Rare diseases
Investigational molecules
Locations
377 conditions with trials, the busiest first
796 trials across Europe, recruiting first
A study of navenibart for long-term safety in patients with hereditary angioedema
AustriaA study to evaluate the effectiveness of bleomycin sulfate for treating patients with low-flow vascular malformations using electrosclerotherapy.
SloveniaA study to evaluate the safety and effectiveness of surlorian in adults with RYR1-related myopathy
FranceStudy on the Effects of Bisoprolol and Verapamil in Patients with Non-Obstructive Hypertrophic Cardiomyopathy
DenmarkPhase 3 Study of Pridopidine Hydrochloride for Safety and Efficacy in Adults with Huntington’s Disease
BelgiumA Phase 2 Study of MZE782 Safety, Tolerability, and Efficacy in Adults with Phenylketonuria
FranceA Phase 3 Study of Engasertib to Evaluate Safety and Effectiveness in Adults with Moderate to Severe Hereditary Hemorrhagic Telangiectasia
BelgiumZodasiran for Adolescents With Homozygous Familial Hypercholesterolemia: A Phase 3 Study of Efficacy and Safety
DenmarkEfficacy and safety of votoplam in adults with Huntington’s disease: a randomized, double‑blind, placebo‑controlled Phase 3 trial
AustriaPhase 2 Study of ALN-6400 Safety and Tolerability in Female Patients with Von Willebrand Disease and Heavy Menstrual Bleeding
GermanyStudy of Short‑and Long‑Term Hemodynamic and Physiological Effects of Mavacamten in Patients with Obstructive Hypertrophic Cardiomyopathy
SwedenPhase IIb Open‑Label Study of Intrathecal Melpida (AAV9/AP4M1) in Patients with Hereditary Spastic Paraplegia Type 50 (SPG50)
DenmarkThe 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.
