Ospedale Pediatrico Bambino Gesu'
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Neoplasms
The Ospedale Pediatrico Bambino Gesu' is a leading hub for pediatric oncology, where investigators are testing innovative cellular and molecular therapies to improve survival for children with blood and solid cancers.
- Acute lymphoblastic leukemia and high‑risk B‑cell ALL
- Relapsed or refractory acute myeloid leukemia
- Neuroblastoma and other solid tumors
- Lymphoblastic lymphoma (B‑cell and T‑cell subtypes)
- Anaplastic large cell lymphoma and ALK‑positive malignancies
With 84 investigators across 25 trials, the site emphasizes safety, tolerability, and long‑term outcomes of novel agents such as CAR‑T cells and targeted kinase inhibitors.
Hemic and Lymphatic Diseases
Research in this area focuses on rare blood disorders and lymphatic conditions, aiming to evaluate new targeted drugs and gene‑based treatments for pediatric patients.
- Chronic myeloid leukemia (CML) in children
- Severe hemophilia A requiring factor replacement
- Beta (β)‑thalassemia (transfusion‑dependent and non‑transfusion‑dependent)
- Refractory systemic autoimmune diseases with hematologic involvement
- Proteinuria associated with lymphatic disorders
The program seeks to define optimal dosing and assess clinical activity while prioritizing patient safety.
Immune System Diseases
The department explores therapies that modulate the immune response, including CAR‑T approaches for autoimmune conditions and novel immunosuppressive strategies.
- Refractory systemic autoimmune diseases (e.g., SLE, systemic sclerosis)
- Steroid‑refractory acute graft‑versus‑host disease
- Anti‑CD19 CAR‑T therapy for B‑cell driven autoimmunity
- CD7‑CAR T cells for relapsed/refractory T‑cell ALL/LBL
- Immune‑related adverse events after prior CAR‑T treatment
Studies aim to improve disease control while monitoring long‑term immune safety profiles.
Nervous System Diseases
Clinical investigations target severe epilepsy syndromes and demyelinating disorders, testing both disease‑modifying drugs and adjunctive therapies.
- Dravet syndrome and Lennox‑Gastaut syndrome
- Relapsing‑remitting multiple sclerosis in children
- Adjunctive soticlestat therapy with standard anti‑seizure regimens
- Long‑term safety of novel agents for refractory seizures
- Neurodevelopmental outcomes in treated pediatric cohorts
The goal is to assess efficacy in seizure reduction and disease progression while ensuring tolerability in young patients.
Cardiovascular Diseases
Studies address inherited lipid disorders and renal‑cardiac complications, evaluating long‑term lipid‑lowering and renoprotective agents.
- Familial hypercholesterolemia (heterozygous and homozygous)
- Inclisiran for sustained LDL‑cholesterol reduction
- Finerenone combined with ACE‑inhibitor or ARB in chronic kidney disease
- Proteinuria management in pediatric renal disease
- Cardiovascular risk monitoring in metabolic disorders
Research emphasizes safety over extended treatment periods and impact on cardiovascular outcomes.
Congenital, Hereditary, and Neonatal Diseases
The site conducts trials on inherited metabolic and genetic conditions, focusing on enzyme replacement and gene‑based therapies for children.
- Gaucher disease type I and type III
- Hypophosphatasia and related bone metabolism disorders
- Beta (β)‑thalassemia with novel luspatercept therapy
- Early‑onset metabolic syndromes with genetic confirmation
- Neonatal screening follow‑up studies
Objectives include establishing safe dosing, improving quality of life, and evaluating long‑term disease control.
Nutritional and Metabolic Diseases
Research targets metabolic bone disease, iron‑related disorders, and nutrition‑linked conditions, aiming to enhance therapeutic options for pediatric patients.
- Hypophosphatasia treatment with ALXN1850
- Beta (β)‑thalassemia management with luspatercept
- Nutritional support strategies for chronic kidney disease
- Metabolic monitoring in children receiving enzyme therapies
- Bone health outcomes in long‑term metabolic disease management
Studies prioritize efficacy, safety, and functional improvement in daily activities.
Skin and Connective Tissue Diseases
Investigations include connective‑tissue manifestations of autoimmune disease and rare dermatologic conditions, testing targeted immunotherapies.
- Systemic sclerosis and related skin fibrosis
- Dermatomyositis with refractory disease activity
- Autoimmune cutaneous involvement in systemic lupus erythematosus
- Novel biologic agents for pediatric connective‑tissue disorders
- Quality‑of‑life assessments in treated cohorts
The focus is on reducing disease activity while monitoring safety in a pediatric population.
Site Overview
Activity Indicator
Last 6 months
Trial Flow
How this site's trials are distributed across therapeutic areas and active substances (IMPs), and how they're positioned within their overall lifecycle status. Flow thickness represents the number of unique trials.
Top 4 of 27 therapeutic areas and 15 of 410 IMPs, ranked by number of trials.
Compounds & Molecules Timeline
The time span of clinical-trial activity for the main active substances (IMPs) studied at this site over the last 8 years. Each bar runs from the earliest trial start to the most recent end; a substance with a trial still open has no recorded end and is shown as ongoing.
Top 40 of 394 compounds, ranked by number of trials.
Sponsor & Partnership
Top sponsors by active trials, and the ones that have come back for more than one study.
Sponsor Network
Top 90 by active trials.
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Partnership History
Sponsors with repeated collaboration (≥ 2 trials).
- 1Novartis Pharma AG17trials
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Sponsor Network Graph
How sponsors and therapeutic areas connect across this site's active trials: each link is at least one active trial shared between them, and bigger nodes mean more activity. Click a node to see its exact connections.
Country Benchmark
Compared to 566 other sites in Italy
Therapeutic Area Benchmark — Italy
Select a therapeutic area to compare against other sites in Italy.
Competitor Comparison
Similar sites in the same country running a comparable number of trials (within ±30%), scored on how closely their trial portfolios match this one. Higher percentages mean a more similar profile.
Country: Italy
Market Share
Country: Italy
Therapeutic Area Market Share
Select a therapeutic area to view its disease coverage breakdown.
Heat Maps & Advanced Analytics
How often specific conditions appear in trials across this site and comparable sites from the same country. Each row is a site, each column a condition, and each cell the number of trials on it — darker means more activity.
Other Trials in Ospedale Pediatrico Bambino Gesu'
lock Showing 1 of 298 trials — the rest is available on a paid plan.
| Trial | Sponsor | Phase | Status | Start Date | End Date |
|---|---|---|---|---|---|
| (21466) A multicenter, randomized, double-blind, placebo-controlled, Phase 3 study to investigate the efficacy, safety, and PK/ PD of finerenone, in addition to standard-of-care, in pediatric patients, 6 months to < 18 years of age, with heart failure (HF) and left ventricular systolic dysfunction (LVSD) | Bayer AG | Phase III | Recruiting | Jan 2026 | — |
IMP list
lock Showing 1 of 410 compounds — the rest is available on a paid plan.
| IMP | Trials | Status | First start | Last start |
|---|---|---|---|---|
| Cyclophosphamide | 22 | Unknown | 2014-03-27 | 2025-11-26 |
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