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RECRUITING
NCT07514338
PHASE2

Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease

Sponsor: Pharming Technologies B.V.

View on ClinicalTrials.gov

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

Interventions

DRUG

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