Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
Study of fampridine treatment for patients with spinocerebellar ataxia SCA27B caused by FGF14 gene mutation
FranceStudy of bitopertin to improve sunlight tolerance in adults with erythropoietic protoporphyria or X-linked protoporphyria
BelgiumStudy of zorevunersen to reduce seizures in patients with Dravet syndrome
FranceStudy of drug interactions between leriglitazone, gemfibrozil, itraconazole, and carbamazepine and food effects in healthy male volunteers for adrenoleukodystrophy treatment
PolandStudy of laroprovstat (AZD0780) tablets to lower cholesterol levels in adults with inherited high cholesterol condition (heterozygous familial hypercholesterolemia)
BulgariaSafety Study of Switching from Current Treatment to Garadacimab in Patients Age 12 and Older with Hereditary Angioedema
GermanySafety and effectiveness study of inhaled ARCT-032 (mRNA therapy) in adults with Cystic Fibrosis
GreeceStudy of ABBV-CLS-628 to evaluate safety and effectiveness in adults with Autosomal Dominant Polycystic Kidney Disease
BelgiumStudy on the Effects of Tebapivat and AG-946 in Patients with Sickle Cell Disease
BelgiumStudy on the Effects of Leniolisib in Patients with Activated Phosphoinositide 3-Kinase Delta Syndrome
GermanyStudy on the Effects and Safety of GLM101 for Patients with PMM2-CDG
BelgiumStudy on Denosumab to Prevent Breast Cancer in Women with BRCA1 Mutation
AustriaThe therapies tested most often in Congenital, familial and genetic disorders trials.
Tell us about your condition – we search every trial in Europe and connect you with the right site.
