Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease
Starting soon · Phase 2
Conditions studied: Mitochondrial Diseases
In brief
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.
Key facts
- Study ID
- NCT07514338
- Run by
- Pharming Technologies B.V.
- People needed
- 140
- Starts
- 2026-06-09
- Expected to finish
- 2029-03-31
- Last updated by the study team
- 2026-04-07
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Completed the FALCON study (age 18 years or older), and in the opinion of the investigator and sponsor has been compliant with the study requirements
- Willingness and ability to attend study appointments within the specified time windows
- Willingness and ability to complete electronic patient-reported outcomes
- Concomitant medications likely to remain stable throughout participation in the study where clinically possible
- Willingness to suspend treatment with idebenone during the study
You may not qualify if…
- The subject is, in the investigator's opinion, unlikely to comply with the protocol, e.g., due to cognitive impairment, or is unsuitable for any reason.
- Any medical, psychiatric, laboratory or other condition that may negatively affect the benefit-risk considerations of study participation or interfere with the interpretation of study results and, in the judgment of the investigator and/or the medical monitor, would make the subject inappropriate for entry into this study.
- Subjects not enrolling directly at the FALCON study completion visit (FALCON Week 48) or the safety follow-up visit (FALCON Week 53) study will be required to fulfill the additional exclusion criteria below during the screening visit:
- General fatigue or muscle weakness due to causes other than mitochondrial disease, in the opinion of the investigator.
Full record on ClinicalTrials.gov
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