The FALCON Study

Abliva discovers and develops medicines for the treatment of mitochondrial diseases. Lead candidate, KL1333 - a powerful regulator of the essential co-enzymes NAD⁺ and NADH, is in clinical trials. The global FALCON study is now recruiting.

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (FALCON)

Brief summary

The primary objective of the Phase 2 FALCON study is to evaluate the efficacy of KL1333 on selected disease manifestations of primary mitochondrial disease 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.

The FALCON study is currently active in Denmark, France, Spain, Belgium, the US, and the UK. Please click the button above to see study locations.

Detailed description

The FALCON study is investigating whether the study medicine, KL1333, improves fatigue levels and physical abilities of people living with mitochondrial disease. We are also evaluating the tolerability of the study medicine. For this study, the effects of KL1333 are compared with those from a placebo (a pill that looks like the study medicine but contains no active medicine). The study medicine (or placebo) is a tablet that is taken twice daily during the treatment period of 48 weeks.

Participation in the FALCON study is divided into 3 parts:

  • Screening and baseline: 8-12 weeks
  • Treatment: 48 weeks
  • Safety follow-up: 5 weeks Total duration: 61 – 65 weeks

Patients who complete the screening phase and are enrolled in the study are randomly assigned to receive either the study medicine (KL1333) or placebo (no active medication). Patients are more likely to receive the study medication than placebo (for every five people who take part, three receive KL1333 and two receive placebo). Neither the participants nor the study team know who is receiving the study medicine or placebo and participants are not able to change which treatment they are assigned.

More information

See full description at NCT05650229.


To learn more about this study, you or your doctor may contact the study research staff using the contact information provided on

Please refer to this study by its identifier (NCT number): NCT05650229.

Abliva contact: