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Proyectos y Estudios
Ensayo de fase III, doble ciego, controlado con placebo para evaluar la eficacia y la seguridad de apitegromab (SRK-015) en pacientes con atrofia muscular espinal de inicio tardío que reciben tratamiento de fondo con nusinersén o risdiplam.

Ensayo de fase III, doble ciego, controlado con placebo para evaluar la eficacia y la seguridad de apitegromab (SRK-015) en pacientes con atrofia muscular espinal de inicio tardío que reciben tratamiento de fondo con nusinersén o risdiplam.

Datos básicos

Protocolo:: SRK-015-003
EUDRACT:: 2021-005314-34
NCT:
Centro:: HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Año de incio:: 2022
Año de finalización:: 2024

ENSAYO CLÍNICO

Objetivos del proyecto

Objetivo Principal: Principal de eficacia: Evaluar la eficacia de apitegromab en comparación con placebo con el uso de HFMSE en pacientes de 2 a 12 años de edad Objetivos Secundarios: Secundarios clave de eficacia: -Evaluar la eficacia de apitegromab en comparación con placebo en función del número de pacientes con mejoría clínica, en pacientes de 2 a 12 años de edad. -Evaluar la eficacia de apitegromab en comparación con placebo midiendo los cambios en la función de las extremidades superiores mediante el módulo revisado de extremidades superiores (RULM) en pacientes de 2 a 12 años de edad. -Evaluar la eficacia de apitegromab en comparación con placebo midiendo los cambios en el número de hitos del desarrollo motor de la Organización Mundial de la Salud (OMS) en pacientes de 2 a 12 años de edad. Otros secundarios de eficacia - Evaluar mejor la eficacia de apitegromab en comparación con placebo mediante la evaluación de variaciones en las medidas de valoración adicionales de la función motora y variaciones en HFMSE en otros puntos temporales preespecificados en pacientes de 2 a 12 años de edad. Refiéranse al texto en inglés

Documentos

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Participantes

Juan Francisco Vázquez Costa

Investigador Colaborador
Eugenia Ibáñez Albert

Investigador Colaborador
Inmaculada Pitarch Castellano

Investigador Principal
José Luis Poveda Andrés

Investigador Colaborador
Nancy Carolina Ñungo Garzón

Data Manager / Study Coordinator
Ana Vaño Bellver

Investigador Colaborador
Silvia Pérez Albertos

Data Manager / Study Coordinator
Jordi Tortosa Carreres

Investigador Colaborador

Grupos

Patología Neuromuscular y Ataxias

Investigador Principal

Teresa Sevilla Mantecón
Medicina Física y Rehabilitación

Investigador Principal

Isabel Vázquez Arce
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

SCHOLAR ROCK, INC.

Resultados del Ensayo Clínico

Publicaciones en Revistas 74

[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 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

Advances in the early diagnosis of amyotrophic lateral sclerosis.

Garcia-Casanova, Pilar H, Vazquez-Costa, Juan F

Article. 10.1080/14737175.2025.2471556. 2025

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

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 trajectories of patients with multiple sclerosis from onset and their relationship with serum neurofilament light chain levels

Quintanilla-Bordas, Carlos; (...); Casanova, Bonaventura

Article. 10.3389/fneur.2024.1477335. 2024

Comparative Assessment of Simoa and Lumipulse for Measuring Serum Neurofilament Light Chain in Multiple Sclerosis Patients

Gasque-Rubio, Raquel; (...); Perez-Miralles, Francisco

Article. 10.1155/2024/1950913. 2024

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 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

Dystrophinopathy Phenotypes and Modifying Factors in DMD Exon 45-55 Deletion

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

Article. 10.1002/ana.26461. 2022

Endothelial Biomarkers Are Superior to Classic Inflammatory Biomarkers in Community-Acquired Pneumonia.

González-Jiménez P; (...); Méndez R

Article. 10.3390/biomedicines12102413. 2024

Evaluating the complement C1q levels in serum and cerebrospinal fluid in multiple sclerosis patients: Could it serve as a valuable marker in clinical practice?

Tortosa-Carreres J; (...); Pérez-Miralles FC

Article. 10.1016/j.jneuroim.2024.578428. 2024

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

Genetic Variants Associated with Biological Treatment Response in Inflammatory Bowel Disease: A Systematic Review.

Plaza J; (...); Moret-Tatay I

Article. 10.3390/ijms25073717. 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

High Levels of Cerebrospinal Fluid Kappa Free Light Chains Relate to IgM Intrathecal Synthesis and Might Have Prognostic Implications in Relapsing Multiple Sclerosis.

Castillo-Villalba, Jessica; (...); Casanova, Bonaventura

Article. 10.3389/fimmu.2022.827738. 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

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

Predictive potential of serum and cerebrospinal fluid biomarkers for disease activity in treated multiple sclerosis patients.

Tortosa-Carreres J; (...); Pérez-Miralles FC

Article. 10.1016/j.msard.2024.105734. 2024

Presence of good humanization practices in the healthcare of patients with rare diseases in Pharmacy Services

Company Albir MJ, Poveda Andrés JL, Edo Solsona MD

Article. 10.1016/j.farma.2023.06.0131130. 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

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

Treatment of spinal muscular atrophy in European countries: A call to action

Vazquez-Costa, Juan F.; (...); Cattinari, Maria Grazia

Article. 10.1111/ene.15522. 2022

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