1. Inici
  2. Projectes i Estudis Clínics
  3. Estudio en fase III, de 1 grupo, multicéntrico y sin enmascaramiento, de extensión del estudio ARGX-113-2007 para investigar la seguridad, la tolerabilidad y la eficacia a largo plazo del efgartigimod PH20 s.c. en participantes de 18 años o más con miopatía inflamatoria idiopática activa.

Estudio en fase III, de 1 grupo, multicéntrico y sin enmascaramiento, de extensión del estudio ARGX-113-2007 para investigar la seguridad, la tolerabilidad y la eficacia a largo plazo del efgartigimod PH20 s.c. en participantes de 18 años o más con miopatía inflamatoria idiopática activa.

Dades bàsiques

Protocol:
ARGX-113-2011
EURDRACT:
NCT:
Centre:
HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Any inici:
2023
Any de finalització:
ENSAYO CLÍNICO

Documents

  • No hi ha documents

Participants

Grups d'Investigació

Finançadors - Promotors

ARGENX BV

Resultats de l'Assaig Clínic

[Translated article] Knowledge of biological therapy in patients with immune-mediated diseases. BIOINFO study.

Segui-Solanes, Carlos; (...); Borras-Blasco, Joaquin

Article. 10.1016/j.farma.2024.08.002. 2024

  • Open Access.

[Translated article] New challenges in advanced therapies.

Megías-Vericat JE; (...); Poveda Andrés JL

Article. 10.1016/j.farma.2024.05.001. 2024

  • Open Access.

[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


Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP-mediated disease reveals characteristic features useful for diagnosis.

Esteller D; (...); Díaz-Manera J

Article. 10.1007/s00415-023-11862-4. 2023

  • Open Access.

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


Benefits of physical activity self-monitoring in patients with haemophilia: a prospective study with one-year follow-up.

Pérez-Alenda S; (...); Núñez-Cortés R

Article. 10.1111/hae.14988. 2024

  • Open Access.

Clinical and Genetic Analysis of Patients With TK2 Deficiency.

Ceballos F; (...); Domínguez-González C

Article. 10.1212/NXG.0000000000200138. 2024

  • Open Access.

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


Correction to: Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP-mediated disease reveals characteristic features useful for diagnosis.

Esteller D; (...); Díaz-Manera J

Correction. 10.1007/s00415-023-12178-z. 2024

  • Open Access.

Cost-effectiveness and cost-utility analysis of haemate-P versus other von willebrand disease treatments in Spain.

Megias-Vericat, Juan E; (...); Yan, Songkai

Article. 10.1080/13696998.2025.2474886. 2025


Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model

Poyatos-Garcia, Javier; (...); Vilchez, Juan J

Article. 10.1186/s13395-024-00353-3. 2024

  • Open Access.

Description of clinical and genetic features of 122 patients included in the Spanish Pompe registry

Martinez-Marin, Rafael Jenaro; (...); Diaz-Manera, Jordi

Article. 10.1016/j.nmd.2023.10.001. 2024


Development and testing of a mobile app for systematic collection of patient-reported outcomes (PROs) in patients with hemophilia.

Megias-Vericat, J E; (...); Poveda Andres, J L

Letter. 10.1016/j.thromres.2024.109122. 2024


Digenic inheritance involving a muscle-specific protein kinase and the giant titin protein causes a skeletal muscle myopathy.

Topf, Ana; (...); Straub, Volker

Article. 10.1038/s41588-023-01651-0. 2024

  • Open Access.

Disseminated toxoplasma infection after hematopoietic stem cell transplantation with myositis and encephalitis.

Asensi Cantó P; (...); Guerreiro M

Article. 10.1111/tid.14067. 2023

  • Open Access.

DRP2 mutations as a cause of Charcot Marie Tooth in Spain

Sivera, R.; (...); Sevilla, T.

Meeting Abstract. 2023


Expanding the Clinical Spectrum of DRP2-Associated Charcot-Marie-Tooth Disease.

Sivera R; (...); Sevilla T

Article. 10.1212/WNL.0000000000209174. 2024


Expanding the phenotype of GARS1 mutations

Sivera, R.; (...); Teresa, S.

Meeting Abstract. 2023


Insights into phenotypic variability caused by GARS1 pathogenic variants.

Jiménez-Jiménez J; (...); Sivera R

Article. 10.1111/ene.16416. 2024

  • Open Access.

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

  • Open Access.

Mental, emotional and social dimensions of quality of life and their relationship with physical and functional status in adults with haemophilia.

Rodríguez MA; (...); Chimeno-Hernández A

Article. 10.1016/j.thromres.2024.109181. 2024

  • Open Access.

Muscle MRI-phenotyping of patients with likely pathogenic anoctamin 5 variants

Poulsen, N.; (...); Vissing, J.

Meeting Abstract. 10.1016/j.nmd.2024.07.187. 2024

  • Open Access.

New challenges of advanced therapies.

Megías-Vericat JE; (...); Poveda Andrés JL

Article. 10.1016/j.farma.2023.11.008. 2024

  • Open Access.

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

  • Open Access.

Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.

Tawil R; (...); Jiang JG

Article. 10.1016/S1474-4422(24)00073-5. 2024


Short-term and long-term safety profile of Givinostat in Duchenne muscular dystrophy

Vucinic, D.; (...); Muelas, N.

Meeting Abstract. 10.1016/j.nmd.2024.07.289. 2024

  • Open Access.

Spanish Pompe registry: new data based on the 130 patients included

Martinez Marin, R.; (...); Manera, J. Diaz

Meeting Abstract. 10.1016/j.nmd.2023.07.343. 2023


Spanish Pompe Registry: overview based on the 130 patients included

Marin, R. Martinez; (...); Manera, J. Diaz

Meeting Abstract. 10.1016/j.nmd.2024.07.732. 2024

  • Open Access.

Spinocerebellar Ataxia 36 is a Frequent Cause of Hereditary Ataxia in Eastern Spain

Baviera-Munoz, Raquel; (...); Bataller, Luis

Article. 10.1002/mdc3.13740. 2023

  • Open Access.

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


Campos d'Estudi

Compartir el projecte