This is an open-label, multi-centre study in subjects with a genetically confirmed
mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G
mutation who completed study KH176-202. In the KH176-203 study subjects will be receiving
KH176 100 mg BID or KH176 50 mg bid in die (BID) (as determined by the investigator based on
safety / tolerability considerations) for a year, thereby ensuring continued treatment with
KH176 after study KH176-202. A final follow-up visit is scheduled 4 weeks after the intake of
the last dose of study medication for patients not rolling over into the compassionate use
program. Primary safety data and secondary efficacy (endpoint) data will be monitored and
reviewed every three months by an independent Data Safety Monitoring Board (DSMB) to evaluate
potential risks and benefits.
Phase:
Phase 2
Details
Lead Sponsor:
Khondrion BV
Collaborators:
Certara Julius Clinical, The Netherlands ProPharma Group