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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:  Creixement i desenvolupament
  • Service: Endocrinologia pediàtrica
  • Phase: Fase III
  • Recruiting: Closed
  • Status: Finalizado

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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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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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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 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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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:  Càncer i malalties hematològiques infantils
  • Service: Oncohematologia Pediàtrica
  • Phase: Fase I
  • 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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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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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 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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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 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: Fase II
  • Recruiting: Open
  • Status: Activo

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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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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 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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Controller: Fundació Hospital Universitari Vall Hebron Institut de Recerca – VHIR- G60594009.
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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.
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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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Estudio de fase 2 de la infusión de células T autólogas, diferenciadas y de sangre periférica expandidas y transducidas con un lentivirus para expresar un receptor de antígeno quimérico con especificidad anti-CD19 (A3B1) junto con regiones coestimuladoras 4-1BB y CD3z (ARI- 0001 células) en niños y adolescentes (de 0 a 18 años) con leucemia linfoblástica aguda CD19+ resistente o refractaria al tratamiento

  • Protocol code: CART19-BE-03PED
  • EudraCT code: 2022-001101-52
  • Pathology: Acute lymphoblastic leukemia
  • Pharmac: Autologous, differentiated, peripheral blood T-cells expanded and transduced with a lentivirus to express a chimeric antigen receptor with anti-CD19 specificity (A3B1) coupled with 4-1BB and CD3z co-stimulatory regions (ARI-0001 cells)
  • Promoter: Fundació Sant Joan de Déu
  • Principal investigator:  Díaz de Heredia Rubio, Maria Cristina
  • Research group:  Childhood Cancer and Blood Disorders
  • Service: Pediatric Oncohematology
  • Phase: Phase II
  • Recruiting: Open
  • Status: Activo

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Evaluación de la eficacia y seguridad de la inmunoterapia subcutánea (Beltavac®) con extracto alergénico polimerizado de mezcla de ácaros del polvo en pacientes con rinitis/rinoconjuntivitis alérgica

  • Protocol code: PRO-RCT-ACAROS-2018-01
  • EudraCT code: 2018-003427-11
  • Pathology: Allergic rhinoconjunctivitis due to exposure of house dust mites
  • Pharmac: Subcutaneous immunotherapy (Beltavac®)
  • Promoter: Probeltepharma
  • Principal investigator:  Garriga Baraut, Maria Teresa
  • Research group:  Growth and Development
  • Service: Al·lergologia i Pneumologia pediàtrica
  • Phase: Phase III
  • Recruiting: Open
  • Status: Activo

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Estudio de extensión, abierto de 52 semanas para evaluar la pimavanserina en niños y adolescentes con irritabilidad asociada al trastorno del espectro autista (TEA)

  • Protocol code: ACP-103-070
  • EudraCT code: 2021-005388-32
  • Pathology: Irritability associated with autistic disorder in children and adolescents with ASD
  • Pharmac: Pimavanserin
  • Promoter: Acadia
  • Principal investigator:  Gisbert Gustemps, Laura
  • Research group:  Psiquiatria, salut mental i addiccions
  • Service: Psiquiatria
  • Phase: Phase II
  • Recruiting: Closed
  • Status: Cerrado

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Estudio de fase 2 abierto para evaluar la seguridad, tolerabilidad, farmacocinética y farmacodinámica de sotatercept (MK-7962) en niños de entre uno y menos de 18 años con HAP que reciben el tratamiento habitual.

  • Protocol code: MK-7962-008
  • EudraCT code: 2022-000478-25
  • Pathology: Pulmonary arterial hypertension
  • Pharmac: Sotatercept (MK-7962)
  • Promoter: Merck Sharp & Dohme
  • Principal investigator:  Moreno Galdó, Antonio
  • Research group:  Growth and Development
  • Service: Al·lergologia i Pneumologia pediàtrica
  • Phase: Phase II
  • Recruiting: Closed
  • Status: Finalizado

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Ensayo de extensión abierto para evaluar la seguridad a largo plazo de KVD900, un inhibidor oral de la calicreína plasmática, para el tratamiento ademanda de las crisis de angioedema en pacientes adolescentes y adultos con angioedema hereditario de tipo I o II

  • Protocol code: KVD900-302
  • EudraCT code: 2021-001176-42
  • Pathology: Hereditary Angioedema Type I or II
  • Pharmac: KVD900 (oral Plasma Kallikrein Inhibitor)
  • Promoter: Kalvista Pharmaceuticals
  • Principal investigator:  Guilarte Clavero, Mar
  • Research group:  Malalties sistèmiques
  • Service: Al·lergologia
  • Phase: Phase III
  • Recruiting: Closed
  • Status: Cerrado

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Estudio de fase I/II multicéntrico en el que se evalúan la eficacia y la seguridad de ribociclib (LEE011) en combinación con topotecán y temozolomida (TOTEM) en pacientes pediátricos con neuroblastoma recidivante o refractario y otros tumores sólidos

  • Protocol code: CLEE011Q12101
  • EudraCT code: 2021-005617-14
  • Pathology: Relapsed or refractory neuroblastoma and other solid tumors (including medulloblastoma, high grade glioma, malignant rhabdoid tumors, and rhabdomyosarcoma)
  • Pharmac: Ribociclib (LEE011) in combination with topotecan and temozolomide (TOTEM)
  • Promoter: Novartis
  • Principal investigator:  Moreno Martín-Retortillo, Lucas
  • Research group:  Childhood Cancer and Blood Disorders
  • Service: Pediatric Oncohematology
  • Phase: Phase II
  • Recruiting: Open
  • Status: Activo

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Odevixibat (A4250) in children with biliary atresia (BOLD-EXT)

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Estudio en fase III, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia y la seguridad de mitapivat en sujetos pediátricos con deficiencia de piruvato cinasa que no reciben transfusiones con regularidad, seguido de un periodo de extensión abierto de 5 años

  • Protocol code: AG348-C-023
  • EudraCT code: 2021-003333-11
  • Pathology: Pyruvate Kinase Deficiency
  • Pharmac: Mitapivat
  • Promoter: Agios Pharmaceuticals
  • Principal investigator:  Murciano Carrillo, Thaïs
  • Research group:  Childhood Cancer and Blood Disorders
  • Service: Pediatric Oncohematology
  • Phase: Phase III
  • Recruiting: Closed
  • Status: Finalizado

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