Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

The primary objective of the FALCON study is to evaluate the efficacy of KL1333 on selected disease manifestations of primary mitochondrial disease (PMD) following 48 weeks of treatment. This objective involves evaluating the efficacy of KL1333 versus placebo on fatigue symptoms and impacts on daily living as well as on functional lower extremity strength and endurance. Additionally, the study evaluates the safety and tolerability of KL1333.

NCT Number	NCT05650229
Lead Sponsor	Pharming Technologies B.V.
Conditions	Primary Mitochondrial Disease
Enrollment	180 participants
Start Date	2022-12-13
Primary Completion	2027-10 (estimated)
Study Completion	2027-11 (estimated)
Updated on ClinicalTrials.gov	2026-06-26