Hôpital des enfants

Toulouse 31300

1 studies enrolling now · 10 studies all time

What they study most

Acute Myeloid Leukemia, Cystic Fibrosis, Duchenne Muscular Dystrophy (DMD), Hypophosphatasia, IGF1 Deficiency, Infantile Hemangioma, Osteosarcoma, Pulmonary Arterial Hypertension

Studies at this site

• 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
• Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a Cancer of the Blood — Running, not enrolling
• A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH) — Completed
• A Study to Compare the Efficacy and Safety of Ifosfamide and Etoposide With or Without Lenvatinib in Children, Adolescents and Young Adults With Relapsed and Refractory Osteosarcoma — Completed
• A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids — Stopped early
• Extension Study of Ataluren (PTC124) in Cystic Fibrosis — Completed
• Study of Ataluren (PTC124™) in Cystic Fibrosis — Completed
• Study of Ataluren (PTC124) in Cystic Fibrosis — Stopped early
• Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP) — APPROVED_FOR_MARKETING
• Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy — Completed