Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] – Therapeutics [E02]

The Cardiology Department at Nosokomeio Paidon I Agia Sofia is advancing novel diagnostic tools and therapeutic devices to improve patient monitoring and treatment delivery.

  • Development of pediatric pharmacokinetic profiling for targeted therapies
  • Implementation of advanced imaging for early detection of cardiac anomalies
  • Evaluation of new device-based drug delivery systems in children

These efforts aim to refine precision medicine approaches across multiple disease areas.

Diseases [C] – Bacterial Infections and Mycoses [C01]

Clinical studies focus on complicated urinary tract infections and acute pyelonephritis in pediatric patients, testing newer antimicrobial regimens.

  • Assessment of meropenem‑vaborbactam safety in children
  • Comparison of treatment durations for acute pyelonephritis
  • Exploration of resistance patterns to guide therapy

The goal is to enhance outcomes and reduce recurrence of severe bacterial infections.

Diseases [C] – Cardiovascular Diseases [C14]

Research targets heart failure due to left ventricular systolic dysfunction and familial hypercholesterolemia, evaluating novel agents and combination therapies.

  • Long‑term safety of finerenone added to ACE inhibitors
  • Impact on proteinuria and NT‑proBNP levels
  • LDL‑C reduction with inclisiran in heterozygous familial hypercholesterolemia

These trials seek to improve cardiac function and reduce cardiovascular risk in children and adolescents.

Diseases [C] – Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Studies address growth disorders such as Turner syndrome, Noonan syndrome, and short stature, comparing growth hormone regimens.

  • Weekly somapacitan versus daily Norditropin® effectiveness
  • Height velocity outcomes across different growth indications
  • Safety monitoring of long‑term hormone therapy

The aim is to support optimal growth trajectories in affected children.

Diseases [C] – Digestive System Diseases [C06]

The site conducts trials on inflammatory bowel disease, including Crohn’s disease and ulcerative colitis, testing biologics and small molecules.

  • Vedolizumab maintenance therapy efficacy
  • Upadacitinib for clinical remission and endoscopic response
  • Evaluation of mucosal healing with novel agents

These investigations aim to achieve durable remission and improve quality of life for pediatric patients.

Diseases [C] – Hemic and Lymphatic Diseases [C15]

Ongoing research evaluates treatments for hemophilia and thalassemia, focusing on prophylaxis and transfusion reduction.

  • Concizumab prophylaxis effectiveness in hemophilia A/B with inhibitors
  • SP‑420 dose‑response in transfusion‑dependent α/β‑thalassemia
  • Mitapivat impact on anemia in non‑transfusion‑dependent thalassemia

The objective is to lessen bleeding episodes and transfusion burden while enhancing patient safety.

Diseases [C] – Hormonal diseases [C19]

Clinical trials aim to improve management of congenital adrenal hyperplasia and related endocrine disorders.

  • Crinecerfont efficacy in reducing adrenal steroid levels
  • Pharmacokinetic profiling of new hormone modulators
  • Safety monitoring of long‑term endocrine therapy

These studies seek to provide better hormonal control and reduce disease complications.

Diseases [C] – Immune System Diseases [C20]

Investigations focus on autoimmune conditions such as Behçet’s disease and juvenile idiopathic arthritis, testing targeted immunomodulators.

  • Apremilast long‑term safety in Behçet’s oral ulcers
  • Sarilumab dosing for systemic juvenile idiopathic arthritis
  • Filgotinib efficacy in polyarticular‑course juvenile idiopathic arthritis

The goal is to control inflammation, reduce flare frequency, and improve functional outcomes.

Diseases [C] – Musculoskeletal Diseases [C05]

Trials target muscular dystrophies and related disorders, evaluating gene‑based and anti‑inflammatory therapies.

  • Vamorolone safety regarding vertebral fractures in Duchenne muscular dystrophy
  • Viltolarsen intravenous dosing in boys with DMD
  • Assessing functional improvement with novel anti‑fibrotic agents

These efforts aim to preserve muscle strength and delay disease progression.

Diseases [C] – Neoplasms [C04]

The department participates in pediatric oncology studies for neuroblastoma, ependymoma, and rhabdomyosarcoma, exploring intensified chemotherapy and immunotherapy.

  • High‑dose chemotherapy versus tandem regimens in high‑risk neuroblastoma
  • CAR‑T cell therapy safety and efficacy in hematologic malignancies
  • Targeted RAF inhibition in low‑grade glioma

Objectives include improving event‑free survival and reducing late sequelae.

Diseases [C] – Nutritional and Metabolic Diseases [C18]

Research addresses iron overload and metabolic complications in thalassemia and sickle cell disease, testing chelation and metabolic modulators.

  • REGN7999 subcutaneous dosing for iron overload reduction
  • Etavopivat efficacy in sickle cell disease anemia management
  • Rilzabrutinib prevention of vaso‑occlusive crises

The aim is to improve organ health and reduce disease‑related complications.

Diseases [C] – Pathological Conditions, Signs and Symptoms [C23]

Studies evaluate biomarkers and clinical signs such as proteinuria and renal dysfunction in chronic kidney disease.

  • Finerenone impact on urine protein excretion
  • Long‑term safety of finerenone combined with ACEI/ARB
  • Assessment of renal outcomes in pediatric heart failure patients

These trials seek to mitigate renal injury and enhance overall patient prognosis.