Rare diseases
Investigational molecules
Locations
Piazza Di Sant'onofrio 4, 00165 Rome, Italy
Bambino Gesu Childrens Hospital in Rome, Italy specialises in clinical trials of conditions such as Migraine, Systemic lupus erythematosus, B-cell type acute leukaemia, Generalised tonic-clonic seizure, and other specialisations. This site conducts important research.
The department is conducting studies on severe eosinophilic asthma in children, testing new biologic agents to lower attack frequency.
Ongoing trials evaluate benralizumab and tezepelumab for safety and efficacy in reducing exacerbations and improving lung function.
Research focuses on rare autoinflammatory diseases and broader autoimmune conditions, seeking targeted therapies that modulate immune pathways.
Trials assess agents such as MAS825, inebilizumab, and anifrolumab for disease control and safety in pediatric populations.
The site is evaluating novel biologics for chronic inflammatory bowel conditions, aiming to achieve durable remission in young patients.
Studies test mirikizumab, guselkumab, and long‑term vedolizumab therapy to improve mucosal healing and quality of life.
Investigations target bleeding disorders and pediatric blood cancers, exploring both replacement strategies and immune‑based treatments.
Trials include marstacimab for hemophilia, blinatumomab as post‑transplant maintenance, and protocols to refine transplant conditioning.
Pediatric rare‑disease programs evaluate disease‑modifying agents for muscle and nerve disorders, aiming to preserve function and delay progression.
Ongoing studies test givinostat, N‑Acetyl‑L‑Leucine, and gene‑replacement therapy HGT‑1110 for safety and functional improvement.
The hospital investigates treatments for inherited and hormonal conditions that affect growth, kidney function, and metabolic balance.
Clinical work includes evaluating atumelnant for adrenal disease control and BAY 3401016 to reduce albuminuria in Alport syndrome patients.
Clinical trials currently enrolling participants at this site.
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