A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults with Tuberous Sclerosis Complex (TSC)-related Epilepsy(TrustTSC OLE).

Dades bàsiques

Protocol:
1042-TSC-3002
EURDRACT:
2021-003384-10
NCT:
Centre:
HOSPITAL UNIVERSITARI I POLITÈCNIC LA FE
Any inici:
2023
Any de finalització:
ENSAYO CLÍNICO

Documents

  • No hi ha documents

Participants

Grups d'Investigació

Finançadors - Promotors

MARINUS PHARMACEUTICALS EMERALD LIMITED

Resultats de l'Assaig Clínic


[Translated article] Cybersecurity: a priority for pharmacy services in the age of artificial intelligence

Marin, Cayetano M. Hernandez, Monte-Boquet, Emilio, Andres, Jose Luis Poveda

Editorial Material. 10.1016/j.farma.2024.08.009. 2024

  • Open Access.

[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

  • Open Access.

[Translated article] Knowledge of biological therapy in patients with immune-mediated diseases. BIOINFO study.

Segui-Solanes, Carlos; (...); Borras-Blasco, Joaquin

Article. 10.1016/j.farma.2024.08.002. 2024

  • Open Access.

[Translated article] New challenges in advanced therapies.

Megías-Vericat JE; (...); Poveda Andrés JL

Article. 10.1016/j.farma.2024.05.001. 2024

  • Open Access.

[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

  • Open Access.

12-Month Effectiveness and Tolerability of Brivaracetam in the Real-World: The International EXPERIENCE Pooled Analysis

Villanueva, Vicente; (...); Steinhoff, Bernhard J.

Meeting Abstract. 10.1212/WNL.0000000000201896. 2023


Anti-GAD65-related epilepsy and its clinical spectrum

Marin Gracia, M.; (...); Villanueva, V.

Meeting Abstract. 2024


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

  • Open Access.

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

  • Open Access.

BRIVA-ONE study: 12-month outcomes of brivaracetam monotherapy in clinical practice

Villanueva, V.; (...); Sansa, G.

Meeting Abstract. 2024


BRIVA-ONE study: 12-month outcomes of brivaracetam monotherapy in clinical practice.

Villanueva V; (...); Serratosa JM

Article. 10.1002/epi4.13078. 2024

  • Open Access.

Brivaracetam effectiveness and tolerability in older and younger adults with epilepsy: EXPERIENCE, a pooled analysis of international data from retrospective studies.

Faught, Edward; (...); Villanueva, Vicente

Article. 10.1016/j.yebeh.2024.109922. 2024

  • Open Access.

Candidate Selection for Implantation: Noninvasive Predictors of Seizure Onset Zone Focality and Surgical Outcome in People with Drug-Resistant Epilepsy Evaluated by Intracranial Video-EEG Monitoring-A Retrospective Cohort Study

Jannone-Pedro, Nicolas; (...); Villanueva, Vicente

Article. 10.1155/2023/3455061. 2023

  • Open Access.

Cenobamate in real-life setting: Final outcomes of an expanded access program

Villanueva-Haba, V.; (...); Massot-Tarrus, A.

Meeting Abstract. 2023


CLINICAL AND ECONOMIC CONSEQUENCES OF EPILEPSY TREATMENT ACROSS INCREMENTAL TREATMENT LINES IN SPAIN: A REAL LIFE DATABASE ANALYSIS

Toledano, R.; (...); Hernandez, I

Meeting Abstract. 2023


Clinical and economic implications of epilepsy management across treatment lines in Spain: a real-life database analysis.

Toledano R; (...); Pérez-Domper P

Article. 10.1007/s00415-023-11958-x. 2023

  • Open Access.

Cognitive phenotypes in patients with drug-resistant temporal lobe epilepsy: Relationships with cortisol and affectivity.

Cano-Lopez, Irene; (...); Gonzalez-Bono, Esperanza

Article. 10.1080/13854046.2024.2375605. 2024


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


COST-EFFECTIVENESS ANALYSIS OF DIFELIKEFALIN FOR THE TREATMENT OF CHRONIC KIDNEY DISEASE ASSOCIATED-PRURITUS IN SPAIN

Sanchez-Alvarez, Emilio E.; (...); Hernaez Colque, M.

Meeting Abstract. 2024


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


Cybersecurity, a priority for pharmacy services in the age of artificial intelligence.

Hernández Marín CM, Monte-Boquet E, Poveda Andrés JL

Editorial Material. 10.1016/j.farma.2024.08.001. 2024

  • Open Access.

Design of two parallel randomized, double-blind, placebo-controlled phase 3 studies to evaluate the safety and efficacy of XEN1101 as adjunctive therapy in the treatment of focal onset epilepsy

Perucca, E.; (...); Beatch, G. N.

Meeting Abstract. 2023


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

  • Open Access.

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

  • Open Access.

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


Development and validation of the Epilepsy Perceived Stress Inventory for Adults (EPSI-A): A pilot study

Catalan-Aguilar, Judit; (...); Gonzalez-Bono, Esperanza

Article. 10.1016/j.yebeh.2024.110142. 2024

  • Open Access.

Effect of concomitant medications on adverse event frequency, severity, and resolution in a cenobamate open-label trial

Steinhoff, B.; (...); Villanueva, V.

Meeting Abstract. 2024


Effectiveness and Tolerability of 12-Month Brivaracetam in the Real World: EXPERIENCE, an International Pooled Analysis of Individual Patient Records.

Villanueva V; (...); Steinhoff BJ

Article. 10.1007/s40263-023-01033-4. 2023

  • Open Access.

Effectiveness and tolerability of brivaracetam in patients with epilepsy stratified by comorbidities and etiology in the real world: 12-month subgroup data from the international EXPERIENCE pooled analysis.

Szaflarski, Jerzy P; (...); Villanueva, Vicente

Article. 10.1007/s00415-024-12253-z. 2024

  • Open Access.

Effectiveness, safety and tolerability of perampanel by age group when used to treat people with focal and generalized epilepsy in clinical practice: The PERMIT Extension study.

Wheless J; (...); Villanueva V

Article. 10.1016/j.yebeh.2023.109369. 2023

  • Open Access.

Efficacy and safety of stiripentol in the prevention and cessation of status epilepticus: A systematic review.

Specchio N; (...); Trinka E

Article. 10.1002/epi4.13036. 2024

  • Open Access.

Efficacy of add-on Cenobamate treatment in refractory epilepsy due to Rasmussen's encephalitis.

Schulze-Bonhage A; (...); Villanueva V

Article. 10.1002/epi4.13060. 2024

  • Open Access.

Efficacy of stiripentol in the management of status epilepticus: A systematic review

Auvin, S.; (...); Trinka, E.

Meeting Abstract. 2024


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

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

Article. 10.3390/ijms25073717. 2024

  • Open Access.

Impact of antiseizure medication with a very long half-life on long term video-EEG monitoring in focal epilepsy.

Hampel, Kevin G.; (...); Villanueva, Vicente

Article. 10.1016/j.seizure.2023.12.010. 2023


Impact of Polytherapy on Memory Functioning in Patients With Drug-Resistant Epilepsy: The Role of Attention and Executive Functions.

Lozano-Garcia, Alejandro; (...); Gonzalez-Bono, Esperanza

Article. 10.1093/arclin/acad086. 2023


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

  • Open Access.

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

  • Open Access.

Isavuconazole Pharmacokinetics in Critically Ill Patients: Relationship with Clinical Effectiveness and Patient Safety.

Martin-Cerezuela, Maria; (...); Ramirez Galleymore, Paula

Article. 10.3390/antibiotics13080706. 2024

  • Open Access.

Long-term, quality of life in epilepsy inventory-31 (QOLIE-31) improvements in adults with focal onset seizures treated with XEN1101 in an ongoing, open-

Brandt, C.; (...); Beatch, G. N.

Meeting Abstract. 2024


Management of patients with Dravet syndrome in real-world clinical practice across five countries in Europe: Impact of treatment-related burden on health outcomes

Ross, E.; (...); Andrews, J. S.

Meeting Abstract. 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

  • Open Access.

New challenges of advanced therapies.

Megías-Vericat JE; (...); Poveda Andrés JL

Article. 10.1016/j.farma.2023.11.008. 2024

  • Open Access.

Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes

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

Review. 10.3390/biology12091159. 2023

  • Open Access.

OPTIMIZING MANAGEMENT AND OUTCOMES OF ACTIVATED PHOSPHOINOSITIDE 3-KINASE DELTA SYNDROME (APDS) IN SPAIN: BEWAYAPDS PROJECT

Flores-Moreno, S.; (...); Gil, A.

Meeting Abstract. 2024


Perampanel for the treatment of Asian people with epilepsy: Real-world evidence from the PERMIT extension study.

Wu, Tony; (...); Villanueva, Vicente

Article. 10.1016/j.jns.2024.123173. 2024


Perampanel for the treatment of epilepsy with genetic aetiology: Real-world evidence from the PERMIT Extension study.

Delanty N; (...); Villanueva V

Article. 10.1016/j.eplepsyres.2024.107339. 2024

  • Open Access.

Perampanel for the Treatment of People with Idiopathic Generalized Epilepsy in Clinical Practice.

Trinka E; (...); Villanueva V

Article. 10.1111/epi.17631. 2023

  • Open Access.

Perampanel for treatment of epilepsy with tumour, vascular or traumatic brain injury aetiology: Evidence from the PERMIT extension study

Strzelczyk, A.; (...); Villanueva, V.

Meeting Abstract. 2024


Perampanel for treatment of focal and generalized epilepsy in everyday clinical practice: Evidence from permit extension

Trinka, Eugen; (...); Villanueva, Vicente

Meeting Abstract. 10.1016/j.jns.2023.121561. 2023


Perampanel for Treatment of People with a Range of Epilepsy Aetiologies in Clinical Practice: Evidence from the PERMIT Extension Study.

Strzelczyk, Adam; (...); Villanueva, Vicente

Article. 10.1007/s40120-024-00618-5. 2024

  • Open Access.

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

  • Open Access.

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

  • Open Access.

Presurgical Use of Cenobamate for Adult and Pediatric Patients Referred for Epilepsy Surgery: Expert Panel Recommendations.

Laxer KD; (...); Villanueva V

Article. 10.1007/s40120-024-00651-4. 2024

  • Open Access.

Prognostic value of the 5-SENSE Score to predict focality of the seizure-onset zone as assessed by stereoelectroencephalography: a prospective international multicentre validation study.

Astner-Rohracher, Alexandra; (...); Frauscher, Birgit

Article. 10.1136/bmjno-2024-000765. 2024

  • Open Access.

Prospective study of cenobamate on cognition, affectivity and quality of life in focal epilepsy.

Catalan-Aguilar, Judit; (...); Villanueva, Vicente

Article. 10.1002/epi4.12857. 2023

  • Open Access.

Real-life evidence about the use of intravenous brivaracetam in urgent seizures: The BRIV-IV study.

Villanueva V; (...); Escalza I

Article. 10.1016/j.yebeh.2023.109384. 2023


Real-world clinical burden of non-seizure symptoms among patients with dravet syndrome or Lennox- Gastaut syndrome in Europe: Results from a global multinational survey

Berthoz, F. Truong; (...); Andrews, J. S.

Meeting Abstract. 2024


Real-world safety and effectiveness of cenobamate in patients with focal onset seizures: outcomes from an Expanded Access Program.

Villanueva, Vicente; (...); Rodriguez-Uranga, Juan J.

Article. 10.1002/epi4.12757. 2023

  • Open Access.

Retrospective chart review study of use of cannabidiol (CBD) independent of concomitant clobazam use in patients with Lennox-Gastaut syndrome or Dravet syndrome.

Nabbout, Rima; (...); Cross, J. Helen

Article. 10.1016/j.seizure.2023.05.003. 2023

  • Open Access.

Spanish consensus on the management of concomitant antiseizure medications when using cenobamate in adults with drug-resistant focal seizures.

Carreño M; (...); Villanueva V

Article. 10.1002/epi4.12936. 2024

  • Open Access.

Stereoencephalography-guided thermocoagulation: Experience in our centre

De Castro Garcia, A. Celdran; (...); Villanueva, V.

Meeting Abstract. 2024


Stress phenotypes in epilepsy: impact on cognitive functioning and quality of life.

Catalán-Aguilar J; (...); Cano-López I

Article. 10.3389/fpsyg.2023.1100101. 2023

  • Open Access.

Sustained =90% response and seizure freedom in patients with focal-onset seizures treated with cenobamate

Alvarez-Baron, E., Thangavelu, K., Villanueva, V.

Meeting Abstract. 2023


The significance of very long half-life in the context of antiseizure medication withdrawal during long-term video-EEG monitoring.

Hampel, Kevin Gil; (...); Villanueva, Vicente

Letter. 10.1016/j.seizure.2024.01.006. 2024


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

  • Open Access.

Therapeutic strategies during cenobamate treatment initiation: Delphi panel recommendations

Steinhoff, Bernhard J.; (...); Villanueva, Vicente

Review. 10.1177/17562864241256733. 2024

  • Open Access.

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 patterns and disease burden among patients with Lennox- Gastaut syndrome in Europe: A real-world survey

Benitez, A.; (...); Andrews, J. S.

Meeting Abstract. 2024


Use of Stiripentol in Patients with Dravet Syndrome: Common Practice Among Experts in Spain.

Garcia-Penas, Juan Jose; (...); Sanchez-Carpintero, Rocio

Article. 10.1007/s40120-024-00677-8. 2024

  • Open Access.

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


What do 90-99% responder rates mean in patients treated with cenobamate: results from an open label extension study

Serratosa, J.; (...); Villanueva, V.

Meeting Abstract. 2023


What do 90-99% responder rates mean in patients treated with cenobamate: results from the open label extension (OLE) of study C017?

Serratosa, J. M.; (...); Villanueva, V.

Meeting Abstract. 2023


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