Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
A Study of Zanidatamab for Patients with Previously Treated HER2-Expressing Solid Tumors
FranceTesting dapagliflozin to slow kidney function decline in patients with autosomal dominant polycystic kidney disease
AustriaA Study Testing AOC 1020 to Improve Movement in Adults with Facioscapulohumeral Muscular Dystrophy
DenmarkA Study of Repinatrabit Compared to Placebo for Treatment of Patients with Phenylketonuria
CzechiaA Study of Mibavademab for Weight Loss in Children, Adolescents and Adults with Obesity Caused by LEP Gene Mutations
GermanyStudy of lidocaine to reduce opioid use in patients with sickle cell disease experiencing severe pain crisis
FranceLong-Term Study of Vosoritide Safety and Effectiveness in Children with Hypochondroplasia Until Adult Height is Reached
FranceA study testing sirolimus to slow disease progression in patients with familial adenomatous polyposis
DenmarkA Study of ADX-324 to Prevent Attacks in Patients With Hereditary Angioedema
AustriaTitle: Evaluating low-dose prednisone with standard therapy to improve heart function in patients with cardiac manifestations of Fabry disease
PolandStudy of Apitegromab Treatment in Children Under 2 Years Old with Spinal Muscular Atrophy (SMA)
BelgiumStudy of alpelisib in children and adults with PIK3CA-Related Overgrowth Spectrum (PROS)
AustriaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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