Rare diseases
Investigational molecules
Locations
374 conditions with trials, the busiest first
796 trials across Europe, recruiting first
A Study of Migalastat Safety and Effects in Children Aged 2 to 12 Years with Fabry Disease and Suitable Gene Changes
BelgiumA Study of Mibavademab for Weight Loss in Children, Adolescents and Adults with Obesity Caused by LEP Gene Mutations
Germany
The NetherlandsStudy of Zirconium-89 Girentuximab as a Diagnostic Tool for Patients with Von-Hippel Lindau Disease
ItalyFirst study of TTX-381 gene therapy safety in patients with eye problems caused by CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2)
GermanyStudy of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders
The NetherlandsStudy on the Effects of Alglucosidase Alfa Enzyme Therapy in Children and Adults with Pompe Disease
The NetherlandsStudy on the Effects of Mitapivat on Brain Blood Flow and Oxygen Use in Patients with Sickle Cell Anemia
The NetherlandsStudy on the Safety and Effectiveness of Regorafenib for Patients with Arterio-Venous Malformations Unresponsive to Standard Treatment
BelgiumStudy on the Effectiveness of Biotin and Biotin Sodium for Children with Metabolic Disorders
SpainStudy on the Effectiveness and Safety of Ultevursen for Patients with Retinitis Pigmentosa Due to USH2A Gene Mutations
FranceStudy on the Safety and Effectiveness of DT-DEC01 for Patients with Duchenne Muscular Dystrophy
PolandThe therapies tested most often in Congenital, familial and genetic disorders trials.
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