This a randomized placebo controlled, double-blind phase II study to explore the
pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years)
with genetically confirmed mitochondrial disease of which the gene defect is known to
decrease one or more oxidative phosphorylation system enzymes and who suffer from motor
symptoms ("KHENERGYC").
Phase:
Phase 2
Details
Lead Sponsor:
Khondrion BV
Collaborators:
Ardena Author Certara Europees Fonds voor Regionale Ontwikkeling (EFRO) Julius Clinical, The Netherlands ProPharma Group