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Study identifier: NCT05650229 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

Condition: Primary Mitochondrial Disease  ·  Sponsor: Pharming Technologies B.V.

PhasePhase 2
Planned participants180
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Communication Manager  ·  +31 71 524 7400  ·  medinfo@pharming.com

Always discuss trial participation with your own doctor first.

Locations (55)

University of California, Irvine - ALS & Neuromuscular CenterOrange, California, United StatesRecruiting
The Regents of the University of California - San DiegoSan Diego, California, United StatesRecruiting
UCSF Movement Disorders ClinicSan Francisco, California, United StatesNot Yet Recruiting
Children's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus LocationAurora, Colorado, United StatesRecruiting
Yale University School of MedicineNew Haven, Connecticut, United StatesNot Yet Recruiting
Novel Clinical Research Center, LLCMiami, Florida, United StatesRecruiting
Rare Disease Research, LLCAtlanta, Georgia, United StatesRecruiting
IU Health University HospitalIndianapolis, Indiana, United StatesNot Yet Recruiting
Johns Hopkins University School of MedicineBaltimore, Maryland, United StatesNot Yet Recruiting
Mayo ClinicRochester, Minnesota, United StatesRecruiting
Washington University School of Medicine - Center for Advanced Medicine (CAM) - Neuroscience CenterSt Louis, Missouri, United StatesNot Yet Recruiting
Tekton Marlboro NeurologyMarlboro, New Jersey, United StatesRecruiting

+ 43 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT05650229