Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study of Mavacamten for Adolescents with Symptomatic Obstructive Hypertrophic Cardiomyopathy
FranceStudy of Sotorasib Treatment in Patients with Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) with KRAS G12C Mutation Who Have Previously Received Treatment
FranceStudy on the Effects of Fidrisertib for Treating Fibrodysplasia Ossificans Progressiva in Children and Adults
BelgiumStudy of Eliglustat alone or with Imiglucerase in children and adolescents aged 2-17 years with Gaucher disease type 1 and type 3
FranceStudy on the Effects of EryDex (Dexamethasone Sodium Phosphate) for Children Aged 6-9 with Ataxia Telangiectasia
BelgiumStudy on the Long-term Safety of FLT201 for Patients with Gaucher Disease Type 1
SpainStudy on the Safety and Effects of Tolvaptan for Infants and Young Children with Autosomal Recessive Polycystic Kidney Disease
BelgiumStudy on the Effects of Dirocaftor, Posenacaftor, and Nesolicaftor for Adults with Cystic Fibrosis
BelgiumStudy on the Long-Term Safety and Effectiveness of Vosoritide for Children with Achondroplasia
GermanyLong-term safety study of garadacimab (CSL312) for prevention of hereditary angioedema attacks
CzechiaStudy on the Effects of Atidarsagene Autotemcel for Patients with Late Juvenile Metachromatic Leukodystrophy
ItalyStudy on Long-Term Safety of ZYN002 Gel for Children, Adolescents, and Young Adults with Fragile X Syndrome
IrelandThe therapies tested most often in Congenital, familial and genetic disorders trials.
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