. -Determinar la actividad y el efecto sobre la calidad de vida de efgartigimod IV. -Determinar el efecto del tratamiento con efgartigimod sobre la respuesta de anticuerpos a las vacunas.

Documents

No hi ha documents

Participants

José Luis Poveda Andrés

Investigador Colaborador
Teresa Sevilla Mantecón

Investigador Principal
Marina Frasquet Carrera

Investigador Colaborador
Silvia Pérez Albertos

Data Manager / Study Coordinator
Adrián Colás Benito

Investigador Principal
Juan Sebastian Aller Alvarez

Investigador Colaborador

Grups d'Investigació

Patología Neuromuscular y Ataxias

Investigador Principal

Teresa Sevilla Mantecón
Farmacogenética

Investigador Principal

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

Investigador Principal

Leonor Puchades Carrasco
Unidad mixta de investigación en enfermedades raras (CIPF)

Leader

José María Millán Salvador
Grupo de Investigación de Farmacia

Investigador Principal

Javier García Pellicer

Finançadors - Promotors

ARGENX BV

Resultats de l'Assaig Clínic

Publicacions en Revistes 87

[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 description of variant transthyretin amyloidosis (ATTRv) stage 1 patients and asymptomatic carriers in Spain: the EMPATIa study

Davila, Lucia Galan; (...); Tarilonte, Patricia

Article. 10.1186/s13023-024-03304-9. 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

A novel TRMT5 mutation causes a complex inherited neuropathy syndrome: the role of nerve pathology in defining a demyelinating neuropathy.

Argente-Escrig, Herminia; (...); Sevilla, Teresa

Article. 10.1111/nan.12817. 2022

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

Asymptomatic HyperCKemia in the Pediatric Population A Prospective Study Utilizing Next-Generation Sequencing and Ancillary Tests

Marti, Pilar; (...); Vilchez, Juan Jesus

Article. 10.1212/WNL.0000000000210116. 2025

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 and genetic characteristics of 21 Spanish patients with biallelic pathogenic SPG7 mutations.

Baviera-Muñoz R; (...); Espinós C

Article. 10.1016/j.jns.2021.118062. 2021

Clinical features, mutation spectrum and factors related to reaching molecular diagnosis in a cohort of patients with distal myopathies.

Muelas, Nuria; (...); Vilchez, Juan J

Article. 10.1007/s00415-024-12821-3. 2025

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

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

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

Effects of Spinal Cord Stimulation in Patients with Small Fiber and Associated Comorbidities from Neuropathy After Multiple Etiologies

Canos-Verdecho, Angeles; (...); Morales-Suarez-Varela, Maria

Article. 10.3390/jcm14020652. 2025

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

Hereditary transthyretin amyloidosis caused by the Val142Ile variant in Spain.

de Frutos, Fernando; (...); Gonzalez-Costello, Jose

Article. 10.1016/j.rec.2024.12.012. 2025

Hereditary transthyretin amyloidosis caused by Val142Ile variant in Spain: phenotypic characteristics, geographic distribution and population frequency

Seminario, F. De Frutos; (...); Gonzalez-Costello, J.

Meeting Abstract. 10.1093/eurheartj/ehae666.2073. 2024

Impact of SARS-CoV-2 infection and COVID-19 pandemic on the morbidity and mortality of amyotrophic lateral sclerosis patients in Valencia, Spain

Garcia-Casanova, Pilar H.; (...); Vazquez-Costa, Juan F.

Article. 10.1111/ene.16465. 2024

Insights into phenotypic variability caused by GARS1 pathogenic variants.

Jiménez-Jiménez J; (...); Sivera R

Article. 10.1111/ene.16416. 2024

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

ITPR3-associated neuropathy: Report of a further family with adult onset intermediate Charcot-Marie-Tooth disease.

Cabello-Murgui, Javier; (...); Sivera, Rafael

Article. 10.1111/ene.16485. 2024

Management of Hereditary Transthyretin Amyloidosis (ATTRv) Patients and Asymptomatic Carriers in Spain: The EMPATIa Study.

Losada Lopez, Ines; (...); Setaro, Francesca

Article. 10.3390/jcm13247587. 2024

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

Onasemnogene Abeparvovec Administration via Peripherally Inserted Central Catheter: A Case Report.

Pitarch Castellano, Inmaculada; (...); Poveda Andres, Jose L

Case Reports. 10.3390/children11050590. 2024

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

PATIENT-REPORTED SYMPTOM BURDEN OF CHARCOT-MARIE-TOOTH DISEASE TYPE 1A (CMT1A): FINDINGS FROM A REAL-WORLD DIGITAL STUDY

Thomas, Florian; (...); Boutalbi, Youcef

Meeting Abstract. 2022

Patient-Reported Symptom Burden of Charcot-Marie-Tooth Disease Type 1A: Findings From an Observational Digital Lifestyle Study.

Thomas, Florian P; (...); Boutalbi, Youcef

Article. 10.1097/CND.0000000000000426. 2022

Real life experience of tafamidis for the treatment of Spanish patients with Val30Met transthyretin amyloidosis with polyneuropathy.

Sanso, Maria Antonia Ribot; (...); Gonzalez-Moreno, Juan

Article. 10.1016/j.medcli.2024.01.008. 2024

Risdiplam in non-sitter patients aged 16years and older with 5q spinal muscular atrophy.

Nungo Garzon, Nancy Carolina; (...); Vazquez-Costa, Juan F

Article. 10.1002/mus.27804. 2023

Role of the nigrosome 1 absence as a biomarker in amyotrophic lateral sclerosis

Isabel Moreno-Gambin, Maria; (...); Vázquez-Costa JF

Article. 10.1007/s00415-021-10729-w. 2021

Safety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised double-blind, placebo-controlled study

Antozzi, Carlo; (...); Sun, Hong

Article. 2025

Safety and Efficacy of Nipocalimab in Patients With Generalized Myasthenia Gravis: Results From the Randomized Phase 2 Vivacity-MG Study

Antozzi, Carlo; (...); Arroyo, Santiago

Article. 10.1212/WNL.0000000000207937. 2024

SERUM AUTOANTIBODY LOWERING BY THE ANTI-FCRN MONOCLONAL ANTIBODY, NIPOCALIMAB, CORRELATES WITH CLINICAL IMPROVEMENT IN GENERALIZED MYASTHENIA GRAVIS PATIENTS

Guptill, Jeffrey; (...); Sun, Hong

Meeting Abstract. 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

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

VIVACITY-MG: A PHASE 2, MULTICENTER, RANDOMIZED, DOUBLE- BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACO-KINETICS, PHARMACODYNAMICS, AND IMMUNOGENICITY OF NIPOCALIMAB ADMINISTERED TO ADULTS WITH GENERALIZED MYASTHENIA GRAVIS

Antozzi, Carlo; (...); Arroyo, Santiago

Meeting Abstract. 2023

WORK IMPACTS IN PATIENTS WITH CHARCOT-MARIE-TOOTH DISEASE TYPE 1A (CMT1A): FINDINGS FROM A REAL-WORLD DIGITAL STUDY

Thomas, Florian; (...); Boutalbi, Youcef

Meeting Abstract. 2022

Ensayo abierto no controlado para evaluar la farmacocinética, farmacodinámica, seguridad y actividad de efgartigimod en niños de 2 a menos de 18 años con miastenia grave generalizada.

Dades bàsiques

Objectius del projecte

Documents

Participants

Grups d'Investigació

Finançadors - Promotors

Resultats de l'Assaig Clínic

Campos d'Estudi

Compartir el projecte