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Primary Mitochondrial DiseasePhase 2FALCONNCT05650229

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

A Phase 2 study testing KL1333 in people with Primary Mitochondrial Disease, run by Pharming Technologies B.V. at 55 sites in 11 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Primary Mitochondrial Disease
What is being tested?
KL1333
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
180 participants
Where
55 sitesin 11 countries
Study dates
13 Dec 2022to Nov 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:
  • m.3243A>G associated MELAS-MIDD spectrum disorders,
  • single large scale mtDNA deletion associated KSS-CPEO spectrum disorders,
  • other multisystemic mtDNA-related disease (including MERRF).
  • Presence of chronic mitochondrial fatigue:
Show all 24Show fewer
  • History of mitochondrial fatigue for at least 3 months prior to the Screening Visit AND
  • Presence of at least moderate level of fatigue, assessed by PROMIS® Fatigue PMD Short form raw score ≥ 27 at Screening and Baseline
  • Presence of mitochondrial myopathy defined as:
  • Myopathy (proximal muscle weakness), NMDAS Section III Clinical Assessment, item 5 score ≥ 1, which reads: "minimal reduction in hip flexion and/or shoulder abduction only (e.g. MRC 4+/5)". For the inclusion only hip flexion, but not shoulder abduction, should be taken into account. AND / OR
  • Exercise Tolerance: NMDAS Section I, item 9 score ≥ 1, which reads: "unlimited on flat - symptomatic on inclines or stairs".
  • Patients must be able to perform at least 2 repetitions and the maximal capacity must not exceed 17 repetitions in males or 16 repetitions in females in a 30s STS test at screening.
  • Clinically stable, apart from symptoms associated with the diagnosis of mitochondrial disease, at Screening and Baseline, as determined by medical history, physical examination, 12-lead ECG, vital signs measurements, and clinical laboratory evaluations at Screening, as assessed by the investigator.
  • The patient is willing and able to attend study appointments within the specified time windows.
  • Willingness and ability to complete electronic PROs.
  • Willingness to maintain a stable diet during the Screening and study periods.
  • Patients who take any mitochondrial disease-focused vitamins or supplemental therapies, including coenzyme Q10 (CoQ10), niacin/nicotinamide (vitamin B3), and L-arginine, has been on a stable dose regimen of these for 3 months prior to randomisation and intends to stay on a stable dose for the duration of the study period.
  • Willingness to suspend treatment with idebenone during the study.
  • Female patient is not pregnant and at least one of the following conditions apply:
  • Not a woman of childbearing potential (WOCBP)
  • WOCBP must agree not to try and become pregnant and use a highly effective method of contraception from the time of informed consent through at least 36 days (~5 half-lives of KL1333 plus 30 days) after the last dose of investigational medicinal product (IMP) administration.
  • Male patients with female partner(s) of childbearing potential must agree to use a male condom in addition to using highly effective contraception throughout the treatment period and for 96 days after the last dose of IMP administration. The requirement to use a male condom also applies to male patients with a pregnant or breastfeeding partner.
  • Female patients must agree not to breastfeed starting at Screening and throughout the study period and for 36 days after the last dose of IMP administration.
  • Female patients must agree to not donate ova throughout the study period and for 36 days after the last dose of IMP administration, and male patients must agree to not donate sperm throughout the study period and for 96 days after the last dose of IMP administration.

Who cannot join

  • Primary mitochondrial disease with predominant neurodegenerative phenotypes, such as, but not limited to, Leigh syndrome, Leber hereditary optic neuropathy (LHON) and Neuropathy ataxia-retinitis pigmentosa syndrome (NARP).
  • Primary mitochondrial disease nuclear DNA mutations or mutations causing mtDNA destabilisation. Genetic mtDNA variants of uncertain significance, likely pathogenic, or pathogenic mutations with degrees of heteroplasmy below what can be considered to definitely cause PMD.
  • General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator.
  • Significant cardiovascular disease (e.g., sustained or symptomatic arrhythmia; dilated heart chambers or reduced function; Mobitz II atrioventricular block or greater) OR abnormal ECG that is clinically significant, as determined by the investigator. Any QTcF > 450 msec for male patients and > 470 msec for female patients is exclusionary. In the case of an exclusionary QTcF, the ECG can be repeated twice and the average of 3 QTcF intervals should be used to determine the QTcF eligibility.
  • Recent history of unstable disease, inadequately controlled neurological manifestations or not recovered from stroke-like episodes including but not limited to:
  • stroke-like episodes within the last 6 months
Show all 23Show fewer
  • more than 1 seizure/month within the last 6 months
  • hospitalised for Status Epilepticus within the last 6 months
  • more than 4 days of migraine episodes/month within the last 6 months
  • History of inflammatory bowel disease, gastric erosions, peptic ulcer disease, or gastrointestinal bleeding episodes. Gastroesophageal reflux disease diagnosed by objective endoscopic or radiographic means, and clinically symptomatic at any point over the last 6 months.
  • The patient has one or more clinical laboratory test values outside the reference range, based on the blood and urine samples taken at the Screening Visit, that are of potential risk to the patient's safety, or the patient has, at the Screening Visit:
  • estimated glomerular filtration rate (eGFR) calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation <30 mL/min/1.73 m2
  • a serum total bilirubin value > 1.5 times the upper limit of the reference range unless elevation is related to Gilbert's syndrome and the investigator can rule out any underlying liver dysfunction based on other tests, the patient has a Child-Pugh score ≤6, and after discussing the case with the medical monitor
  • a serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value > 2 times the upper limit of the reference range. Values between 2 and 3 times the upper limit of the reference range may be allowed if concomitant to elevation in creatine kinase as long as the investigator can rule out any underlying liver dysfunction based on other tests, the patient has a Child-Pugh score ≤6, and after discussing the case with the medical monitor
  • The patient has, in the investigator's opinion, severe ataxia, neuropathy, balance problems or other medical condition that would interfere the evaluation of the 30s STS test.
  • Untreated or undertreated sleep apnoea, in the opinion of the investigator.
  • Use of idebenone within 14 days prior to the first dose.
  • Patients have a history of unstable or severe pulmonary, immunological, oncological, hepatic disease, renal disease, or another medically significant illness other than PMD or takes medication that could, in the investigator's opinion, interfere with the assessments of safety, tolerability, or efficacy, or interfere with the conduct or interpretation of the study.
  • The patient is, in the investigator's opinion, unlikely to comply with the protocol e.g. due to cognitive impairment or is unsuitable for any reason.
  • The patient has an immediate family member (defined as family members residing at the same address) who participates in the study.
  • Female patients with a positive pregnancy result at Screening or at Baseline.
  • A patient cannot participate if they received an investigational drug 30 days or 5 half-lives prior to the Screening Visit (whichever is longer), or plans to use an investigational drug (other than the study intervention) during the study
  • Hypersensitivity to the active substance or to any of the excipients or placebo.

Trial locations and contacts

55 sites in 11 countries. Contact the study team or a site near you to ask about taking part.

Communication Manager contact

+31 71 524 7400

medinfo@pharming.com

55 sites in 11 countries

United States20 sites, 20 recruiting

  • Akron Children's Hospital

    Akron, Ohio, 44307

    Josselyn Coppenger, 330-543-6164, jcoppenger@akronchildrens.org

    Bruce Cohen, MD

    Recruiting

  • Rare Disease Research, LLC

    Atlanta, Georgia, 303129

    Scott Batchelor, MD, 678-883-6897, sbatchelor@rarediseaseresearch.com

    Recruiting

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

    Aurora, Colorado, 80045

    Austin Larson, MD, (303) 724-2338, austin.larson@childrenscolorado.org

    Recruiting

  • Johns Hopkins University School of Medicine

    Baltimore, Maryland, 21205

    Hilary Vernon, MD, hvernon1@jhmi.edu

    Hilary Vernon

    Not yet recruiting

  • Neurology and Neuromuscular Care Center-Neurology Rare Disease Center

    Flower Mound, Texas, 75028

    Diana Castro, MD, 214-648-2166, drcastro@neuromdcenter.com

    Diana Castro

    Not yet recruiting

  • Baylor College of Medicine (BCM)

    Houston, Texas, 77030

    Fernando Scaglia, MD, 832-822-4280, fscaglia@bcm.edu

    Recruiting

  • The University of Texas Health Science Center at Houston

    Houston, Texas, 77030

    William Guerra, William.h.Guerra@uth.tmc.edu

    Mary Kay Koenig, MD

    Recruiting

  • IU Health University Hospital

    Indianapolis, Indiana, 46202

    Melissa Lah, MD, 406-597-3274, mspurr@iu.edu

    Melissa Lah

    Not yet recruiting

  • Tekton Marlboro Neurology

    Marlboro, New Jersey, 07746

    Amor Mehta, MD, 732-557-2316, amor.mehta@tektonresearch.com

    Amor Mehta

    Recruiting

  • Novel Clinical Research Center, LLC

    Miami, Florida, 33186

    Juan Remos, MD, 786-803-8887, jremos@associatesinresearch.com

    Juan Remos

    Recruiting

  • Yale University School of Medicine

    New Haven, Connecticut, 06519

    Bhaskar Roy, MD, 857-445-9112, bhaskar.roy@yale.edu

    Bhaskar Roy

    Not yet recruiting

  • Columbia University Irving Medical Center

    New York, New York, 100032

    Hirano Michio, MD, 212-305-1048, mh29@cumc.columbia.edu

    Not yet recruiting

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029-6574

    Ibrahim Elsharkawi, MD, 212-659-8512, ibrahim.elsharkawi@mssm.edu

    Ibrahim Elsharkawi

    Not yet recruiting

  • University of California, Irvine - ALS & Neuromuscular Center

    Orange, California, 92868

    Mozaffar Tahseen, MD Professor, 714-456-2332, mozaffar@uci.edu

    Recruiting

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Zarazuela Zolkipli-Cunningham, MD, zolkipliz@chop.edu

    Zarazuela Zolkipli-Cunningham

    Not yet recruiting

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

    Uta Lichter-Konecki, MD, (412) 692-8865, uta.lichterkonecki@chp.edu

    Jennifer Baker, 412-692-6378, Jennifer.Baker@chp.edu

    Recruiting

  • Mayo Clinic

    Rochester, Minnesota, 55905

    Tan Khoon Ghee Queenie, MD, 507-284-2120, Tan.KhoonGheeQueenie@mayo.edu

    Recruiting

  • The Regents of the University of California - San Diego

    San Diego, California, 292093

    Richard Haas, MD, +1 (858)-246-2288, rhaas@health.ucsd.edu

    Recruiting

  • UCSF Movement Disorders Clinic

    San Francisco, California, 94158

    Swathy Chandrashekhar, MD, swathy.chandrashekhar@ucsf.edu

    Swathy Chandrashekhar

    Not yet recruiting

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

    St Louis, Missouri, 63110

    Conrad Weihl, MD, 314-747-6394, weihlc@neuro.wustl.edu

    Not yet recruiting

Other trials in United States

France9 sites, 9 recruiting

  • Centre Hospitalier Universitaire d'Angers

    Angers, 49933

    Marco Spinazzi, MD, marco.spinazzi@chu-angers.fr

    Marco Spinazzi

    Recruiting

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

    Bordeaux

    Aurelien Trimouille, MD, +33 5 57 82 03 63, aurelien.trimouille@chu-bordeaux.fr

    Aurelien Trimouille, MD

    Recruiting

  • Hopital Roger Salengro, CHRU de Lille

    Lille

    Celine Tard, +33 3 20 44 59 62, celine.tard@chu-lille.fr

    Celine Tard, MD

    Recruiting

  • Centre Hospitalier Universitaire de Nantes

    Nantes, 44093

    Yann Pereon, MD, yann.pereon@univ-nantes.fr

    Yann Pereon

    Not yet recruiting

  • Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2

    Nice

    Sabrina Sacconi, MD, +33(0)492037267, sacconi.s@chu-ncie.fr

    Sabrina Sacconi, MD

    Recruiting

  • CHU de NICE - Hôpital Archet 2

    Nice

    Véronique Paquis-Flucklinger, MD

    Recruiting

  • Hopital Universitaire Necker Enfants Malades

    Paris, 75015

    Benoit Funalot, MD, +330663848836, benoit.funalot@aphp.fr

    Benoit Funalot

    Not yet recruiting

  • Groupe Hospitalier Pitie-Salpetriere

    Paris

    Anthony Behin, +33 142163713, anthony.behin@aphp.fr

    Anthony Behin, MD

    Recruiting

  • Hopitaux Universitaires de Strasbourg

    Strasbourg, 67091

    Aleksandra Nadaj-Pakleza, aleksandra.nadaj-pakleza@chru-strasbourg.fr

    Aleksandra Nadaj-Pakleza

    Recruiting

Other trials in France

Spain6 sites, 5 recruiting

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035

    Raul Juntas Morales, MD, 33467337822, raul.juntas@vallhebron.cat

    Raul Juntas Morales

    Recruiting

  • CIBERER- IDIBAPS, Faculty of Medicine, University of Barcelona

    Barcelona

    Pablo Iglesias García, +34 932275400 ext. 4840-4622, PIGLESIA@recerca.clinic.cat

    Gloria Garrabou, MD

    Recruiting

  • Hospital de la Santa Creu i Sant Pau

    Barcelona

    Completed

  • Hospital General Universitario de Catalunya

    Barcelona

    Susana Andrade Olivié, +34 931751575, s.andrade@udic.es

    Josep Gamez Carbonell, MD

    Recruiting

  • Hospital Universitario 12 de Octubre

    Madrid

    Montserrat Morales, MD, montserrat.morales@salud.madrid.org

    Montserrat Morales, MD

    Recruiting

  • Universitat de Valencia

    Valencia, 46026

    Nuria Muelas Gomez, MD, 961244000, muelas_nur@gva.es

    Nuria Muelas Gomez

    Not yet recruiting

Other trials in Spain

Italy5 sites, 5 recruiting

  • IRCCS Institute of Neurological Sciences of Bologna- Universita di Bologna

    Bologna, 40139

    Maria Lucia Valentino, MD, +39 051 4966114, marialucia.valentino@unibo.it

    Domenica Chiara Milano, +39 051 4966114, domenichiara.milano@ausl.bologna.it

    Recruiting

  • Azienda Ospedaliera Universitaria Gaetano Martino Messina

    Messina, 98125

    Olimpia Musumeci, MD Professor, +39902212672, Messina olimpia.musumeci@unime.it

    Selene Drago, +39902212672, sedrago@unime.it

    Recruiting

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133

    Costanza Lamperti, MD, +39 0223932622, costanza.lamperti@istituto-besta.it

    Valeria Nicoletta, +39 0223932622, valeria.nicoletta@instituto-besta.it

    Recruiting

  • Azienda Ospedaliero Universitaria Pisana

    Pisa, 56126

    Michelangelo Mancuso, MD, +39050992567, michelangelo.mancuso@unipi.it

    Riani Pierangela, +39050992567, p.riani@ao-pisa.toscana.it

    Recruiting

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

    Roma, 00168

    Servidei Serenella, MD, +393385091541, serenella.servidei@policlinicogemelli.it

    Guido Primiano, +393385091541, guidoalessandro.primiano@policlinicogemelli.it

    Recruiting

Other trials in Italy

Australia4 sites, 4 recruiting

  • Perron Institute

    Nedlands, Western Australia, 6009

    William Wallefeld, MD, wwallefeld@gmail.com

    William Wallefeld

    Not yet recruiting

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050

    Timothy Fazio, MD, 0391382341, timothy.fazio@mh.org.au

    Timothy Fazio

    Not yet recruiting

  • Neuroscience Research Australia

    Randwick, New South Wales, 2031

    Carolyn Sue, MD, 294-631-828, c.sue@neura.edu.au

    Carolyn Sue

    Not yet recruiting

  • Royal North Shore Hospital

    Saint Leonards, New South Wales, 2065

    Christina Liang, MD, 294-631-828, Christina.Liang@health.nsw.gov.au

    Christina Liang

    Not yet recruiting

Other trials in Australia

Belgium3 sites, 3 recruiting

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

    Brussels

    Gauthier Remiche, MD, +3225555676, gauthier.remiche@erasme.ulb.ac.be

    Christiane Kitoko, Christiane.kitoko@hubruxelles.be

    Recruiting

  • Universitair Ziekenhuis Gent

    Ghent

    Fien Demeulemeester, +32 9 332 57 00, fien.demeulemeester@uzgent.be

    Dimitri Hemelsoet, MD, dimitri.hemelsoet@uzgent.be

    Recruiting

  • Universitair Ziekenhuis Leuven Gasthuisberg Campus

    Leuven

    Kristl Claeys, MD, kristl.claeys@uzleuven.be

    Kristl Claeys, MD

    Recruiting

Other trials in Belgium

United Kingdom3 sites, 3 recruiting

  • Department of Clinical Neurosciences, Addenbrooke's Hospital

    Cambridge

    Add-tr.mitoteam@nhs.net

    Rita Horvath, MD

    Recruiting

  • University College London Hospitals Nhs Foundation Trust

    London

    Louise Germain, 0203 108 6308, l.germain@nhs.net

    Robert Pitceathly, MD

    Recruiting

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

    Newcastle

    Grainne Gorman, MD, +44 (0)191 2820340, grainne.gorman@ncl.ac.uk

    Grainne Gorman, MD

    Recruiting

Other trials in United Kingdom

Germany2 sites, 2 recruiting

  • Charite - Universitaetsmedizin Berlin

    Berlin, 10117

    Phillip Mergenthaler, MD, +49 30 450 560 020, philipp.mergenthaler@charite.de

    Recruiting

  • Universitaetsklinikum Halle

    Halle, 6120

    Alexander Mensch, MD, 345-557-2932, alexander.mensch@uk-halle.de

    Recruiting

Other trials in Germany

Czechia1 site, 1 recruiting

  • Charles University and General University Hospital

    Prague, 128 08

    Hana Krejcova, MD, Hana.Krejcova@vfn.cz

    Hana Krejcova

    Not yet recruiting

Other trials in Czechia

Denmark1 site, 1 recruiting

  • Copenhagen Neuromuscular Center, Rigshospitalet

    Copenhagen

    Nicolai Rasmus Preisler, MD, +45 3545 55 24, nicolai.rasmus.preisler@regionh.dk

    Nicolai Preisler, MD

    Recruiting

Other trials in Denmark

Netherlands1 site, 1 recruiting

  • Radboud University Medical Center

    Nijmegen, 6525

    Christiaan Saris, MD, +31 243 668 374, c.saris@radboudumc.nl

    Recruiting

Other trials in Netherlands

About this study

Official title: 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

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.

Read the detailed 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 weeks
  • Treatment: 48 weeks
  • Safety follow-up: 5 weeks Total duration: 61 - 65 weeks

Patients who complete the screening phase and are enrolled in the study are randomly assigned to receive either the study medicine (KL1333) or placebo (no active medication). Patients are more likely to receive the study medication than placebo (for every five people who take part, three receive KL1333 and two receive placebo). Neither the participants nor the study team know who is receiving the study medicine or placebo and participants are not able to change which treatment they are assigned.

Being tested
KL1333, Placebo
Keywords
Chronic fatigue, Myopathy, Muscle weakness, Exercise intolerance, Diabetes
Medical subject headings
Muscular Diseases, Muscle Weakness, Diabetes Mellitus

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • KL1333Drug

    Twice daily

  • PlaceboDrug

    Twice daily

Study groups in NCT05650229
Study groupRoleWhat they receive
KL1333ExperimentalTwice daily
Receives: Drug: KL1333
Matching PlaceboPlacebo comparatorTwice daily
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form

    Change in t-score. Higher scores indicate greater fatigue severity.

    Measured: Baseline and 48 Weeks

  2. Change in 30 Second Site-to-Stand Test.

    Change in number of stands

    Measured: Baseline and 48 Weeks

Secondary outcomes (9)

  1. Change in Quality of Life in Neurological Disorders (Neuro-QoL) Lower Extremity Function (Mobility) Short Form

    Change in t-score. Higher scores indicate greater functional ability.

    Measured: Baseline and 48 weeks

  2. Change in Individual Activity Assessments - Interference

    Assessing how much Primary Mitochondrial Disease symptoms get in the way of the ability to perform individually selected activities. A 5 point scale ranging from "No interference" to "Completely interferes"

    Measured: Baseline and 48 Weeks

  3. Individual Activity Assessment - Change

    Assessing the ability to perform individually selected activities compared to baseline. A 5 point scale ranging from "Much worse" to "Much better"

    Measured: 48 weeks

  4. Change in Patient Global Impression of Severity

    Scale ranging from 1 (none) to 4 (severe).

    Measured: Baseline and 48 weeks

  5. Score on Patient Global Impression of Change

    Scale ranging from 2 (much better) to -2 (much worse).

    Measured: 48 weeks

  6. Change in Clinician Global Impression of Severity

    Scale ranging from 1 (none) to 4 (severe).

    Measured: Baseline and 48 weeks

  7. Score on Clinician Global Impression of Change

    Scale ranging from 2 (much better) to -2 (much worse).

    Measured: 48 weeks

  8. Change in Newcastle Mitochondrial Disease Adult Scale (NMDAS) - subscales I-III

    Measured on a 6-point rating scale from 0 to 5. Total score across all 3 subscales ranges from 0 to 140. Higher scores indicate more extensive and severe system involvements.

    Measured: Baseline and 48 weeks

  9. Change from baseline in Glycated haemoglobin (HbA1c) for subjects with diabetes

    mmol/mol

    Measured: Baseline and 48 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Dec 2022
  2. Study start13 Dec 2022
  3. Primary completionOct 2027 (estimated)
  4. Study completionNov 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
180 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05650229
Sponsor's protocol ID
KL1333 2020-104A
Acronym
FALCON
Status last verified
Jun 2026
First submitted
6 Dec 2022
Last updated
26 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing KL1333 in people with Primary Mitochondrial Disease, run by Pharming Technologies B.V. at 55 sites in 11 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 24 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 26 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 55 sites in United States, France, Spain, Italy, Australia and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Communication Manager (+31 71 524 7400, medinfo@pharming.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form (time frame: Baseline and 48 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05650229.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2026.

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