Rare diseases
Investigational molecules
Locations
375 conditions with trials, the busiest first
803 trials across Europe, recruiting first
Phase 2/3 Randomized Study of SKY-0515 for Safety and Efficacy in Adults with Huntington’s Disease
GermanyStudy of melpida with drug combination in patients with Hereditary Spastic Paraplegia Type 50 (SPG50)
SpainStudy of REC-4881 in Adults with Familial Adenomatous Polyposis to Assess Safety and Efficacy
GermanySafety and Tolerability Study of Subcutaneous Pegtibatinase in Adults and Children with Classical Homocystinuria (Phase 1/2)
FranceEffect of Stopping Long‑Term Azithromycin in Stable Non‑Cystic Fibrosis Bronchiectasis Patients: A Randomized Controlled Trial
FranceSafety and Tolerability of Intrathecal ION337 in Patients with Dravet Syndrome
BelgiumSafety and Efficacy Study of FOXP3‑T4 and Aldesleukin in Patients with IPEX Syndrome
FrancePhase 2 Study of AGA2115 in Adults with Osteogenesis Imperfecta (COL1A1/COL1A2 Variants) Evaluating Bone Density
DenmarkLong‑Term Monitoring of Malignancy Risk in Early‑Onset Metachromatic Leukodystrophy Patients Treated with atidarsagene autotemcel
FranceLong-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)
DenmarkLong‑Term Effects of Sirolimus on Volume and Color of Port‑Wine Stain in Patients with Vascular Malformation Undergoing Surgery and Laser Therapy
NorwaySafety and pharmacodynamics of cangrelor tetrasodium in paediatric patients with congenital heart disease undergoing percutaneous vascular procedures
AustriaThe therapies tested most often in Congenital, familial and genetic disorders trials.
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