Portal de investigación
ESTUDIO DOBLE CIEGO, CONTROLADO CON PLACEBO, PARA EVALUAR LA EFICACIA Y SEGURIDAD DE UN TRATAMIENTO DE 24 SEMANAS CON REN001 EN PACIENTES CON MIOPATÍA MITOCONDRIAL PRIMARIA (MMP).
Datos básicos
- Código:
- REN001-201
- Protocolo:
- REN001-201
- EUDRACT:
- 2020-002855-40
- NCT:
- Centro:
- HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
- Dotación:
- Año de incio:
- 2021
- Año de finalización:
- 2022
Documentos
- No hay documentos
Participantes
Grupos
Financiadores - Promotores
Resultados del Ensayo Clínico
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
Analysis of chemical contamination by hazardous drugs with BD HD Check(R) system in a tertiary hospital
Valero García S; (...); Poveda Andrés JL
Article. 10.1177/10781552211038518. 2021
Analysis of Juvenile Onset Pompe Disease patients included in the Spanish Pompe Registry
Martinez Marin, R.; (...); Diaz Manera, J.
Meeting Abstract. 2022
Analysis of Juvenile onset Pompe disease patients included in the Spanish Pompe Registry
Martinez Marin, R.; (...); Diaz Manera, J.
Meeting Abstract. 10.1016/j.nmd.2022.07.144. 2022
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
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
Baricitinib against severe COVID-19: effectiveness and safety in hospitalised pretreated patients.
Iglesias Gómez R; (...); Poveda Andrés JL
Article. 10.1136/ejhpharm-2021-002741. 2021
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 and genetic spectrum of a large cohort of delta-sarcoglycan muscular dystrophy
Alonso-Perez, J; (...); Diaz-Manera, J
Meeting Abstract. 10.1016/j.nmd.2021.07.202. 2021
Clinical and genetic spectrum of a large cohort of patients with delta-sarcoglycan muscular dystrophy.
Alonso-Perez, Jorge; (...); Diaz-Manera, Jordi
Article. 10.1093/brain/awab301. 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, Histological, and Genetic Features of 25 Patients with Autosomal Dominant Progressive External Ophthalmoplegia (ad-PEO)/PEO-Plus Due to TWNK Mutations.
Bermejo-Guerrero, Laura; (...); Martin, Miguel A.
Article. 10.3390/jcm11010022. 2021
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
CuidAME: a new registry for longitudinal data collection of Spanish SMA patients
Segovia, S; (...); Nascimento, A
Meeting Abstract. 10.1016/j.nmd.2021.07.284. 2021
CuidAME: Registry for longitudinal data collection of Spanish SMA patients
Segovia-Simon, S.; (...); Nascimento, A.
Meeting Abstract. 10.1016/j.nmd.2022.07.078. 2022
Charcot-Marie-Tooth disease due to MORC2 mutations in Spain
Sivera R; (...); Sevilla T
Article. 10.1111/ene.15001. 2021
Delphi consensus on recommendations for the treatment of spinal muscular atrophy in Spain (RET-AME consensus)
Pitarch Castellano, I; (...); Vazquez-Costa, J. F.
Article. 10.1016/j.nrl.2021.07.008. 2022
Delphi consensus on recommendations for the treatment of spinal muscular atrophy in Spain (RET-AME consensus).
Pitarch Castellano I; (...); Vázquez-Costa JF
Practice Guideline. 10.1016/j.nrleng.2021.07.002. 2022
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
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 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
Disseminated toxoplasma infection after hematopoietic stem cell transplantation with myositis and encephalitis.
Asensi Cantó P; (...); Guerreiro M
Article. 10.1111/tid.14067. 2023
Dystrophinopathy Phenotypes and Modifying Factors in DMD Exon 45-55 Deletion
Poyatos-Garcia, Javier; (...); Jesus Vilchez, Juan
Article. 10.1002/ana.26461. 2022
Effectiveness of Caplacizumab Nanobody in Acquired Thrombotic Thrombocytopenic Purpura Refractory to Conventional Treatment
Palanques-Pastor T; (...); Poveda Andrés JL
Article. 10.1159/000517813. 2021
Fetal exposure to tofacitinib during the first trimester: A healthy newborn case report
Fernandez-Sanchez, M; (...); Poveda-Andres, JL
Article. 10.1002/bdr2.1942. 2021
Genotype-phenotype correlations in valosin-containing protein disease: a retrospective muticentre study.
Schiava, Marianela; (...); Diaz-Manera, Jordi
Article. 10.1136/jnnp-2022-328921. 2022
Guide to Good Humanization Practices in Patients with Rare Diseases. who vadis
Editorial Material. 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
High incidence of pulmonary thromboembolism in hospitalized SARS-CoV-2 infected patients despite thrombo-prophylaxis.
El-Qutob D; (...); Carrera-Hueso FJ
Article. 10.1016/j.hrtlng.2022.02.003. 2022
Hospital pharmacy initiatives for improving the management of patients with congenital coagulopathies
Bruno Montoro-Ronsano, Jose; (...); Alberto Vella, Vincenzo
Article. 10.7399/fh.11729. 2022
Hospitalization budget impact during the COVID-19 pandemic in Spain.
Carrera-Hueso, F. J.; (...); Crespo-Palomo, C.
Article. 10.1186/s13561-021-00340-0. 2021
How can artificial intelligence optimize value-based contracting?
Poveda, Jose Luis; (...); Medrano, Ignacio H.
Letter. 10.1186/s40545-022-00475-3. 2022
Immune-mediated necrotising inflammatory myopathy. A rare side effect of statins.
Alarcon Chulilla, Mara; (...); Real Collado, Jose Tomas
Article. 10.1016/j.endien.2022.10.001. 2022
KY mutations are a cause of distal neuromyopathies
Muelas, N.; (...); Vilchez, J.
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
Management of flexible cavovarus foot in patients with Charcot-Marie-Tooth disease: midterm results.
Megías-Vericat JE; (...); Poveda-Andrés JL
Article. 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
Metrics of progression and prognosis in untreated adults with thymidine kinase 2 deficiency: An observational study.
Dominguez-Gonzalez, Cristina; (...); Martin, Miguel Angel
Article. 10.1016/j.nmd.2022.07.399. 2022
Mitochondrial developmental encephalopathy with bilateral optic neuropathy related to homozygous variants in IMMT gene.
Marco-Hernández AV; (...); Martínez-Castellano F
Article. 10.1111/cge.14093. 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
Muscle MRI characteristic pattern for late-onset TK2 deficiency diagnosis
Dominguez-Gonzalez, C; (...); Paradas, C
Meeting Abstract. 10.1016/j.nmd.2021.07.358. 2021
Muscle MRI characteristic pattern for late-onset TK2 deficiency diagnosis.
Dominguez-Gonzalez, Cristina; (...); Paradas, Carmen
Article. 10.1007/s00415-021-10957-0. 2022
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
National Survey on the Use of Mobile Applications in Patients with Hemophilia and Other Coagulopathies
Eduardo Megias, Juan; (...); Poveda Andres, Jose Luis
Meeting Abstract. 10.1182/blood-2022-169938. 2022
Nusinersen in adult patients with 5q spinal muscular atrophy: Amulticenter observational cohorts' study.
Vazquez-Costa, Juan F.; (...); Hervas, David
Article. 10.1111/ene.15501. 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
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
Pediatric inherited peripheral neuropathy: a prospective study at a Spanish referral center
Argente-Escrig H; (...); Sevilla T
Article. 10.1002/acn3.51432. 2021
Pharmacometabolomics by NMR in Oncology: A Systematic Review.
Gómez-Cebrián N; (...); Pineda-Lucena A
Article. 10.3390/ph14101015. 2021
Pharmacotherapeutic management of advanced therapy medicinal products
Luis Poveda-Andres, Jose; (...); Valero-Garcia, Silvia
Article. 10.7399/fh.13036. 2022
Preparation and administration of insulin infusions in neonatology: A national survey.
Garcia-Robles, Ana; (...); Aguar Carrascosa, Marta
Article. 10.1016/j.anpede.2020.06.015. 2021
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
Spanish Pompe registry: analysis of the first 100 patients included
Reyes-Leiva, D; (...); S Spanish Pompe Study Grp
Meeting Abstract. 10.1016/j.nmd.2021.07.224. 2021
Spanish Pompe Registry: New data based on the 130 patients included
Marin, R. Martinez; (...); Manera, J. Diaz
Meeting Abstract. 2023
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: Update of the 122 patients included
Martinez Marin, R.; (...); Diaz Manera, J.
Meeting Abstract. 10.1016/j.nmd.2022.07.145. 2022
Spinocerebellar Ataxia 36 is a Frequent Cause of Hereditary Ataxia in Eastern Spain
Baviera-Munoz, Raquel; (...); Bataller, Luis
Article. 10.1002/mdc3.13740. 2023
Systematic Review of Pharmacogenetics of ABC and SLC Transporter Genes in Acute Myeloid Leukemia
Megías-Vericat JE; (...); Montesinos P
Review. 10.3390/pharmaceutics14040878. 2022
Telemedicine in Neuromuscular Diseases During Covid-19 Pandemic: ERN-NMD European Survey.
El-Hassar L; (...); Sacconi S
Article. 10.3233/JND-221525. 2022
The association of salivary caffeine levels with serum concentrations in premature infants with apnea of prematurity
Garcia-Robles, Ana; (...); Saenz Gonzalez, Pilar
Article. 10.1007/s00431-022-04628-z. 2022
The contribution of fenfluramine to the treatment of Dravet syndrome in Spain through Multi-Criteria Decision Analysis.
Gil-Nagel A; (...); Gil A
Article. 10.1016/j.yebeh.2022.108711. 2022
Transcriptomic Evidence of the Immune Response Activation in Individuals With Limb Girdle Muscular Dystrophy Dominant 2 (LGMDD2) Contributes to Resistance to HIV-1 Infection
Diez-Fuertes F; (...); Rodríguez-Mora S
Article. 10.3389/fcell.2022.839813. 2022
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
Use of NGS for diagnosis of asymptomatic hyperCKemia in childhood
Marti, P; (...); VILCHEZ, J
Meeting Abstract. 10.1016/j.nmd.2021.07.340. 2021
Validación de la versión española de la Charcot-Marie-Tooth Disease Pediatric Scale (CMTPedS).
Pitarch-Castellano I; (...); Burns J
Article. 10.33588/rn.7403.2021489. 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.
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