Portal de investigación
Estudio de fase 2, aleatorizado, en doble ciego, controlado con placebo y cruzado de dos vías, para evaluar la eficacia, seguridad y tolerabilidad de NMD670 en adultos deambulantes con atrofia espinal muscular de tipo 3.
Datos básicos
- Protocolo:
- NMD670-02-0001
- EUDRACT:
- 2022-002301-24
- NCT:
- Centro:
- HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
- Año de incio:
- 2023
- Año de finalización:
Objetivos del proyecto
Objetivo Principal: Evaluar los cambios en la fuerza y función musculares tras la administración de NMD670 400 mg bid (2 veces al día) durante 21 días, en comparación con placebo, en participantes con SMA Objetivos Secundarios: - Evaluar cambios en la fuerza muscular tras la administración de NMD670 400 mg bid durante 21 días, en comparación con placebo, en participantes con SMA - Evaluar adicionalmente cambios en la fuerza y función musculares tras la administración de NMD670 400 mg bid durante 21 días, comparado con placebo, en participantes con SMA - Evaluar cambios en la transmisión en la unión neuromuscular tras la administración de NDM670 400 mg bid durante 21 días, comparado con placebo, en participantes con SMA - Evaluar la seguridad y la tolerabilidad de NMD670, en comparación con placebo, a lo largo de su administración durante 21 días, en participantes con SMA Objetivos exploratorios: - Evaluar cambios en la calidad de vida y la severidad de la fatiga comunicados por el paciente tras 21 días de tratamiento con NMD670, en comparación con placebo, en participantes con SMA - Explorar la exposición al fármaco tras la administración de NMD670 400 mg bid durante 21 días en participantes con SMA
Documentos
- No hay documentos
Grupos
Financiadores - Promotores
Resultados del Ensayo Clínico
[Translated article] New challenges in advanced therapies.
Megías-Vericat JE; (...); Poveda Andrés JL
Article. 10.1016/j.farma.2024.05.001. 2024
[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
Advances in the early diagnosis of amyotrophic lateral sclerosis.
Garcia-Casanova, Pilar H, Vazquez-Costa, Juan F
Article. 10.1080/14737175.2025.2471556. 2025
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
Autologous stem cell ovarian transplant induced long-lasting effects on the plasma proteome of women with premature ovarian insufficiency
Buigues, A.; (...); Herraiz, S.
Meeting Abstract. 2023
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
Bioengineering an Ovarian-Specific ECM Hydrogel to Treat Premature Ovarian Failure
Frances-Herrero, Emilio; (...); Cervello, Irene
Meeting Abstract. 2023
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
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
Characterizing SOD1 mutations in Spain. The impact of genotype, age, and sex in the natural history of the disease.
Vazquez-Costa, Juan F.; (...); Garcia-Redondo, Alberto
Article. 10.1111/ene.15661. 2022
Deciphering reproductive aging in women using a NOD/SCID mouse model for distinct physiological ovarian phenotypes.
Marchante, Maria; (...); Herraiz, Sonia
Article. 10.18632/aging.205086. 2023
Descriptive analysis of the spinal muscular atrophy population treated with Nusinersen included in the CuidAME project
Sotoca Fernandez, J.; (...); Fernandez-Garcia, M.
Meeting Abstract. 10.1016/j.nmd.2024.07.462. 2024
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
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
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
Article. 10.1080/21678421.2023.2248199. 2024
Drug-drug interactions associated with FLT3 inhibitors for acute myeloblastic leukemia: current landscape.
Solana-Altabella, Antonio; (...); Montesinos, Pau
Article. 10.1080/17512433.2023.2174523. 2023
Expanding the Clinical Spectrum of DRP2-Associated Charcot-Marie-Tooth Disease.
Article. 10.1212/WNL.0000000000209174. 2024
Growth factor-loaded ovarian extracellular matrix hydrogels promote in vivo ovarian niche regeneration and enhance fertility in premature ovarian insufficiency preclinical models.
Frances-Herrero, Emilio; (...); Cervello, Irene
Article. 10.1016/j.actbio.2024.07.056. 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
In vivo follicular activation (IVFA) through repeated ovarian punction: results of an experimental study in a premature ovarian insufficiency rodent model
Rico, R. Galan; (...); Diaz-Garcia, C.
Meeting Abstract. https://doi.org/10.1093/humrep/deae108.961. 2024
Incidence and Risk Factors for Development of Cardiac Toxicity in Adult Patients with Newly Diagnosed Acute Myeloid Leukemia
Boluda, Blanca; (...); Montesinos, Pau
Article. 10.3390/cancers15082267. 2023
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
Kit Ligand Depletion Reverts the Positive Effects of Plasma Rich in Both Stem Cell-Secreted and Platelet-Enclosed Factors on Ovulation and Ovarian Niche
Buigues, Anna; (...); Herraiz, Sonia
Meeting Abstract. 2023
Lifesaving Treatments for Spinal Muscular Atrophy: Global Access and Availability
Armengol, Victor D.; (...); Roy, Bhaskar
Meeting Abstract. 2023
Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.
Armengol, Victor D; (...); Roy, Bhaskar
Article. 10.1212/CPJ.0000000000200224. 2024
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
Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen
Andres-Benito, Pol; (...); Povedano, Monica
Article. 10.3390/ijms25073810. 2024
New challenges of advanced therapies.
Megías-Vericat JE; (...); Poveda Andrés JL
Article. 10.1016/j.farma.2023.11.008. 2024
Nicotinamide mononucleotide (NMN) supplementation during germinal vesicle (GV) rescue could increase the total number of mature oocytes available in women of advanced maternal age (AMA)
Rodriguez Varela, C.; (...); Escriba, M. J.
Meeting Abstract. 10.1016/j.fertnstert.2022.08.427. 2024
Nicotinamide mononucleotide supplementation improves oocyte developmental competence in different ovarian damage conditions.
Ramirez-Martin, Noelia; (...); Herraiz, Sonia
Article. 10.1016/j.ajog.2025.02.006. 2025
Nicotinamide Mononucleotide Supplementation Improves Oocyte Quality in Chemotherapy Induced Ovarian Damage Mouse Models
Ramirez-Martin, Noelia; (...); Herraiz, Sonia
Meeting Abstract. 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
Outcomes of a Pilot Newborn Screening Program for Spinal Muscular Atrophy in the Valencian Community.
Berzal-Serrano, Alba; (...); Millan, Jose M
Article. 10.3390/ijns11010007. 2025
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
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
Pilot study for the implementation of newborn screening for spinal muscular atrophy in Valencia
Berzal-Serrano, Alba; (...); Millan, Jose M.
Meeting Abstract. 10.2174/1871530323666230914122955. 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
SHORT-TERM ORAL SUPPLEMENTATION WITH NICOTINAMIDE MONONUCLEOTIDE RESTORES OOCYTE QUALITY IN CHEMOTHERAPY-INDUCED OVARIAN DAMAGE MOUSE MODELS
Buigues, Anna; (...); Herraiz, Sonia
Meeting Abstract. 2023
Single intraovarian dose of stem cell- and platelet-secreted factors mitigates age-related ovarian infertility in a murine model.
Marchante, Maria; (...); Herraiz, Sonia
Article. 10.1016/j.ajog.2023.01.018. 2023
Spinal Muscular Atrophy Update in Best Practices: Recommendations for Diagnosis Considerations.
Schroth, Mary; (...); Vazquez-Costa, Juan F.
Article. 10.1212/CPJ.0000000000200310. 2024
Systemic changes induced by autologous stem cell ovarian transplant in plasma proteome of women with impaired ovarian reserves.
Buigues, Anna; (...); Herraiz, Sonia
Article. 10.18632/aging.205400. 2023
Telomere Maintenance Improves in the Ovary After SC-PRP Treatment.
Polonio, Alba M.; (...); Varela, Elisa
Meeting Abstract. 2024
The 4-step ASCOT technique modifies the plasma proteome of patients with premature ovarian insufficiency to resemble that of normoresponder women
BUIGUES, A.; (...); HERRAIZ, S.
Meeting Abstract. https://doi.org/10.1093/humrep/deae108.1103. 2024
The cross-sectional area of the median nerve: An independent prognostic biomarker in amyotrophic lateral sclerosis
Martinez-Paya, J. J.; (...); Vazquez-Costa, J. F.
Article. 10.1016/j.nrl.2022.01.008. 2024
The cross-sectional area of the median nerve: An independent prognostic biomarker in amyotrophic lateral sclerosis.
Martinez-Paya, J J; (...); Vazquez-Costa, J F
Article. 10.1016/j.nrleng.2024.07.003. 2024
Underlying mechanisms in clinical manifestations of polycystic ovarian morphology (PCOM) and PCOS women
Ramirez Martin, N.; (...); HERRAIZ, S.
Meeting Abstract. 2023
Underlying molecular mechanisms of Polycystic Ovary Syndrome and Morphology: focusing on the ovary
Ramirez Martin, N.; (...); HERRAIZ, S.
Meeting Abstract. https://doi.org/10.1093/humrep/deae108.936. 2024
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