New drug aims to ease fatigue in mitochondrial disease

NCT ID NCT05650229

Recruiting now Symptom relief Sponsor: Pharming Technologies B.V. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 2 trial tests whether KL1333 can reduce fatigue and improve leg strength in adults with primary mitochondrial disease, a genetic condition that affects energy production. About 180 participants will receive either KL1333 or a placebo twice daily for 48 weeks. The study measures changes in fatigue symptoms and physical function.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: KL1333
What this could lead to: If it works, this could point toward a treatment that reduces fatigue and improves daily function for people with primary mitochondrial disease.
What could go wrong: This is an early Phase 2 trial with only 180 participants, so results may not apply to everyone. It is also a placebo-controlled study, meaning some may not receive the active drug.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

diabetes mellitus mitochondrial disease muscle tissue disorder Muscle Weakness

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PRIMARY MITOCHONDRIAL DISEASE

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Akron Children's Hospital
RECRUITING

Akron, Ohio, 44307, United States
Azienda Ospedaliera Universitaria Gaetano Martino Messina
RECRUITING

Messina, 98125, Italy
Azienda Ospedaliero Universitaria Pisana
RECRUITING

Pisa, 56126, Italy
Baylor College of Medicine (BCM)
RECRUITING

Houston, Texas, 77030, United States
CHU de NICE - Hôpital Archet 2
RECRUITING

Nice, France
CIBERER- IDIBAPS, Faculty of Medicine, University of Barcelona
RECRUITING

Barcelona, Spain
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
RECRUITING

Bordeaux, France
Centre Hospitalier Universitaire d'Angers
RECRUITING

Angers, 49933, France
Centre Hospitalier Universitaire de Nantes
NOT_YET_RECRUITING

Nantes, 44093, France
Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2
RECRUITING

Nice, France
Charite - Universitaetsmedizin Berlin
RECRUITING

Berlin, 10117, Germany
Charles University and General University Hospital
NOT_YET_RECRUITING

Prague, 128 08, Czechia
Children's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus Location
RECRUITING

Aurora, Colorado, 80045, United States
Columbia University Irving Medical Center
NOT_YET_RECRUITING

New York, New York, 100032, United States
Copenhagen Neuromuscular Center, Rigshospitalet
RECRUITING

Copenhagen, Denmark
Department of Clinical Neurosciences, Addenbrooke's Hospital
RECRUITING

Cambridge, United Kingdom
Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITING

Milan, 20133, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS - Universita Cattolica del Sacro Cuore
RECRUITING

Roma, 00168, Italy
Groupe Hospitalier Pitie-Salpetriere
RECRUITING

Paris, France
Hopital Roger Salengro, CHRU de Lille
RECRUITING

Lille, France
Hopital Universitaire Necker Enfants Malades
NOT_YET_RECRUITING

Paris, 75015, France
Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel
RECRUITING

Brussels, Belgium
Hopitaux Universitaires de Strasbourg
RECRUITING

Strasbourg, 67091, France
Hospital General Universitario de Catalunya
RECRUITING

Barcelona, Spain
Hospital Universitario 12 de Octubre
RECRUITING

Madrid, Spain
Hospital Universitario Vall d'Hebron
RECRUITING

Barcelona, 08035, Spain
Hospital de la Santa Creu i Sant Pau
COMPLETED

Barcelona, Spain
IRCCS Institute of Neurological Sciences of Bologna- Universita di Bologna
RECRUITING

Bologna, 40139, Italy
IU Health University Hospital
NOT_YET_RECRUITING

Indianapolis, Indiana, 46202, United States
Icahn School of Medicine at Mount Sinai
NOT_YET_RECRUITING

New York, New York, 10029-6574, United States
Johns Hopkins University School of Medicine
NOT_YET_RECRUITING

Baltimore, Maryland, 21205, United States
Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
Neurology and Neuromuscular Care Center-Neurology Rare Disease Center
NOT_YET_RECRUITING

Flower Mound, Texas, 75028, United States
Neuroscience Research Australia
NOT_YET_RECRUITING

Randwick, New South Wales, 2031, Australia
Novel Clinical Research Center, LLC
RECRUITING

Miami, Florida, 33186, United States
Perron Institute
NOT_YET_RECRUITING

Nedlands, Western Australia, 6009, Australia
Radboud University Medical Center
RECRUITING

Nijmegen, 6525, Netherlands
Rare Disease Research, LLC
RECRUITING

Atlanta, Georgia, 303129, United States
Royal Melbourne Hospital
NOT_YET_RECRUITING

Parkville, Victoria, 3050, Australia
Royal North Shore Hospital
NOT_YET_RECRUITING

Saint Leonards, New South Wales, 2065, Australia
Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust
RECRUITING

Newcastle, United Kingdom
Tekton Marlboro Neurology
RECRUITING

Marlboro, New Jersey, 07746, United States
The Children's Hospital of Philadelphia
NOT_YET_RECRUITING

Philadelphia, Pennsylvania, 19104, United States
The Regents of the University of California - San Diego
RECRUITING

San Diego, California, 292093, United States
The University of Texas Health Science Center at Houston
RECRUITING

Houston, Texas, 77030, United States
UCSF Movement Disorders Clinic
NOT_YET_RECRUITING

San Francisco, California, 94158, United States
UPMC Children's Hospital of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Universitaetsklinikum Halle
RECRUITING

Halle, 6120, Germany
Universitair Ziekenhuis Gent
RECRUITING

Ghent, Belgium
Universitair Ziekenhuis Leuven Gasthuisberg Campus
RECRUITING

Leuven, Belgium
Universitat de Valencia
NOT_YET_RECRUITING

Valencia, 46026, Spain
University College London Hospitals Nhs Foundation Trust
RECRUITING

London, United Kingdom
University of California, Irvine - ALS & Neuromuscular Center
RECRUITING

Orange, California, 92868, United States
Washington University School of Medicine - Center for Advanced Medicine (CAM) - Neuroscience Center
NOT_YET_RECRUITING

St Louis, Missouri, 63110, United States
Yale University School of Medicine
NOT_YET_RECRUITING

New Haven, Connecticut, 06519, United States

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