An Interventional, Randomised, Double-blind, Parallel-group, Placebo-controlled, Flexible-dose, Adaptive Study of the Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
Abliva AB
Summary
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.
Description
The FALCON study is investigating whether the study medicine, KL1333, improves fatigue levels and physical abilities of people living with mitochondrial disease. The investigators 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 wee…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Age 18 years or older. * A confirmed PMD diagnosis caused by a known pathogenic gene mutation or deletion of the mitochondrial genome (category 6 of the International Classification of Inborn Metabolic Disorders \[ICIMD\])12 according to American College of Medical Genetics (ACMG)/Association of Molecular Pathology (AMP) criteria1, with multisystemic disease expressions, including: 1. m.3243A\>G associated MELAS-MIDD spectrum disorders, 2. single large scale mtDNA deletion associated KSS-CPEO spectrum disorders, 3. other multisystemic mtDNA-related disease (includ…
Interventions
- DrugKL1333
Twice daily
- DrugPlacebo
Twice daily
Locations (35)
- University of California, Irvine - ALS & Neuromuscular CenterOrange, California
- The Regents of the University of California - San DiegoSan Diego, California
- Children's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus LocationAurora, Colorado
- Rare Disease Research, LLCAtlanta, Georgia
- Mayo ClinicRochester, Minnesota
- Washington University School of Medicine - Center for Advanced Medicine (CAM) - Neuroscience CenterSt Louis, Missouri