Portal de recerca
Estudio de fase 2a de TPN-101 en pacientes con ELA/DFT C9ORF72 (esclerosis lateral amiotrófica y/o demencia frontotemporal).
Dades bàsiques
- Protocol:
- TPN-101-C9-201
- EURDRACT:
- 2021-002251-11
- NCT:
- Centre:
- HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
- Any inici:
- 2022
- Any de finalització:
- 2024
Objectius del projecte
Objetivo Principal: Evaluar la seguridad y tolerabilidad de TPN-101 en pacientes con C9ORF72 ELA / DFT Objetivos Secundarios: • Evaluar las concentraciones de TPN-101 en plasma y líquido cefalorraquídeo (LCR) • Evaluar la interacción con la diana de TPN-101 en sangre • Evaluar los biomarcadores relacionados con la enfermedad • Evaluar los efectos farmacodinámicos (FD) de TPN-101 en sangre y LCR • Evaluar los biomarcadores inflamatorios en sangre y/o LCR • Evaluar el estado clínico y funcional
Documents
- No hi ha documents
Grups d'Investigació
Finançadors - Promotors
Resultats de l'Assaig Clínic
[Translated article] Good humanisation practices in the healthcare of patients with rare diseases in Pharmacy Services
Company Albir MJ, Andrés JLP, Solsona MDE
Article. 10.1016/j.farma.2023.10.004. 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
Amyloid brain-dedicated PET images can diagnose Alzheimer's pathology with Centiloid Scale.
Gandia-Ferrero MT; (...); Martí-Bonmatí L
Article. 10.1016/j.ejmp.2024.103345. 2024
Angiotensin II Receptor Blockers Reduce Tau/ASS42 Ratio: A Cerebrospinal Fluid Biomarkers' Case-Control Study.
Garcia-Lluch, Gemma; (...); Chafer-Pericas, Consuelo
Article. 10.3390/pharmaceutics15030924. 2023
Artificial Intelligence on FDG PET Images Identifies Mild Cognitive Impairment Patients with Neurodegenerative Disease.
Prats-Climent, Joan; (...); Marti-Bonmati, Luis
Article. 10.1007/s10916-022-01836-w. 2022
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
Assessment of COVID-19 lockdown effect on early Alzheimer Disease progression.
Pena-Bautista, Carmen; (...); Chafer-Pericas, Consuelo
Article. 10.1007/s00415-023-11899-5. 2023
Assessment of Lipid Peroxidation in Alzheimer's Disease Differential Diagnosis and Prognosis.
Ferre-Gonzalez, Laura; (...); Chafer-Pericas, Consuelo
Article. 10.3390/antiox11030551. 2022
Assessment of Plasma and Cerebrospinal Fluid Biomarkers in Different Stages of Alzheimer's Disease and Frontotemporal Dementia.
Álvarez-Sánchez L; (...); Cháfer-Pericás C
Article. 10.3390/ijms24021226. 2023
Attitudes toward seeking professional help among patients with early Alzheimer's disease
Villarejo-Galende, A.; (...); Maurino, J.
Meeting Abstract. 2023
Awareness of Diagnosis in Persons with Early-Stage Alzheimer's Disease: An Observational Study in Spain.
Villarejo-Galende, Alberto; (...); Maurino, Jorge
Article. 10.1007/s40120-022-00367-3. 2022
Cardiovascular Risk Scales Association with Cerebrospinal Fluid Alzheimer's Disease Biomarkers in Cardiovascular Low Cardiovascular Risk Regions.
García-Lluch G; (...); Cháfer-Pericás C
Article. 10.14283/jpad.2024.16. 2024
Clinical trials in pediatric ALS: a TRICALS feasibility study.
Kliest T; (...); Van Den Berg LH
Article. 10.1080/21678421.2021.2024856. 2022
Clinical usefulness of cerebrospinal fluid biomarkers in Alzheimer Disease.
Pena-Bautista, Carmen; (...); Chafer-Pericas, Consuelo
Article. 10.1111/eci.13910. 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
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
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
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
Discordant Amyloid Status Diagnosis in Alzheimer's Disease.
García-Vallés L; (...); Cháfer-Pericás C
Article. 10.3390/biomedicines10112880. 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
Article. 10.1080/21678421.2023.2248199. 2024
Early Alzheimer's Disease Screening Approach Using Plasma Biomarkers
Alvarez-Sanchez, Lourdes; (...); Chafer-Pericas, Consuelo
Article. 10.3390/ijms241814151. 2023
Emotion recognition and baseline cortisol levels relationship in early Alzheimer Disease.
Ferrer-Cairols, I.; (...); Chafer-Pericas, C.
Article. 10.1016/j.biopsycho.2023.108511. 2023
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 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
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
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
How can artificial intelligence optimize value-based contracting?
Poveda, Jose Luis; (...); Medrano, Ignacio H.
Letter. 10.1186/s40545-022-00475-3. 2022
IgG antibody response to pneumococcal-conjugated vaccine (Prevenar®13) in children with immunodeficiency disorders.
Garrido-Jareño M; (...); Pemán-García J
Article. 10.1007/s00430-022-00759-0. 2023
Insights from a 7-Year Dementia Cohort (VALCODIS): ApoE Genotype Evaluation.
Baquero, Miguel; (...); Chafer-Pericas, Consuelo
Article. 10.3390/jcm13164735. 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
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
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
Medical help-seeking intentions among patients with early Alzheimer's disease
Villarejo-Galende, Alberto; (...); Maurino, Jorge
Article. 10.3389/fpsyt.2023.1290002. 2023
Metabolic Phenotyping in Prostate Cancer Using Multi-Omics Approaches.
Gomez-Cebrian, Nuria; (...); Puchades-Carrasco, Leonor
Article. 10.3390/cancers14030596. 2022
Misfolded alpha-synuclein detection by RT-QuIC in dementia with lewy bodies: a systematic review and meta-analysis.
Peña-Bautista C; (...); Ferreira D
Article. 10.3389/fmolb.2023.1193458. 2023
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
Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen
Andres-Benito, Pol; (...); Povedano, Monica
Article. 10.3390/ijms25073810. 2024
New approach for early and specific Alzheimer disease diagnosis from different plasma biomarkers.
Forte, Anabel; (...); Chafer-Pericas, Consuelo
Article. 10.1016/j.cca.2024.117842. 2024
New insights into the genetic etiology of Alzheimer's disease and related dementias
Bellenguez, Celine; (...); CHARGE
Article. 10.1038/s41588-022-01024-z. 2022
Novel Ultrasensitive Detection Technologies for the Identification of Early and Minimally Invasive Alzheimer's Disease Blood Biomarkers.
Alvarez-Sanchez, Lourdes; (...); Chafer-Pericas, Consuelo
Article. 10.3233/JAD-215093. 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
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
Person-centered Assessment in Early Alzheimers Disease (P1-1.Virtual)
Villarejo Galende, Alberto; (...); Maurino, Jorge
Meeting Abstract. 2022
Plasma Lipidomics Approach in Early and Specific Alzheimer's Disease Diagnosis.
Pena-Bautista, Carmen; (...); Chafer-Pericas, Consuelo
Article. 10.3390/jcm11175030. 2022
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
Prognostic value of cerebrospinal fluid biomarkers in mild cognitive impairment due to Alzheimer disease.
Lopez-Cuevas, R; (...); Chafer-Pericas, C
Article. 10.1016/j.nrleng.2020.07.024. 2023
Relationship between neuroimaging and emotion recognition in Mild Cognitive Impairment patients.
Gandia-Ferrero, Maria Teresa; (...); Marti-Bonmati, Luis
Article. 10.1016/j.bbr.2023.114844. 2024
Relationship between Plasma Lipid Profile and Cognitive Status in Early Alzheimer Disease.
Pena-Bautista, Carmen; (...); Chafer-Pericas, Consuelo
Article. 10.3390/ijms25105317. 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
Spinal Muscular Atrophy Update in Best Practices: Recommendations for Diagnosis Considerations.
Schroth, Mary; (...); Vazquez-Costa, Juan F.
Article. 10.1212/CPJ.0000000000200310. 2024
Systematic review of brain and blood lipidomics in Alzheimer's disease mouse models.
Ferre-Gonzalez, Laura, Lloret, Ana, Chafer-Pericas, Consuelo
Article. 10.1016/j.plipres.2023.101223. 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
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
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.
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