Pharming Technologies B.V.
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.
This is a 12-month open-label extension (OLE) study to evaluate the safety, tolerability, and efficacy of KL1333 in subjects previously treated with KL1333 or placebo in Study KL1333-2020-104A (hereafter referred to as FALCON). Subjects can be enrolled in this extension study either directly at the FALCON study completion visit (FALCON Week 48) or the safety follow-up visit (FALCON Week 53) or later in time. Subjects who previously received KL1333 in FALCON will receive retreatment whereas subjects previously treated with placebo will be treatment naïve to KL1333. The study consists of a scr…
Inclusion Criteria: * 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 Exclusion Criteria: * The subject is, in the investigator's opinion, unlikely to…
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