Efficacy of KL1333 in adults with primary mitochondrial disease
Research type
Research Study
Full title
An interventional, randomised, double-blind, parallel-group, placebo-controlled, flexible-dose, adaptive study of the efficacy of KL1333 in adult patients with primary mitochondrial disease
IRAS ID
1004582
Contact name
Yi Shiau NG
Contact email
Sponsor organisation
Abliva AB
Eudract number
2021-002846-33
Research summary
The research study is aimed at patients with primary mitochondrial disease (PMD) with genetically confirmed multi-systemic (affects more than one body system) mitochondrial DNA-related disease.
The purpose of this study is to investigate if the study drug, KL1333, is effective in improving the symptoms of fatigue and impacts on daily living and functional capacity (physical abilities) in people with PMD. Additionally, the safety and how well KL1333 is tolerated in the body at different doses, will be evaluated. KL1333 will be compared with placebo in this research study. The study will also measure the amount of KL1333 in your blood at various times (pharmacokinetics or PK).
The total study duration per patient from baseline to the end of follow-up will be approximately 64 weeks (8-12 weeks Screening, 48 weeks treatment and 4 weeks Safety Follow-Up). Study procedures for all the participants will be:
• Blood and urine tests
• Current health and treatment review
• Vital signs and physical exam
• Dietary information and review
• Stool sample
• Electrocardiogram (ECG)
• Complete questionnaires about health, fatigue, daily activities, pain and sleep
• 30 second sit to stand test
Approximately 120 to 180 participants will take part in the study in 7 to 12 countries.REC name
East Midlands - Derby Research Ethics Committee
REC reference
22/EM/0021
Date of REC Opinion
19 May 2022
REC opinion
Further Information Favourable Opinion