Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease (FALCON)
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 and the UK. Additional sites will be activated throughout Europe and the US. Please click the button above to see study locations.
You may be eligible to enter the study if you:
- have a confirmed mitochondrial disease diagnosis due to a known disease-causing gene mutation or deletion in the mitochondrial DNA
- are experiencing chronic mitochondrial fatigue and myopathy (muscle weakness and/or exercise intolerance)
- are not involved in any other ongoing clinical study
You may not be eligible to enter the study if you:
- have a primary mitochondrial disease with predominant neurodegenerative expressions
- have a primary mitochondrial disease caused by nuclear DNA mutations
- have significant cardiovascular disease
- have a recent history of unstable disease
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.
See full description at ClinicalTrials.gov: NCT05650229.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided on clinicaltrials.gov.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05650229.
Abliva contact: firstname.lastname@example.org.