FALCON study: Efficacy of KL1333 in adult patients with primary mitochondrial disease

Status: Closed to recruitment for Wave 1 of the study. Wave 2 recruitment to commence in the near future.

ABLIVA Falcon Study logo
ABLIVA logo

Background

Mitochondria are found in nearly all cells of the body and their job is to make the energy the body needs in order to function properly. When there are genetic mutations in the mitochondrial DNA (mtDNA) or nuclear DNA (nDNA), mitochondria are not able to make energy as they should. Such mitochondrial disorders may affect any part of the body, but more frequently the brain, heart or muscles, which require a lot of energy. Some common symptoms of primary mitochondrial disease (PMD) are fatigue, myopathy (muscle disease), exercise intolerance, and signs of metabolic dysfunction (e.g., diabetes, a serious condition where your blood glucose level is too high). KL1333 works by promoting energy production in the cells. Thus, it is hoped that KL1333 can reduce the symptoms of PMD.

Objectives

The aims for this study are to:

  • Evaluate the effect of the study drug (KL1333 vs placebo) on fatigue symptoms and impacts on daily living
  • Evaluate the effect of the study drug (KL1333 vs placebo) on functional extremity strength and endurance

Research aims

The main aim of this study is to learn how well the study drug works and how safe the study drug is, compared with placebo. A placebo is an inactive material that looks like the study drug but does not have any active study drug.

Who can take part?

  • Individuals with a confirmed mitochondrial disease diagnosis due to a known disease-causing gene mutation or deletion in the mitochondrial DNA
  • Individuals experiencing chronic mitochondrial fatigue and myopathy (muscle weakness and/or exercise intolerance)

We will ask participants to:

  • Attend at least 8 visits to Addenbrookes Hospital, Cambridge, over a 65 week period
  • Take study medication (KL1333 or placebo) daily in the morning and evening, following instructions provided
  • Complete all study assessments, including medical history, physical examinations, blood samples, electronic questionnaires and exercise assessment

Interim Results

For Wave 1 results, please visit the FALCON study news pages.

Study Website

For further information about the study, please visit the FALCON study webpages or the Lily Foundation webpages

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