Rare diseases
Investigational molecules
Locations
459 conditions with trials, the busiest first
1 397 trials across Europe, recruiting first
Efficacy and safety of votoplam in adults with Huntington’s disease: a randomized, double‑blind, placebo‑controlled Phase 3 trial
AustriaIndapamide for Reducing Disability Progression in Adults with Primary and Secondary Progressive Multiple Sclerosis
The NetherlandsA Phase 2a Study of Empasiprubart IV Monotherapy in Adults with AChR‑Ab Seropositive Generalized Myasthenia Gravis
BelgiumPharmacokinetics and safety of subcutaneous vs intravenous human normal immunoglobulin in adults with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
CzechiaStudy of hu14.18K322A with temozolomide and irinotecan in children and adolescents with high‑risk neuroblastoma
DenmarkSafety and Feasibility of Home Use On-Body Injection System for Subcutaneous Ocrelizumab in Adults with Multiple Sclerosis
FrancePhase IIb Open‑Label Study of Intrathecal Melpida (AAV9/AP4M1) in Patients with Hereditary Spastic Paraplegia Type 50 (SPG50)
DenmarkSafety and Tolerability of Intrathecal TRCN-1023 in Adults with Amyotrophic Lateral Sclerosis (ALS) – Randomized Placebo‑Controlled Phase 1/2 Trial
GermanyEfficacy of intravenous prasinezumab versus placebo in Parkinson's disease patients with severe GBA mutation: a 104‑week randomized double‑blind study
FranceLong‑Term Safety and Efficacy Study of ENTR‑601‑45 and ENTR‑601‑44 in Participants with Duchenne Muscular Dystrophy
BelgiumSafety and Tolerability of Oral SUN-627 in Adults with Non‑Active Progressive Multiple Sclerosis (SPMS/PPMS) – Phase 2a Study
FinlandRandomized Study of Tolebrutinib Versus Rituximab in Adults with Multiple Sclerosis
SwedenThe therapies tested most often in Nervous system disorders trials.
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