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Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

  • Protocol code: 232SM203
  • EudraCT code: 2019-002663-10
  • Pathology: Atrofia muscular espinal
  • Pharmac: Nusinersen (BIIB058)
  • Promoter: Biogen
  • Principal investigator:  Munell Casadesus, Francina
  • Research group:  Neurologia infantil
  • Service: Neurologia pediàtrica
  • Phase: Fase II
  • Recruiting: Closed
  • Status: Cerrado

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Estudio de fase 1 en el que se evalúa LY3295668 (un inhibidor de lacinasa Aurora A) en monoterapia y en politerapia en pacientes con neuroblastomarecidivante o resistente al tratamiento.

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
Purpose: Request for information on VHIR clinical research.
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Rights: You can access, rectify, delete your data, oppose or limit its processing and request its portability, as well as revoke your consent without retroactive effects by contacting lopd@vhir.org. The revocation of consent and the opposition to the processing of your data will prevent the purpose from being carried out. DPO: dpd@ticsalutsocial.org. More information 
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Estudio de fase II, aleatorizado, abierto y multicéntrico para evaluar la reactogenicidad, seguridad e inmunogenicidad de la vacuna pediátrica candidata de subunidades de herpes zóster (PED-HZ/su) de GSK tras su administración intramuscular con una pauta de dos dosis a niños inmunodeprimidos receptores de trasplante renal de 1-17 años de edad.

  • Protocol code: ZOSTER-47 (200075)
  • EudraCT code: 2019-000607-33
  • Pathology: Herpes zóster en sujetos inmunodeprimidos
  • Pharmac: subunit candidate vaccine (PED-HZ/su)
  • Promoter: GlaxoSmithKline
  • Principal investigator:  Martínez Gómez, Xavier
  • Research group:  Epidemologia i salut pública
  • Service: Medicina Preventiva
  • Phase: Fase II
  • Recruiting: Open
  • Status: Activo

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Rights: You can access, rectify, delete your data, oppose or limit its processing and request its portability, as well as revoke your consent without retroactive effects by contacting lopd@vhir.org. The revocation of consent and the opposition to the processing of your data will prevent the purpose from being carried out. DPO: dpd@ticsalutsocial.org. More information 
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Estudio de fase 1/2 del inhibidor de RET oral LOXO 292 en pacientes pediátricos con tumores sólidos avanzados o tumores primarios del sistema nervioso central ambos con alteración de RET.

  • Protocol code: LOXO-RET-18036 (292036) J2G-OX-JZJJ
  • EudraCT code: 2019-000212-28
  • Pathology: Tumores sólidos o primarios del sistema nervioso central con alteración avanzada de RET
  • Pharmac: Oral RET Inhibitor LOXO-292
  • Promoter: Eli Lilly
  • Principal investigator:  Hladun Alvaro, Raquel
  • Research group:  Càncer i malalties hematològiques infantils
  • Service: Oncohematologia Pediàtrica
  • Phase: Fase I
  • Recruiting: Closed
  • Status: Finalizado

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Rights: You can access, rectify, delete your data, oppose or limit its processing and request its portability, as well as revoke your consent without retroactive effects by contacting lopd@vhir.org. The revocation of consent and the opposition to the processing of your data will prevent the purpose from being carried out. DPO: dpd@ticsalutsocial.org. More information 
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A Phase 2 Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With Thymidine Kinase 2 Deficiency (TK2)

  • Protocol code: MT-1621-102
  • EudraCT code: 2018-004277-27
  • Pathology: Deficiencia de timidina quinasa 2
  • Pharmac: Combination pyrimidine nucleos(t)ides
  • Promoter: UCB Biosciences Inc. (United States)
  • Principal investigator:  Munell Casadesus, Francina
  • Research group:  Neurologia infantil
  • Service: Neurologia pediàtrica
  • Phase: Fase II
  • Recruiting: Closed
  • Status: Finalizado

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Estudio global, multicéntrico, abierto, comparativo con controles históricos equiparables, de SHP611 intratecal en sujetos con leucodistrofia metacromática infantil tardía

  • Protocol code: SHP611-201
  • EudraCT code: 2018-003291-12
  • Pathology: Leucodistrofia metacromática tardía
  • Pharmac: Intrathecal SHP611
  • Promoter: Takeda
  • Principal investigator:  Del Toro Riera, Mireia
  • Research group:  Neurologia infantil
  • Service: Neurologia pediàtrica
  • Phase: Fase II
  • Recruiting: Closed
  • Status: Finalizado

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Estudio abierto, de seguridad a largo plazo, de tolerabilidad y de eficacia de GIVINOSTAT en todos los pacientes con DMD que hayan sido tratadospreviamente en uno de los estudios de GIVINOSTAT.

  • Protocol code: DSC/14/2357/51
  • EudraCT code: 2017-000397-10
  • Pathology: Distrofia muscular de Duchenne
  • Pharmac: GIVINOSTAT
  • Promoter: ITALFARMACO
  • Principal investigator:  Munell Casadesus, Francina
  • Research group:  Neurologia infantil
  • Service: Neurologia pediàtrica
  • Phase: Fase III
  • Recruiting: Open
  • Status: Activo

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Seguimiento a largo plazo (LTFU) de pacientes tratados con la terapia celularCART dirigida a CD19 basada en vectores lentivirales

  • Protocol code: CCTL019A2205B
  • EudraCT code: 2014-001673-14
  • Pathology: Patologías del sistema immunitario. Seguimiento a largo plazo de pacientes expuestos a la terapia lentiviral con células CAR-T
  • Pharmac: Lentiviral-Based CD19 directed CAR T-Cell Therapy
  • Promoter: Novartis
  • Principal investigator:  Díaz de Heredia Rubio, Maria Cristina
  • Research group:  Càncer i malalties hematològiques infantils
  • Service: Oncohematologia Pediàtrica
  • Phase: Fase II
  • Recruiting: Open
  • Status: Activo

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A phase 2, multicenter, open-label study to assess appropriate dosing and to evaluate safety ofcrizanlizumab, with or without hydroxyurea/hydroxycarbamide, in sequential,descending age groups of pediatric sickle cell disease patients with vaso-occlusive crisis.

  • Protocol code: CSEG101B2201
  • EudraCT code: 2017-001747-12
  • Pathology: Células Falciformes con crisis vaso-oclusivas.
  • Pharmac: Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide
  • Promoter: Novartis
  • Principal investigator:  Díaz de Heredia Rubio, Maria Cristina
  • Research group:  Càncer i malalties hematològiques infantils
  • Service: Oncohematologia Pediàtrica
  • Phase: Fase II
  • Recruiting: Closed
  • Status: Cerrado

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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Estudio multicéntrico, aleatorizado, con doble enmascaramiento y controlado con placebo de la eficacia y la seguridad de LJPC-501 en pacientes pediátricos de >2 a 17 años de edad con hipotensión resistente a las catecolaminas asociada a shockdistributivo.

  • Protocol code: LJ501-CRH04
  • EudraCT code: 2017-005153-37
  • Pathology: Hipotensión resistente a las catecolaminas asociada a shock distributivo
  • Pharmac: LJPC-501
  • Promoter: La Jolla Pharmaceutical
  • Principal investigator:  Balcells Ramirez, Joan
  • Research group:  Recerca clínica/Innovació en la pneumònia i sèpsia
  • Service: UCI Pediatria
  • Phase: Fase II
  • Recruiting: Closed
  • Status: Cerrado

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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The request for information on a research project or clinical trial does not entail inclusion in it.
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