Ospedale Pediatrico Bambino Gesu | Dipartimento di Oncoematologia e Terapia Cellulare e Genica

Research site

(Unclaimed)

Location

Piazza Sant'Onofrio 4, Rome, Lazio, Italy

Site insights

Top conditions

Top treatments

Ataluren

VX-445

ELX

Tezacaftor

Ivacaftor

Elexacaftor

Emapalumab

TEZ

Ceftazidime

Anti-interferon

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Data sourced from clinicaltrials.gov

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Active trials

11 of 26 total trials

A Study Evaluating the Effects of Filgotinib in Children and Teenagers With Ulcerative Colitis (Galapeduca)

The aim of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of filgotinib as a treatment for UC in children an...

Not yet enrolling

Ulcerative Colitis

Drug: Filgotinib

Status recently updated

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)

This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.The...

Not yet enrolling

Enrolling

Duchenne Muscular Dystrophy (DMD)

Drug: ENTR-601-44 - matching placebo

Drug: ENTR-601-44

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)

This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The...

Begins enrollment in 2 months

Duchenne Muscular Dystrophy (DMD)

Drug: ENTR-601-45

Drug: ENTR-601-45 - matching placebo

Atrasentan in Patients With Proteinuric Glomerular Diseases (AFFINITY)

The AFFINITY Study is a phase 2, open-label, basket study to evaluate the efficacy and safety of atrasentan in patients with proteinuric glomerular d...

Active, not recruiting

Immunoglobulin A Nephropathy

Alport Syndrome

Drug: Atrasentan

Bosutinib in Pediatric Patients With Newly Diagnosed Chronic Phase or Resistant/Intolerant Ph + Chronic Myeloid Leukemia

This is a Phase 1-2, multicenter, international, single-arm, open-label study designed to identify a recommended dose of bosutinib administered orall...

Active, not recruiting

Blastic Phase Chronic Myelogenous Leukemia

Chronic Phase Chronic Myelogenous Leukemia

Drug: Bosutinib

Effect of a Hydrolyzed Rice Protein-based Formula on Growth, Tolerance, and Health-related Quality of Life in Infants With Cow's Milk Protein Allergy. (RIGHT-GO)

The main purpose of this study is to demonstrate that the growth of infants fed with the Test Formula is non-inferior those fed with the Control Form...

Not yet enrolling

Cow Milk Protein Allergy

Other: Control Formula

Other: Test Formula

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participan...

Enrolling

Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)

Drug: Maralixibat

Other: Placebo

Interventional Study of Vosoritide for the Treatment of Children with Hypochondroplasia

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH...

Invitation-only

Hypochondroplasia

Drug: Vosoritide

Drug: Placebo

Long-Term Follow-up Protocol for Participants Treated With Gene-Modified T Cells

This is a prospective study for the long-term follow-up (LTFU) of safety and efficacy for all pediatric and adult participants exposed to Gene-modifi...

Enrolling

Neoplasms

Genetic: Gene-modified (GM) T cell therapy

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome (ASSERT-EXT)

The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS).The partici...

Enrolling

Alagille Syndrome

Drug: Odevixibat

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1...

Active, not recruiting

Cystic Fibrosis

Drug: ELX/TEZ/IVA

Drug: IVA