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Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease
Sponsor: Pharming Technologies B.V.
Summary
The purpose of this study is to investigate if the study medicine, KL1333, is safe, well-tolerated and effective long-term in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical abilities) in people with PMD.
Official title: An Open-label, Single-arm Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of KL1333 (Napazimone) in Patients With Primary Mitochondrial Disease
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
140
Start Date
2026-06-12
Completion Date
2029-03-31
Last Updated
2026-09-16
Healthy Volunteers
No
Conditions
Interventions
Napazimone
Product: KL1333 (international nonproprietary name: napazimone) Dose: Each subject will be up-titrated to his/her maximum well tolerated dose. The starting dose will be 25 mg KL1333 twice daily (BID; total daily dose of 50 mg). If KL1333 is considered to be well tolerated after 4 weeks of treatment, the dose will be increased to 50 mg KL1333 BID (total daily dose of 100 mg). The dose may be lowered from 50 mg BID to 25 mg BID at the investigator's discretion throughout the study in case of tolerability issues. Frequency: Twice daily Route: Oral
Locations (23)
Akron Children's Hospital
Akron, Ohio, United States
Hospital Erasme
Brussels, Belgium
Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel
Brussels, Belgium
University Hospital
Ghent, Belgium
Universitair Ziekenhuis Gent
Ghent, Belgium
University Hospital
Leuven, Belgium
Universitair Ziekenhuis Leuven Gasthuisberg Campus
Leuven, Belgium
Charles University and General University Hospital
Prague, Czechia
Copenhagen Neuromuscular Center, Rigshospitalet
Copenhagen, Denmark
Centre Hospitalier Universitaire d'Angers
Angers, France
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
Bordeaux, France
Hopital Roger Salengro, CHRU de Lille
Lille, France
Centre Hospitalier Universitaire de Nantes
Nantes, France
Centre Hospitalier Universitaire de Nice, Hopital Pasteur 2
Nice, France
CHU de NICE - Hôpital Archet 2
Nice, France
Hopitaux Universitaires de Strasbourg
Strasbourg, France
Universitaetsklinikum Halle
Halle, Germany
Azienda Ospedaliera Universitaria Gaetano Martino Messina
Messina, Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
Azienda Ospedaliero Universitaria Pisana
Pisa, Italy
Radboud University Medical Center
Nijmegen, Netherlands
Hospital General Universitario de Catalunya
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain