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

Adult patients with primary mitochondrial disease

All safety parameters (including AEs, physical examination, vital signs, ECG, C-SSRS, and safety laboratory [hematology, blood chemistry, and urinalysis])., Occurrence of metabolic decompensation and lactic acidosis or image-verified stroke-like episodes consequent to GI AEs are AESIs and will be monitored throughout the study.

Patient-reported mitochondrial fatigue: • PROMIS® Fatigue PMD short form, Functional outcome: • 30-second Sit-to-Stand Test, Patient-reported lower extremity function: • Neuro-QOL Lower Extremity Function (Mobility) - short form, Other patient-reported outcomes: • Patient Global Impression (multiple) - severity and change, Other patient-reported outcomes: • EQ-5D-5L, Global impression of severity of PMD disease expression: • Clinician Global Impression of PMD - severity and change, Assessments of mitochondrial disease progression: • NMDAS, Subscales I-III, Mitochondrial diabetes, subgroup analysis: • HbA1c (in subjects with diabetes)

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