Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
A study to test how etavopivat works and its safety in children with sickle cell disease
FranceA Study Testing Atumelnant Compared to Placebo in Adults with Classic Congenital Adrenal Hyperplasia to Reduce Steroid Dose While Controlling Hormones
AustriaA Study of Atumelnant for Children with Congenital Adrenal Hyperplasia
BelgiumA Study of Enlicitide for Safety and Effectiveness in Children and Adolescents with Heterozygous Familial Hypercholesterolemia
BelgiumA study testing the safety and effectiveness of infigratinib compared to placebo in infants and young children with achondroplasia
NorwayA study testing the safety and effects of ADS-019 in adults aged 18 to 65 years with type 1 myotonic dystrophy
BelgiumA study comparing under-the-skin and into-the-vein alpha1-proteinase inhibitor treatment in patients with alpha1-antitrypsin deficiency
DenmarkStudy of Cenegermin Nasal Spray for Safety and Tolerability in Children with Spastic Cerebral Palsy
ItalyA study using MPPF imaging to examine serotonin pathways in the brainstem of patients with Dravet Syndrome epilepsy
FranceComparing morphine alone to morphine and lidocaine for pain relief in sickle cell anemia patients with vaso-occlusive crisis
BelgiumA study testing lenadogene nolparvovec gene therapy injections into the eye to improve vision in patients with ND4 Leber Hereditary Optic Neuropathy
FranceLong-term study of bitopertin safety and effectiveness in patients with erythropoietic protoporphyria or X-linked protoporphyria
BelgiumThe therapies tested most often in Congenital, familial and genetic disorders trials.
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