Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
A study of navenibart for long-term safety in patients with hereditary angioedema
AustriaA 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)
DenmarkLong‑Term Safety Study of ADX‑324 in Participants with Hereditary Angioedema (Phase 3 Extension)
AustriaLong‑Term Safety Follow‑up of Ornithine Transcarbamylase Deficiency Patients Treated with ECUR‑506D and ECUR‑506A
FranceLong‑Term Safety and Efficacy Study of ENTR‑601‑45 and ENTR‑601‑44 in Participants with Duchenne Muscular Dystrophy
BelgiumEfficacy of efanesoctocog alfa in reducing synovitis in patients with congenital hemophilia A – a multicenter, randomized, open‑label phase 3 trial
AustriaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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