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

Condition(s) studied

Investigational drug(s) / intervention(s)

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

Study 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.

Eligibility

Primary outcome measure(s)

• Adverse events — Through study at least for 48 weeks
  Number of adverse events will be monitored throughout the study for all subjects
• Physical examination — At Baseline Week 0, Week 4, Week 24 and Week 48
  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 — At Baseline Week 0, Week 4, Week 24 and Week 48
  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 — At Baseline Week 0, Week 4, Week 24 and Week 48
  Changes from baseline of ECG parameters will be evaluated.
• Safety laboratory - blood chemistry — At Baseline Week 0, Week 4, Week 24 and Week 48
  Monitoring of the clinically significant laboratory results for sodium, potassium, chloride, bicarbonate/carbon dioxide;, blood urea nitrogen, serum creatinine, glucose, albumin, total protein, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, total bilirubin, direct bilirubin, indirect bilirubin, calcium, gamma-glutamyl transferase, creatine kinase
• Safety laboratory - urinalysis — At Baseline Week 0, Week 4, Week 24 and Week 48
  Monitoring of the clinically significant laboratory results for specific gravity, pH, semi-quantitative "dipstick" evaluation of glucose, protein, bilirubin, ketones, leukocytes, blood microscopy and/or culture to be performed if clinically indicated or if urinalysis results positive.
• Safety laboratory - hematology — At Baseline Week 0, Week 4, Week 24 and Week 48
  Monitoring of the clinically significant laboratory results for Hemoglobin, hematocrit, white blood cell with differentials (monocytes, eosinophils, basophils, neutrophils, lymphocytes) as an absolute value, red blood cell count, platelet count, C-reactive protein
• Occurence of metabolic decompensation and lactic acidosis or image-verified stroke-like episodes consequent to GI AE and AESIs — Through study at least for 48 weeks
  These events will be monitored throughout the study.
• Columbia Suicide Severity Rating Scale (C-SSRS) — At Baseline Week 0
  C-SSRS assesses suicidal ideation and behavior risk through a series of questions to assess for suicidal ideation and behavior, the severity and immediacy of the risk, and the level of support the subject may need. C-SSRS Severity of Ideation scores of 4 or 5 are considered SAEs.

Trial sites (23)

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