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Estudio de fase 1, multicéntrico, abierto, con intensificación de dosis y expansión de cohorte con Niraparib y Dostarlimab en pacientes pediátricos con tumores sólidos recurrentes o refractarios

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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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Data retention time: As long as they are useful for the purpose for which they were collected and responsibilities may arise.
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 clínico de fase 2/3, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia, la seguridad y la farmacocinética del Vericiguat en pacientes pediátricos con insuficiencia cardíaca debido a disfunción sistólica sistémica del ventrículo izquierdo

  • Protocol code: MK-1242-036
  • EudraCT code: 2022-501238-52-00
  • Pathology: Heart failure due to systemic left ventricular systolic dysfunction
  • Pharmac: Vericiguat
  • Promoter: Merck Sharp & Dohme LLC.
  • Principal investigator:  Rosés i Noguer, Ferran
  • Research group:  No té grup assignat
  • Service: Cardiologia Pediàtrica
  • Phase: Fase III
  • Recruiting: Open
  • Status: Activo

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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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Transfers: Your data will not be transferred to third parties except to the Principal Investigator who leads the research in order to be able to respond to your query.
Data retention time: As long as they are useful for the purpose for which they were collected and responsibilities may arise.
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 Multi-center, Randomized, Double-blind, Placebo-controlled Trial toEvaluate the Neurological Effects of EryDex on subjects with Ataxia Telangiectasia

  • Protocol code: IEDAT-04-2022
  • EudraCT code: 2023-509077-23-00
  • Pathology: Ataxia Telangiectasia
  • Pharmac: EryDex
  • Promoter: BIOTRIAL PARIS SAS
  • Principal investigator:  Gómez Andrés, David
  • Research group:  Teràpies i Innovació en Neuropediatria i altres malalties rares pediàtriques.
  • Service: Neurologia pediàtrica
  • Phase: Phase III
  • Recruiting: Closed
  • Status: Finalizado

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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Legitimation: Your consent. The data requested is necessary and your request cannot be processed if you do not provide them.
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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 3 multicéntrico para evaluar la eficacia,seguridad y farmacocinética de la inducción de upadacitinib en régimen abierto,aleatorizado, mantenimiento doble ciego y extensión abierta a largo plazo ensujetos pediátricos con colitis ulcerosa activa de moderada a severa y conrespuesta insuficiente, intolerancia o contraindicaciones médicas al tratamientocon corticosteroides, inmunosupresores o terapias biológicas.

  • Protocol code: M14-658
  • EudraCT code: 2022-501788-41-00
  • Pathology: Ulcerative Colitis
  • Pharmac: Upadacitinib
  • Promoter: abbvie
  • Principal investigator:  Segarra Canton, Oscar
  • Research group:  No té grup assignat
  • Service: Gastroenterologia pediàtrica
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
Purpose: Request for information on VHIR clinical research.
Legitimation: Your consent. The data requested is necessary and your request cannot be processed if you do not provide them.
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Data retention time: As long as they are useful for the purpose for which they were collected and responsibilities may arise.
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/III, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia, la seguridad, la tolerabilidad, la farmacocinética y la farmacodinámica del ácido obeticólico en comparación con placebo después de la hepatoportoenterostomía en pacientes pediátricos con atresia biliar

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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 
The request for information on a research project or clinical trial does not entail inclusion in it.

Estudio en fase III multicéntrico, aleatorizado, doble ciego, de grupos paralelos y controlado con placebo de la eficacia y la seguridad de tezepelumab en niños de 5 a <12 años con asma grave no controlada (HORIZON)

  • Protocol code: D5180C00016
  • EudraCT code: 2022-502984-39-00
  • Pathology: Severe Uncontrolled Asthma
  • Pharmac: Tezepelumab
  • Promoter: AstraZeneca
  • Principal investigator:  de Mir Messa, Inés
  • Research group:  Creixement i desenvolupament
  • Service: Pneumologia pediàtrica
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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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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LIGHTBEAM-U01 Subestudio 01A: Subestudio de fase 1/2 para evaluar la seguridad y la eficacia de zilovertamab vedotina en participantes pediátricos y adultos jóvenes con neoplasias malignas hematológicas o tumores sólidos

  • Protocol code: MK-9999-01A
  • EudraCT code: 2023-507178-41-00
  • Pathology: Hematologic Malignancies or Solid Tumors in Pediatric and Young Adults
  • Pharmac: Zilovertamab vedotina
  • Promoter: Merck Sharp & Dohme Corp.
  • Principal investigator:  Hladun Alvaro, Raquel
  • Research group:  Childhood Cancer and Blood Disorders
  • Service: Pediatric Oncohematology
  • Phase: Fase I
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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Estudio de extensión de fase IV, multicéntrico y abierto para pacientes conenfermedad de células falciformes que hayan completado un estudio anterior de crizanlizumab promocionado por Novartis.

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A phase 3, randomized, double-blind, placebo-controlled, parallel-arm study to evaluate the efficacy and safety of RZ358 in patients with congenital hyperinsulinism.

  • Protocol code: RZ358-301
  • EudraCT code: 2023-503240-13-00
  • Pathology: Hypoglycemia associated with congenital hyperinsulinism
  • Pharmac: RZ358
  • Promoter: Rezolute, Inc.
  • Principal investigator:  Clemente Leon, Maria
  • Research group:  Growth and Development
  • Service: Endocrinologia pediàtrica
  • Phase: Phase III
  • Recruiting: Closed
  • Status: Finalizado

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

  • Protocol code: DNLI-E-0008
  • EudraCT code: 2023-503837-23-00
  • Pathology: Mucopolysaccharidosis Type II
  • Pharmac: Tividenofusp Alfa (DNL310)
  • Promoter: Denali therapeutics Inc.
  • Principal investigator:  Del Toro Riera, Mireia
  • Research group:  Neurologia infantil
  • Service: Neurologia pediàtrica
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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ESTUDIO MULTICENTRO DE CONTINUACIÓN EN DOS PARTES, ALEATORIZADO, CONTROLADO CON PLACEBO, DOBLE CIEGO PARA INVESTIGAR LA SEGURIDAD, TOLERABILIDAD, FARMACOCINÉTICA, FARMACODINÁMICA Y EFICACIA DE RO7204239 EN COMBINACIÓN CON RISDIPLAM (RO7034067) EN PACIENTES CON ATROFIA MUSCULAR ESPINAL

  • Protocol code: BN42644
  • EudraCT code: 2021-003417-19
  • Pathology: Spinal Muscular Atrophy (SMA)
  • Pharmac: RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067)
  • Promoter: ROCHE
  • Principal investigator:  Gómez Andrés, David
  • Research group:  Teràpies i Innovació en Neuropediatria i altres malalties rares pediàtriques.
  • Service: Neurologia pediàtrica
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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A SINGLE-ARM, OPEN-LABEL, MULTI-CENTRE, PHASE IB STUDY EVALUATING THE SAFETY AND PRELIMINARY EFFICACY OF AUTO1 IN PAEDIATRIC PATIENTS WITH CD19-POSITIVE RELAPSED/ REFRACTORY (R/R) B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA (B ALL) AND AGGRESSIVE MATURE B-CELL NON-HODGKIN LYMPHOMA (B NHL)

  • Protocol code: AUTO1-PY1
  • EudraCT code: 2023-506307-26-00
  • Pathology: B-cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL).
  • Pharmac: Aucatzyl
  • Promoter: Autolus Ltd
  • Principal investigator:  Díaz de Heredia Rubio, Maria Cristina
  • Research group:  Childhood Cancer and Blood Disorders
  • Service: Pediatric Oncohematology
  • Phase: Phase I
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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A study evaluating the safety and efficacy of once-weekly dosing of somapacitan in a basket study design in paediatric participants with short stature either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature.

  • Protocol code: NN8640-4469
  • EudraCT code: 2022-501055-87-01
  • Pathology: Small for gestational age / Idiopathic short stature / Noonan syndrome / Turners syndrome
  • Pharmac: Somapacitan
  • Promoter: Novo Nordisk
  • Principal investigator:  Mogas Viñas, Eduard
  • Research group:  Growth and Development
  • Service: Endocrinologia pediàtrica
  • Phase: Phase III
  • Recruiting: Closed
  • Status: Finalizado

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Estudio multicéntrico, aleatorizado, doble ciego, de grupos paralelos y controlado con placebo para evaluar la eficacia y la seguridad de iptacopán (LNP023) en la glomerulopatía C3.

  • Protocol code: CLNP023B12301
  • EudraCT code: 2020-004589-21
  • Pathology: Complement 3 glomerulopathy
  • Pharmac: Iptacopan (LNP023)
  • Promoter: Novartis
  • Principal investigator:  Ariceta Iraola, Gema
  • Research group:  Fisiopatologia renal
  • Service: Nefrologia Pediàtrica
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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A phase 3, open-label study of adjunctive ganaxolone (GNX) treatment in children and adults with tuberous sclerosis complex (TSC)-related epilepsy (TrustTSC OLE)

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Estudio en fase I/II, abierto, de seguridad, tolerabilidad, farmacocinética y actividad antitumoral de repotrectinib en pacientes pediátricos y adultos jóvenes con tumores avanzados o metastásicos con reordenaciones de ALK, ROS1 o NTRK1-3 (CARE)

  • Protocol code: TPX-0005-07
  • EudraCT code: 2019-003055-11
  • Pathology: Advanced solid tumors
  • Pharmac: Repotrectinib
  • Promoter: Turning Point Therapeutics
  • Principal investigator:  Pérez Albert, María Paula
  • Research group:  Childhood Cancer and Blood Disorders
  • Service: Pediatric Oncohematology
  • Phase: Phase I
  • Recruiting: Open
  • Status: Activo

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An Open-Label, Single-Arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Etrasimod in Adolescent Subjects with Moderately to Severely Active Ulcerative Colitis

  • Protocol code: APD334-207
  • EudraCT code: 2022-500345-25-00
  • Pathology: Moderately to severely active ulcerative colitis
  • Pharmac: Etrasimod
  • Promoter: Arena Pharmaceuticals
  • Principal investigator:  Segarra Canton, Oscar
  • Research group:  No té grup assignat
  • Service: Gastroenterologia pediàtrica
  • Phase: Phase II
  • Recruiting: Open
  • Status: Activo

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Estudio abierto sobre la seguridad y la eficacia del cenobamato (YKP3089) en pacientes pediátricos con crisis de inicio parcial (focales)

  • Protocol code: YKP3089C040
  • EudraCT code: 2020-005344-27
  • Pathology: Partial-onset (focal) seizures
  • Pharmac: Cenobamate (YKP3089)
  • Promoter: SK Life Science
  • Principal investigator:  Raspall Chaure, Miquel
  • Research group:  Neurologia infantil
  • Service: Neurologia pediàtrica
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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The request for information on a research project or clinical trial does not entail inclusion in it.

Estudio de fase 3b, multicéntrico y abierto, de la seguridad, tolerabilidad y eficacia del tolvaptán en lactantes de 28 días a menos de 12 semanas de edad con poliquistosis renalautosómica recesiva (ARPKD)

  • Protocol code: 156-12-204
  • EudraCT code: 2020-005991-36
  • Pathology: Autosomal Recessive Polycystic Kidney Disease
  • Pharmac: Tolvaptan
  • Promoter: Otsuka Pharmaceutical
  • Principal investigator:  Ariceta Iraola, Gema
  • Research group:  Kidney Physiopathology
  • Service: Pediatric Nephrology
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
Purpose: Request for information on VHIR clinical research.
Legitimation: Your consent. The data requested is necessary and your request cannot be processed if you do not provide them.
Transfers: Your data will not be transferred to third parties except to the Principal Investigator who leads the research in order to be able to respond to your query.
Data retention time: As long as they are useful for the purpose for which they were collected and responsibilities may arise.
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 
The request for information on a research project or clinical trial does not entail inclusion in it.

Estudio aleatorizado, doble ciego, controlado con placebo, de dosis escalonadas múltiples para evaluar la seguridad, la tolerabilidad, la farmacodinámica, la eficacia y la farmacocinética de DYNE-251 administrado a pacientes con distrofia muscular de Duchenne susceptibles de omisión del exón 51.

  • Protocol code: DYNE251-DMD-201
  • EudraCT code: 2021-005478-24
  • Pathology: Duchenne muscular dystrophy
  • Pharmac: DYNE-251
  • Promoter: Dyne Therapeutics, Inc., a Delaware corporation
  • Principal investigator:  Munell Casadesus, Francina
  • Research group:  Neurologia infantil
  • Service: Neurologia pediàtrica
  • Phase: Phase II
  • Recruiting: Closed
  • Status: Finalizado

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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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Transfers: Your data will not be transferred to third parties except to the Principal Investigator who leads the research in order to be able to respond to your query.
Data retention time: As long as they are useful for the purpose for which they were collected and responsibilities may arise.
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 
The request for information on a research project or clinical trial does not entail inclusion in it.