RecruitingPhase 2NCT05650229

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

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

Sponsor

Abliva AB

Enrollment

180 participants

Start Date

Dec 13, 2022

Study Type

INTERVENTIONAL

Conditions

Primary Mitochondrial Disease

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.

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study tests whether a drug called KL1333 can improve fatigue and muscle weakness in adults with primary mitochondrial disease (PMD) — a group of genetic conditions where the mitochondria (the energy-producing parts of cells) do not work properly. Common types studied include MELAS, Kearns-Sayre syndrome, CPEO, and MERRF. KL1333 is designed to improve mitochondrial energy production. You may be eligible if: - You are 18 years or older - You have a confirmed genetic diagnosis of primary mitochondrial disease (specific types: MELAS-MIDD spectrum, KSS-CPEO spectrum, or other multisystemic mtDNA disease) - You have had significant fatigue for at least 3 months - You have muscle weakness (myopathy) affecting hip or shoulder movements - You can perform at least 2 sit-to-stand repetitions in 30 seconds You may NOT be eligible if: - Your mitochondrial disease mainly causes neurodegeneration (e.g., Leigh syndrome, LHON, NARP) - Your fatigue or muscle weakness has a cause other than mitochondrial disease - You have had a stroke-like episode, frequent seizures, or status epilepticus in the past 6 months - You have a significant heart condition or abnormal ECG (QTcF too high) - You have inflammatory bowel disease, peptic ulcers, or recent GI bleeding - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGKL1333

Twice daily

DRUGPlacebo

Twice daily

Locations(35)

University of California, Irvine - ALS & Neuromuscular Center

Orange, California, United States

The Regents of the University of California - San Diego

San Diego, California, United States

Children's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus Location

Aurora, Colorado, United States

Rare Disease Research, LLC

Atlanta, Georgia, United States

Mayo Clinic

Rochester, Minnesota, United States

Washington University School of Medicine - Center for Advanced Medicine (CAM) - Neuroscience Center

St Louis, Missouri, United States

Columbia University Irving Medical Center

New York, New York, United States

Akron Children's Hospital

Akron, Ohio, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Baylor College of Medicine (BCM)

Houston, Texas, United States

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel

Brussels, Belgium

Universitair Ziekenhuis Gent

Ghent, Belgium

Universitair Ziekenhuis Leuven Gasthuisberg Campus

Leuven, Belgium

Copenhagen Neuromuscular Center, Rigshospitalet

Copenhagen, Denmark

Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin

Bordeaux, France

Hopital Roger Salengro, CHRU de Lille

Lille, France

Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2

Nice, France

CHU de NICE - Hôpital Archet 2

Nice, France

Groupe Hospitalier Pitie-Salpetriere

Paris, France

Charite - Universitaetsmedizin Berlin

Berlin, Germany

Universitaetsklinikum Halle

Halle, Germany

IRCCS Institute of Neurological Sciences of Bologna- Universita di Bologna

Bologna, Italy

Azienda Ospedaliera Universitaria Gaetano Martino Messina

Messina, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Azienda Ospedaliero Universitaria Pisana

Pisa, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Roma, Italy

Radboud University Medical Center

Nijmegen, Netherlands

CIBERER- IDIBAPS, Faculty of Medicine, University of Barcelona

Barcelona, Spain

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain

Hospital General Universitario de Catalunya

Barcelona, Spain

Hospital Universitario 12 de Octubre

Madrid, Spain

Department of Clinical Neurosciences, Addenbrooke's Hospital

Cambridge, United Kingdom

University College London Hospitals Nhs Foundation Trust

London, United Kingdom

Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust

Newcastle, United Kingdom

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