Dimethyl fumarate for NBIA (PKAN/MPAN)

Phase 2, randomised, double-blind trial of dimethyl fumarate in Neurodegeneration with brain iron accumulation (NBIA), pantothenate kinase-associated neurodegeneration (PKAN) and Mitochondrial membrane protein-associated neurodegeneration (MPAN) (3 arms), sponsored by Institute of Psychiatry and Neurology. Primary endpoints: Incidence of adverse events and serious adverse events and Disease stabilization on disease rating scales. Planned start Sep 2026; status: withdrawn.