Hôpital Necker-Enfants Malades

Paris 75743

11 studies enrolling now · 56 studies all time

What they study most

AOSD, Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Arginase I Deficiency, Cardiomyopathy, Cardiovascular Abnormalities, Cardiovascular Diseases, Cholesterol Ester Storage Disease(CESD), Congenital Abnormalities, Congenital Diaphragmatic Hernia, Congenital Heart Disease, Congenital Heart Disease With Fontan Circulation

Studies at this site

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome — Recruiting now
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS) — Recruiting now
Beeline: A Phase 3 Study in GRIN-related Neurodevelopmental Disorder — Recruiting now
Prospective Clinical Assessment Study in Children With Hypochondroplasia — Recruiting now
A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease — Recruiting now
Comparison of Weekly Somatrogon to Daily Genotropin in Children Born Small for Gestational Age or With Idiopathic Short Stature. — Recruiting now
A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study — Recruiting now
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD). — Recruiting now
A Study of the Efficacy and Safety of DMX-200 in Patients With FSGS Who Are Receiving an ARB — Recruiting now
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia — Recruiting now
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF — Recruiting now
A Multicenter Multinational Observational Study of Children With Hypochondroplasia — Recruiting now
Evaluation of Oral Tofacitinib in Children Aged 2 to 17 Years Old Suffering From Moderate to Severe Ulcerative Colitis — Running, not enrolling
A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension — Running, not enrolling
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) — Running, not enrolling
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE) — Running, not enrolling
Global Patient Registry of Inherited Retinal Diseases — Running, not enrolling
A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension — Running, not enrolling
A Study of Mirikizumab (LY3074828) in Pediatric Participants With Moderately to Severely or Active Ulcerative Colitis — Completed
A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHD — Running, not enrolling
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD) — Completed
A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia — Stopped early
(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis — Completed
Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE — Completed
A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis — Stopped early
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy — Completed
Multi-center Trial in Adult and Pediatric Patients With Type 1 Diabetes Using Hybrid Closed Loop System and Control at Home — Completed
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B — Completed
Safety and Efficacy Study of reSept ASD Occluder for Treating Secundum ASD — Running, not enrolling
Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects — Completed
Daratumumab Retreatment in Participants With Multiple Myeloma Who Have Been Previously Treated With Daratumumab — Stopped early
Study to Evaluate Safety and Antiviral Activity of Doses of JNJ-53718678 in Children (>=28 Days to <=3 Years) With Respiratory Syncytial Virus Infection — Stopped early
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis — Completed
Trial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) Patients — Completed
Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency — Completed
Entospletinib Plus Intensive Induction/Consolidation Chemotherapy in Newly Diagnosed NPM1-mutated AML — Stopped early
Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa — Completed
An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients. — Stopped early
A 2-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥ 2 Years Old and Young Adults With Dravet Syndrome — Completed
A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody — Completed
A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study) — Completed
Fetal Surgery for Moderate Left Sided Congenital Diaphragmatic Hernia. — Completed
Extension Study of Ataluren (PTC124) in Cystic Fibrosis — Completed
Study of Ataluren (PTC124) in Hemophilia A and B — Stopped early
Study of Ataluren (PTC124™) in Cystic Fibrosis — Completed
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) — Completed
A Long-Term Extension Study of OnabotulinumtoxinA (BOTOX®) for Urinary Incontinence Due to Neurogenic Detrusor Overactivity — Completed
Study of Ataluren (PTC124) in Cystic Fibrosis — Stopped early
Comparison of Inhaled Nitric Oxide and Oxygen in Participants Reactivity During Acute Pulmonary Vasodilator Testing — Completed
Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP) — APPROVED_FOR_MARKETING