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Proyectos y Estudios
Un ensayo de fase III, aleatorizado, doble ciego, controlado por placebo y multicéntrico para evaluar la seguridad y eficacia de AMX0035 frente al placebo durante un tratamiento de 48 semanas en pacientes adultos con esclerosis lateral amiotrófica (ELA).

Un ensayo de fase III, aleatorizado, doble ciego, controlado por placebo y multicéntrico para evaluar la seguridad y eficacia de AMX0035 frente al placebo durante un tratamiento de 48 semanas en pacientes adultos con esclerosis lateral amiotrófica (ELA).

Datos básicos

Protocolo:: A35-004
EUDRACT:: 2021-000250-26
NCT:
Centro:: HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Año de incio:: 2021
Año de finalización:: 2024

ENSAYO CLÍNICO

Objetivos del proyecto

Objetivo Principal: 1. Evaluar la seguridad y tolerabilidad del tratamiento con AMX0035 durante 48 semanas en comparación con el placebo en pacientes adultos con ELA. 2. Evaluar el impacto del tratamiento con AMX0035, en comparación con el placebo, en el progreso de la enfermedad durante 48 semanas, basándose en el cambio desde el inicio de la ALSFRS R y la supervivencia. Objetivos Secundarios: 1.Evaluar el impacto del fármaco AMX0035 en comparación con el placebo en la CVL en pacientes adultos con ELA durante 48 sem. 2.Evaluar la CV, mediante el resultado comunicado por el paciente(PRO)del cuestionario de evaluación de 40 puntos de la ELA[ALSAQ-40]durante el tratamiento vs placebo,en pacientes adultos con ELA durante 48 sem. 3.Evaluar AMX0035 vs placebo en el tiempo para cualquier disminución en los sistemas de estadificación de King y MITOS derivados de los datos de la ALSFRS-R durante 48 sem. 4.Evaluar impacto de AMX0035 vs placebo en la supervivencia sin ventilación en pacientes adultos con ELA durante 48 sem. 5.Evaluar AMX0035 vs placebo en el estado de salud del paciente,mediante el uso del sistema descriptivo EQ-5D y la escala visual analógica EQ(EVA EQ)PRO durante el tratamiento de 48 sem. Evaluar la supervivencia general a largo plazo de los pacientes adultos con ELA tratados con MX0035 o placebo

Documentos

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Participantes

Juan Francisco Vázquez Costa

Investigador Principal
José Luis Poveda Andrés

Investigador Colaborador
Nancy Carolina Ñungo Garzón

Data Manager / Study Coordinator
Paula María Lizandra Cortés

Data Manager / Study Coordinator
Nicolás Jannone Pedro

Investigador Colaborador
Silvia Pérez Albertos

Data Manager / Study Coordinator

Grupos

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

Financiadores - Promotores

AMYLYX PHARMACEUTICALS INC

Resultados del Ensayo Clínico

Publicaciones en Revistas 92

[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 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

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 benefits of a Bayesian model for plasma-derived factor VIII/VWF after one year of pharmacokinetic-guided prophylaxis in severe/moderate hemophilia A patients

Megias-Vericat, JE; (...); Poveda, JL

Article. 10.1016/j.thromres.2021.07.009. 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

Clinical trials in pediatric ALS: a TRICALS feasibility study.

Kliest T; (...); Van Den Berg LH

Article. 10.1080/21678421.2021.2024856. 2022

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

Design and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol

Rebollo, Pablo; (...); Vazquez-Costa, Juan F.

Article. 10.1007/s40120-023-00571-9. 2024

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

Diagnostic Efficacy of Genetic Studies in a Series of Hereditary Cerebellar Ataxias in Eastern Spain.

Baviera-Munoz, Raquel; (...); Aller, Elena

Article. 10.1212/NXG.0000000000200038. 2022

Do we really need to calculate a minimal important difference for ALSFRS-R?: A letter in response to 'Clinically meaningful change: evaluation of the Rasch-built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) and the ALSFRS-R' published in Vol. 24(3-4), pp. 311-316

Vázquez-Costa JF

Article. 10.1080/21678421.2023.2248199. 2024

Effectiveness of Caplacizumab Nanobody in Acquired Thrombotic Thrombocytopenic Purpura Refractory to Conventional Treatment

Palanques-Pastor T; (...); Poveda Andrés JL

Article. 10.1159/000517813. 2021

Fatigue, pain, breathing, voice, fatigability, sleep, rest and vulnerability as meaningful outcomes in SMA care: the patients ' and caregivers' voice

Povedano, M.; (...); Rebollo, P.

Meeting Abstract. 10.1016/j.nmd.2022.07.209. 2022

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

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

Intermediate Repeat Expansion in the ATXN2 Gene as a Risk Factor in the ALS and FTD Spanish Population.

Borrego-Hernandez, Daniel; (...); Garcia-Redondo, Alberto

Article. 10.3390/biomedicines12020356. 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

La reserva cognitiva como factor modulador del impacto de la cirugía sobre la memoria visual y la denominación en pacientes con epilepsia del lóbulo temporal.

Cano-Lopez, I; (...); Gonzalez-Bono, E

Article. 10.33588/rn.7308.2021109. 2021

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

Matching-Adjusted Indirect Comparison of Efficacy and Consumption of rVIII-SingleChain Versus Two Recombinant FVIII Products Used for Prophylactic Treatment of Adults/Adolescents with Severe Haemophilia A

Bonanad, S; (...); Santos, S

Article. 10.1007/s12325-021-01853-0. 2021

MEASURING THE EXPERIENCE OF LIVING WITH SPINAL MUSCULAR ATROPHY: THE ROLE OF THE SMA INDEPENDENCE SCALE

Vazquez, J. F.; (...); Madruga, M.

Meeting Abstract. 2022

Metabolic Phenotyping in Prostate Cancer Using Multi-Omics Approaches.

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

Article. 10.3390/cancers14030596. 2022

Multi-Omic Approaches to Breast Cancer Metabolic Phenotyping: Applications in Diagnosis, Prognosis, and the Development of Novel Treatments

Gomez-Cebrian, N; (...); Pineda-Lucena, A

Review. 10.3390/cancers13184544. 2021

National Survey of the SEFH-2019: portfolio of services, healthcare activity, teaching and research in Hospital Pharmacy Services in Spain (vol 45, pg 32, 2021)

Perez-Encinas, Montserrat; (...); Angel Calleja-Hernandez, Miguel

Correction. 10.7399/fh.11740. 2021

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

Pain in Children and Adolescents with Spinal Muscular Atrophy: A Longitudinal Study from a Patient Registry.

Pitarch-Castellano I; (...); Madruga-Garrido M

Article. 10.3390/children10121880. 2023

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 and health practitioner perceptions about the role of hospital pharmacists along the care continuum: the Fharmaconectados Project

Morillo-Verdugo, R; (...); Diaz-Olmo, J

Article. 10.7399/fh.11719. 2021

Patient and health practitioner perceptions about the role of hospital pharmacists along the care continuum: the Fharmaconectados Project.

Morillo-Verdugo R; (...); Díaz-Olmo J

Article. 2021

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

Validation of a Set of Instruments to Assess Patient- and Caregiver-Oriented Measurements in Spinal Muscular Atrophy: Results of the SMA-TOOL Study.

Vazquez-Costa, Juan F.; (...); Madruga-Garrido, Marcos

Article. 10.1007/s40120-022-00411-2. 2022

Validation of Neuromyotype: A smart keyboard for the evaluation of spinal muscular atrophy patients

Lizandra Cortes, P.; (...); Vazquez-Costa, J. F.

Article. 10.1016/j.nrl.2022.05.004. 2024

Validation of Neuromyotype: A smart keyboard for the evaluation of spinal muscular atrophy patients.

Cortes, P Lizandra; (...); Vázquez-Costa JF

Article. 10.1016/j.nrleng.2022.05.001. 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