Open-label, single-arm, non-controlled trial to evaluate the safety and tolerability of treprostinil sodium in children below the age of 18 years with pulmonary arterial hypertension (PAH)]

• Protocol code: TREPaed
• EudraCT code: No aplica
• Research group: Growth and Development
• Service: General Pediatrics and Specialties
• Principal investigator:  Moreno Galdó, Antonio
• Pathology: Malalties del sistema respiratori
• Phase: Fase III

Estudio de fase 2, aleatorizado, abierto y controlado para evaluar la eficacia y la seguridad de rapcabtagene autoleucel frente a un comparador en participantes con miopatías inflamatorias idiopáticas refractarias graves

• Protocol code: CYTB323L12201
• EudraCT code: No aplica
• Research group: Systemic Diseases
• Service: Internal Medicine
• Principal investigator:  Selva O'Callaghan, Albert
• Pathology: Malalties del sistema osteomuscular i del teixit connectiu
• Phase: Fase II

A MULTI-CENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED TRIAL TO ASSESS EFFICACY AND SAFETY OF OMECAMTIV MECARBIL IN PATIENTS WITH SYMPTOMATIC HEART FAILURE WITH SEVERELY REDUCED EJECTION FRACTION

• Protocol code: CY 1033
• EudraCT code: No aplica
• Research group: Cardiovascular Diseases
• Service: Cardiology
• Principal investigator:  Méndez Fernández, Ana Belén
• Pathology: Malalties del sistema circulatori
• Phase: Fase III

Estudio de fase 3, multicéntrico y abierto para evaluar la farmacocinética, la farmacodinamia, la eficacia y la seguridad de Ravulizumab en participantes pediátricos (de 2 a <18 años de edad) con Nefropatía primaria por Inmunoglobulina A (NIgA)

• Protocol code: ALXN1210-IgAN-325
• EudraCT code: No aplica
• Research group: Kidney Physiopathology
• Service: Pediatric Nephrology
• Principal investigator:  Ariceta Iraola, Gema
• Pathology: Símptomes, signes i troballes anormals clíniques i de laboratori, no classificades enlloc més
• Phase: Fase III
• Status: Recruiting volunteers

Estudio aleatorizado, doble ciego para evaluar la eficacia y laseguridad de CAL101 en pacientes con fibrosis pulmonar idiopática.

• Protocol code: CAL-101-201
• EudraCT code: No aplica
• Research group: Pneumology
• Service: Pneumology
• Principal investigator:  Villar Gomez, Ana
• Pathology: Malalties del sistema respiratori
• Phase: Fase II

A phase 1 open-label trial to evaluate the safety, tolerability, pharmacodynamics, pharmacokinetics, and immunogenicity of subcutaneous VIS171 in participants with autoimmune disease(s).

• Protocol code: VIS171-103
• EudraCT code: _
• Research group: Nephrology and kidney transplantation
• Service: Nephrology
• Principal investigator:  Sans Atxer, Laia
• Pathology: Símptomes, signes i troballes anormals clíniques i de laboratori, no classificades enlloc més
• Phase: Fase I

Estudio de fase 1b que combina dinutuximab beta (DB) con regímenes de quimioterapia de inducción en pacientes con neuroblastoma de alto riesgo de reciente diagnóstico.

• Protocol code: SIOPEN-PILOT01
• EudraCT code: No aplica
• Research group: Childhood Cancer and Blood Disorders
• Service: Pediatric Oncohematology
• Principal investigator:  Moreno Martín-Retortillo, Lucas
• Pathology: Tumors
• Phase: Fase I

A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)

• Protocol code: ENTR-601-45-201
• EudraCT code: _
• Research group: Therapeutics and Innovations in Neuropediatrics and other paediatric rare diseases
• Service: General Pediatrics and Specialties
• Principal investigator:  Gómez Andrés, David
• Pathology: Malalties del sistema nerviós
• Phase: Fase I

AFFIRM: A Randomized, double-blind, placebo-controlled, study to evaluate the effect of Seladelpar on clinical outcomes in patients with primary biliary cholangitis (PBC) and compensated cirrhosis.

• Protocol code: CB8025-41837
• EudraCT code: _
• Research group: Liver Diseases
• Service: Hepatology
• Principal investigator:  Villagrasa Vilella, Ares Aurora
• Pathology: Símptomes, signes i troballes anormals clíniques i de laboratori, no classificades enlloc més
• Phase: Fase III