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.
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
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.
Single group
Napazimone
DrugProduct: 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
Adverse events
Number of adverse events will be monitored throughout the study for all subjects
Physical examination
The following parameters and body systems will be examined and any abnormalities described: height and weight; general appearance; skin; head, ears, eyes, nose, and throat; lungs; heart; lower extremity examination; abdomen; neurologic and lymph nodes. Any clinically significant changes from baseline should be recorded as AEs.
Vital signs
Body temperature, systolic and diastolic cuff blood pressure, pulse rate and pulse oximetry will be measured and any clinically significant changes from baseline should be recorded as AEs.
Electrocardiogram
Changes from baseline of ECG parameters will be evaluated.
Patient-reported mitochondrial fatigue
Patient-Reported Outcomes Measurement Information System® Fatigue PMD short form. The PROMIS® Fatigue PMD short form consists of 9 items. Each item has numerical rating scale (NRS) response options consisting of never (1), rarely (2), sometimes (3), often (4), and always (5).
Functional outcome
30 seconds sit-to-stand test
Patient-reported lower extremity function
Neuro-QOL Lower Extremity Function (Mobility) - short form. It is a reliable and validated brief 8-item survey of one's ability to carry out various activities involving the lower limb/trunk region and increasing degrees of bodily movement. Each item has numeric rating scale (NRS) response options consisting of Without any difficulty (5), With a little difficulty (4), With some difficulty (3), With much difficulty (2), and unable to do (1).
Other patient-reported outcome - Patient Global Impression (multiple)
The Patient Global Impression of Severity is rated on a 4-point NRS, assessed on a scale ranging from 1 (none) to 4 (severe) for severity. The Patient Global Impression of Change is rated on a 5-point numeric rating scale (NRS), with the change from baseline assessed on a scale ranging from 2 (much better) to -2 (much worse), with 0 indicating no change from baseline.
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Pharming Technologies B.V.
Lead sponsor
ICON plc
Collaborator
