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

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Active trials

6 of 20 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...

Begins enrollment this month
Cow Milk Protein Allergy
Other: Control Formula
Other: Test Formula

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

Trial sponsors

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PTC Therapeutics logo
Sobi logo
Agios Pharmaceuticals logo
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Cincinnati Children's Hospital Medical Center logo
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