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

Ivacaftor

Emapalumab

Tezacaftor

Elexacaftor

ELX

VX-445

Atrasentan

TEZ

Ceftazidime

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Data sourced from clinicaltrials.gov

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Active trials

7 of 21 total trials

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

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

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

Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis (VALIANT)

This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients wi...

Active, not recruiting

Membranoproliferative Glomerulonephritis (MPGN)

Membranoproliferative Glomerulonephritis

Other: Placebo

Drug: Pegcetacoplan

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