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

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Ataluren
Ivacaftor
Emapalumab
Tezacaftor
Elexacaftor
ELX
VX-445
Atrasentan
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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

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

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

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

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

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

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

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