Istituto Giannina Gaslini

Running trials

• Not yet recruiting

  An open-label, single-arm, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients who have completed a clinical trial (parent study) in the iptacopan pediatric program.

  2026-525677-39-00· Phase 3· Novartis Pharma AG· Source

• Not yet recruiting

  Safety and Efficacy Comparison Between CHF5993 100/6 12.5µg pMDI to Seretide Evohaler 125/25µg pMDI in Adolescent Subjects With Asthma

  NCT07858123· Phase 3· Chiesi Farmaceutici S.p.A.· Source

• Not yet recruiting

  Study of the safety and efficacy of asciminib in pediatric and young adult participants with r/r Ph+ or ABL-class Ph-like ALL

  2025-522019-40-00· Phase 1/2· Novartis Pharma AG· Source

• Recruiting

  Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis

  NCT07809867· Phase 4· Istituto Giannina Gaslini· Source

• Recruiting

  International Study for Treatment of Childhood Relapsed Precursor B-Cell ALL 2020 (IntReALL BCP 2020)

  NCT07476729· Phase 3· Charite University, Berlin, Germany· Source

• Recruiting

  A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

  NCT07587242· Phase 3· Avidity Biosciences, Inc.· Source

• Recruiting

  Fight Osteosarcoma Through European Research evolving study platform from diagnosis to relapse (FOSTER evolving study platform)

  2023-505575-69-01· Phase 3· Institut Gustave Roussy· Source

• Recruiting

  A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping

  2025-523087-20-00· Phase 3· Avidity Biosciences Inc.· Source

• Recruiting

  A Study of Orforglipron (LY3502970) Compared With Dulaglutide in Pediatric Participants With Type 2 Diabetes

  NCT07668336· Phase 3· Eli Lilly and Company· Source

• Recruiting

  A Study to Learn More About How Safe Finerenone is, When it is Taken for a Longer Time With Standard Treatment, in Children and Young Adults With Heart Failure and Left Ventricular Systolic Dysfunction

  NCT07192952· Phase 3· Bayer· Source

• Recruiting

  A Phase 3 Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children with Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders

  2025-523079-44-00· Phase 3· Ascendis Pharma Endocrinology Division A/S· Source

• Recruiting

  Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)

  NCT07224360· Phase 2· CSL Behring· Source

• Active, not recruiting

  A study to learn about how well BAY 3401016 works in adults with Alport syndrome

  2024-516471-33-00· Phase 2· Bayer AG· Source

• Recruiting

  A Study to Evaluate the Efficacy and Safety of Esketamine for Reduction of Symptoms of Major Depressive Disorder

  NCT07227454· Phase 3· Janssen Research & Development, LLC· Source

• Recruiting

  Intranasal Cenegermin in Pediatric Participants with Spastic Cerebral Palsy.

  2025-522786-29-00· Phase 2· Dompe' Farmaceutici S.p.A.· Source

• Recruiting

  Phase II, multicentre, randomised, open-label trial with MAP +/- denosumab regimen for the treatment of patients with metastatic osteosarcoma

  2024-518167-35-01· Phase 2· Associazione Italiana Ematologia Oncologia Pediatrica· Source

• Active, not recruiting

  A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)

  NCT07107945· Phase 3· Boehringer Ingelheim· Source

• Recruiting

  Study on chemotherapy and experimental drugs in patients with Philadelphia Chromosome positive and ABL-class Philadelphia Chromosome-Like B-cell Acute Lymphoblastic Leukemia

  2025-520982-39-00· Phase 3· Universita Degli Studi Di Milano Bicocca· Source

• Recruiting

  A Study to Learn More About How Well Finerenone Works, How Safe it is, and How it Moves Into, Through, and Out of the Body Compared to Placebo When Taken With Standard Treatment in Children With Heart Failure and Left Ventricular Systolic Dysfunction

  NCT07188805· Phase 3· Bayer· Source

• Recruiting

  A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)

  NCT07194850· Phase 2/3· argenx· Source

• Recruiting

  Study of Ravulizumab in Pediatric Participants With Primary IgAN

  NCT07024563· Phase 3· Alexion Pharmaceuticals, Inc.· Source

• Recruiting

  An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome

  NCT05066217· Phase 3· Epygenix· Source

• Recruiting

  Study Combining Dinutuximab Beta With Two Chemotherapy Regimens in Neuroblastoma

  NCT06485947· Phase 1· Princess Maxima Center for Pediatric Oncology· Source

• Recruiting

  A Multi-Arm, Platform Trial For Relapsed Neuroblastoma

  NCT07334301· Phase 1/2· University of Birmingham· Source

• Active, not recruiting

  A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy

  NCT06413706· Phase 2· Eli Lilly and Company· Source

• Active, not recruiting

  LCH-IV International Collaborative Treatment Protocol for Children and Adolescents with Langerhans Cell Histiocytosis

  NCT02205762· Phase 2/3· St. Anna Childrens Cancer Research Institute GmbH· Source

• Recruiting

  A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

  NCT06379789· Phase 1/2· Regeneron Pharmaceuticals· Source

• Enrolling by invitation

  Long-term Safety and Tolerability of BHV-7000

  NCT06443463· Phase 2· Biohaven Therapeutics Ltd.· Source

• Recruiting

  Linfociti T virus-specifici per infezioni virali resistenti alla terapia farmacologica dopo trapianto allogenico di cellule staminali emopoietiche e in pazienti con grave immunodepressione primitiva o secondaria

  NCT05075837· Phase 1· IRCCS Istituto Giannina Gaslini· Source

• Active, not recruiting

  Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

  NCT06392009· Phase 1/2· GRIN Therapeutics, Inc.· Source

• Recruiting

  A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis

  NCT06392386· Phase 2/3· argenx· Source

• Active, not recruiting

  Study to Determine if BHV-7000 is Effective and Safe in Adults With Refractory Focal Onset Epilepsy

  NCT06309966· Phase 2/3· Biohaven Therapeutics Ltd.· Source

• Recruiting

  Current Practice of Ventilation Strategies in Children Undergoing General Anesthesia

  NCT06166706· Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)· Source

• Active, not recruiting

  A study to investigate the efficacy and safety of tezepelumab compared with placebo in children 5 to < 12 years old with severe asthma

  2022-502984-39-00· Phase 3· AstraZeneca AB· Source

• Recruiting

  European Newborn Study: Early Markers for a Better LifE

  NCT07189494· dr. M.J.N.L. Benders· Source

• Recruiting

  TCRaß+CD19+ depleted haploidentical stem cell transplantation followed by immunotherapy in patients with relapsed/resistant high-risk neuroblastoma

  2024-519089-32-02· Phase 2· IRCCS Istituto Giannina Gaslini· Source

• Active, not recruiting

  DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)

  NCT05566795· Phase 3· Day One Biopharmaceuticals, Inc.· Source

• Active, not recruiting

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

  NCT05331183· Phase 3· Vertex Pharmaceuticals Incorporated· Source

• Active, not recruiting

  A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

  NCT05444257· Phase 3· Vertex Pharmaceuticals Incorporated· Source

• Active, not recruiting

  Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)

  NCT05183035· Phase 3· PedAL BCU, LLC· Source

• Recruiting

  Asciminib Roll-over Study

  NCT04877522· Phase 4· Novartis Pharmaceuticals· Source

• Enrolling by invitation

  Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis

  NCT05374590· Phase 2/3· argenx· Source

• Recruiting

  A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescents With Active Class III or IV Lupus Nephritis and the Safety and PK of Obinutuzumab in Pediatric Participants

  NCT05039619· Phase 2· Hoffmann-La Roche· Source

• Recruiting

  Efficacy and Safety Study of Rimegepant for the Preventative Treatment of Migraine in Pediatric Subjects

  NCT05156398· Phase 3· Pfizer· Source

• Recruiting

  Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

  NCT05145127· Phase 3· Pfizer· Source

• Recruiting

  High Dose Steroids in Children With Stroke

  NCT04873583· Phase 3· Insel Gruppe AG, University Hospital Bern· Source

• Recruiting

  SIOP-HRMB: An international prospective trial on clinically high-risk medulloblastoma (HR-MB) in patients older than 3 years.

  2024-510578-25-00· Phase 3· The University Of Birmingham· Source

• Active, not recruiting

  Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

  NCT04833894· Phase 2/3· argenx· Source

• Recruiting

  Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases

  NCT05003986· Phase 2· Travere Therapeutics, Inc.· Source

• Recruiting

  Observational Study in Localized Osteosarcoma

  NCT04890067· Italian Sarcoma Group· Source

Showing 50 of 65.

Recruiting at the same time

How many trials recruit for the same indication here at once. A count, not a rating.

• Childhood cancers20 recruiting
• Acute lymphoblastic leukaemia6 recruiting
• Neuroblastoma4 recruiting
• Sarcoma4 recruiting
• Duchenne muscular dystrophy3 recruiting
• IgA nephropathy3 recruiting
• Myasthenia gravis3 recruiting
• Cerebral palsy2 recruiting
• Epilepsy2 recruiting
• Haemophilia2 recruiting
• Heart failure2 recruiting
• Asthma1 recruiting
• Chronic myeloid leukaemia1 recruiting
• Cystic fibrosis1 recruiting
• Cytomegalovirus infection1 recruiting
• Depression1 recruiting
• Immune thrombocytopenia (ITP)1 recruiting
• Lupus (SLE)1 recruiting
• Migraine1 recruiting
• Myeloproliferative neoplasms1 recruiting
• Sickle cell disease1 recruiting
• Stroke1 recruiting
• Turner syndrome1 recruiting
• Type 2 diabetes1 recruiting

Closed trials

• Recruiting

  Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis

  NCT06699849· Phase 2· CSL Behring· Source

• Completed

  Efficacy, Safety and Tolerability of BHV-7000 in Subjects with Idiopathic Generalized Epilepsy with Generalized Tonic-clonic Seizures – SHINE

  NCT06425159· Phase 2/3· Biohaven Therapeutics Ltd.· Source

• Completed

  A Two-Part Study to Assess the Effectiveness, Safety, and Blood Levels of Repeat Doses of Inhaled ETD001 in People with Cystic Fibrosis

  2023-504092-25-00· Phase 2· Enterprise Therapeutics Limited· Source

• Completed

  Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

  NCT05794139· Phase 2· NMD Pharma A/S· Source

• Completed

  Study to assess safety and identify dose of ribociclib in combination of temozolomide and topotecan (TOTEM) in patients (12 months – 21 years old) with relapsed or refractory solid tumors (Phase I), and further assess efficacy and safety in patients (12 months – 21 years old) with relapsed or refractory neuroblastoma (Phase II)

  NCT05429502· Phase 1/2· Novartis Pharma AG· Source

• Completed

  Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

  NCT05322096· Phase 2· Gedeon Richter Plc.· Source

• Completed

  Dose-finding Study Assessing the Efficacy, Safety, and Pharmacokinetics of Daridorexant in Subjects Aged 10 to < 18 Years With Insomnia Disorder

  NCT05423717· Phase 2· Idorsia Pharmaceuticals Ltd.· Source

• Completed

  A Study of Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic Arthritis

  NCT05083182· Phase 3· Janssen Research & Development, LLC· Source

• Completed

  Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

  NCT05156320· Phase 3· Scholar Rock, Inc.· Source

• Completed

  Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

  NCT05001737· Phase 3· Swedish Orphan Biovitrum· Source

• Active, not recruiting

  Pediatric Long-Term Follow-up and Rollover Study

  NCT03975829· Phase 4· Novartis Pharmaceuticals· Source

• Active, not recruiting

  Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)

  NCT04150497· Phase 1/2· Cellectis S.A.· Source

• Withdrawn

  Idelalisib With Rituximab, Ifosfamide, Carboplatin, Etoposide (RICE) in Children and Adolescents

  NCT03349346· Phase 1· Gilead Sciences· Source

• Completed

  Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

  NCT02913482· Phase 2· Hoffmann-La Roche· Source

• Completed

  Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

  NCT02500381· Phase 3· Sarepta Therapeutics, Inc.· Source

• Completed

  Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

  NCT02369731· PTC Therapeutics· Source

• Completed

  ABCB1/P-glycoprotein Expression as Biologic Stratification Factor for Patients With Non Metastatic Osteosarcoma

  NCT01459484· Phase 2· Italian Sarcoma Group· Source

Conditions

Diseases this institution publishes on or runs trials for.

• Epilepsy73 works · 4 trials
• Neuroblastoma48 works · 6 trials
• Childhood cancers29 works · 33 trials
• Cystic fibrosis25 works · 4 trials
• Spinal muscular atrophy12 works · 3 trials
• Coeliac disease10 works · 0 trials
• Duchenne muscular dystrophy9 works · 6 trials
• Aplastic anaemia4 works · 0 trials