Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Testing Gene Therapy AAV9/AP4M1 in Patients with Hereditary Spastic Paraplegia Type 50
ItalyA study testing sonlicromanol in adults with genetically confirmed mitochondrial DNA 3243A>G variant causing mitochondrial disease
DenmarkTesting the Safety and Effectiveness of CTH120 Compared to Placebo in Adult Men with Fragile X Syndrome
SpainA study testing gene therapy with DREAM01 and imatinib for patients with severe sickle cell disease to improve red blood cell production
FranceA Study of BCX17725 Safety and Effects in Healthy People and Patients with Netherton Syndrome
FranceA study testing fitusiran to prevent bleeding in boys aged 1 to 11 years with hemophilia A or B with or without inhibitors
BelgiumA Study Testing the Safety and Tolerability of ALE1 in Healthy Adults and Adults with Hypophosphatasia
GermanyA study of mirdametinib for infants and toddlers under 24 months of age with neurofibromatosis type 1 associated plexiform neurofibromas
GermanyA Study of Gilteritinib to Eliminate Remaining Cancer Cells in Patients with Acute Myeloid Leukemia and FLT3-ITD Mutation
GreeceStudy of AEF0217 compared to placebo for improving behavior and thinking skills in adults and older adolescents with Down syndrome
FranceA study of guanabenz acetate for children with vanishing white matter disease who started treatment in early childhood
The NetherlandsA study testing ninerafaxstat compared to placebo in patients with symptomatic non-obstructive hypertrophic cardiomyopathy
AustriaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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