Actively Recruiting
Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
Led by Abliva AB · Updated on 2026-04-15
180
Participants Needed
35
Research Sites
254 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Confirmed diagnosis of primary mitochondrial disease caused by a known pathogenic gene mutation or mitochondrial genome deletion with multisystemic symptoms including MELAS-MIDD, KSS-CPEO, or MERRF
- History of mitochondrial fatigue for at least 3 months prior to screening
- At least moderate fatigue level with PROMIS Fatigue PMD Short form raw score 63 27 at screening and baseline
- Presence of mitochondrial myopathy defined by minimal proximal muscle weakness or exercise tolerance symptoms
- Ability to perform at least 2 repetitions and not exceed 17 (male) or 16 (female) repetitions in a 30-second sit-to-stand test at screening
- Clinically stable apart from mitochondrial disease symptoms as determined by medical evaluations
- Willingness and ability to attend study appointments within time windows
- Willingness and ability to complete electronic patient-reported outcomes
- Willingness to maintain stable diet during screening and study
- Stable dose regimen for 3 months prior to randomisation of mitochondrial disease-focused vitamins or supplements like CoQ10, vitamin B3, and L-arginine and intention to maintain during study
- Willingness to suspend idebenone treatment during study
- Female participants not pregnant and either not of childbearing potential or using highly effective contraception from consent through 36 days after last dose
- Male participants with female partners of childbearing potential agree to use condoms plus highly effective contraception during treatment and for 96 days after last dose
- Female participants agree not to breastfeed during study and for 36 days after last dose
- Female participants agree not to donate ova and male participants agree not to donate sperm during study and respective post-treatment periods
You will not qualify if you...
- Primary mitochondrial disease with mainly neurodegenerative forms such as Leigh syndrome, Leber hereditary optic neuropathy, and NARP
- Nuclear DNA mutations causing PMD or uncertain mitochondrial DNA variants
- Fatigue or muscle weakness from causes other than mitochondrial disease
- Significant cardiovascular disease or clinically significant abnormal ECG including QTcF above threshold
- Recent unstable disease or uncontrolled neurological symptoms including recent stroke-like episodes, frequent seizures, status epilepticus hospitalization, or frequent migraines
- History of inflammatory bowel disease, gastric erosions, peptic ulcers, gastrointestinal bleeding, or symptomatic gastroesophageal reflux disease in last 6 months
- Laboratory abnormalities that pose safety risks including low kidney function or elevated liver enzymes outside allowed limits
- Severe ataxia, neuropathy, balance problems, or other conditions interfering with sit-to-stand test
- Untreated or undertreated sleep apnea
- Use of idebenone within 14 days prior to first dose
- History of unstable or severe pulmonary, immunological, oncological, hepatic, or renal disease or other major illness that could interfere with study
- Unlikely to comply with protocol due to cognitive or other reasons
- Immediate family member participating in study
- Positive pregnancy test at screening or baseline
- Use of investigational drugs within 30 days or 5 half-lives prior to screening or planned use during study
- Hypersensitivity to study drug or placebo ingredients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 35 locations
1
University of California, Irvine - ALS & Neuromuscular Center
Orange, California, United States, 92868
Actively Recruiting
2
The Regents of the University of California - San Diego
San Diego, California, United States, 292093
Actively Recruiting
3
Children's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus Location
Aurora, Colorado, United States, 80045
Actively Recruiting
4
Rare Disease Research, LLC
Atlanta, Georgia, United States, 303129
Actively Recruiting
5
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
6
Washington University School of Medicine - Center for Advanced Medicine (CAM) - Neuroscience Center
St Louis, Missouri, United States, 63110
Not Yet Recruiting
7
Columbia University Irving Medical Center
New York, New York, United States, 100032
Not Yet Recruiting
8
Akron Children's Hospital
Akron, Ohio, United States, 44307
Actively Recruiting
9
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Not Yet Recruiting
10
Baylor College of Medicine (BCM)
Houston, Texas, United States, 77030
Actively Recruiting
11
The University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
Actively Recruiting
12
Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel
Brussels, Belgium
Actively Recruiting
13
Universitair Ziekenhuis Gent
Ghent, Belgium
Actively Recruiting
14
Universitair Ziekenhuis Leuven Gasthuisberg Campus
Leuven, Belgium
Actively Recruiting
15
Copenhagen Neuromuscular Center, Rigshospitalet
Copenhagen, Denmark
Actively Recruiting
16
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
Bordeaux, France
Actively Recruiting
17
Hopital Roger Salengro, CHRU de Lille
Lille, France
Actively Recruiting
18
Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2
Nice, France
Actively Recruiting
19
CHU de NICE - Hôpital Archet 2
Nice, France
Actively Recruiting
20
Groupe Hospitalier Pitie-Salpetriere
Paris, France
Actively Recruiting
21
Charite - Universitaetsmedizin Berlin
Berlin, Germany, 10117
Actively Recruiting
22
Universitaetsklinikum Halle
Halle, Germany, 6120
Actively Recruiting
23
IRCCS Institute of Neurological Sciences of Bologna- Universita di Bologna
Bologna, Italy, 40139
Actively Recruiting
24
Azienda Ospedaliera Universitaria Gaetano Martino Messina
Messina, Italy, 98125
Actively Recruiting
25
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy, 20133
Actively Recruiting
26
Azienda Ospedaliero Universitaria Pisana
Pisa, Italy, 56126
Actively Recruiting
27
Fondazione Policlinico Universitario Agostino Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, Italy, 00168
Actively Recruiting
28
Radboud University Medical Center
Nijmegen, Netherlands, 6525
Actively Recruiting
29
CIBERER- IDIBAPS, Faculty of Medicine, University of Barcelona
Barcelona, Spain
Actively Recruiting
30
Hospital de la Santa Creu i Sant Pau
Barcelona, Spain
Completed
31
Hospital General Universitario de Catalunya
Barcelona, Spain
Actively Recruiting
32
Hospital Universitario 12 de Octubre
Madrid, Spain
Actively Recruiting
33
Department of Clinical Neurosciences, Addenbrooke's Hospital
Cambridge, United Kingdom
Actively Recruiting
34
University College London Hospitals Nhs Foundation Trust
London, United Kingdom
Actively Recruiting
35
Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Newcastle, United Kingdom
Actively Recruiting
Research Team
C
Communication Manager
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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