Phase III study to assess efficacy/safety of sonlicromanol in subjects with m.3243A>G PMD
Research type
Research Study
Full title
A Phase III, randomised, double-blind, placebo-controlled, parallel-group, pivotal trial to assess the efficacy and safety of sonlicromanol in adult subjects with a genetically confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G variant.
IRAS ID
1012623
Contact name
Jan Smeitink
Contact email
Sponsor organisation
Khondrion B.V.
Clinicaltrials.gov Identifier
Research summary
Primary Mitochondrial Disease (PMD) is a condition caused by changes (mutations) in a person’s mitochondrial DNA (mtDNA). These changes affect the mitochondria, which are the parts of the cell that produce energy. As a result, the mitochondria don’t work properly, and the body’s organs and tissues may not get the energy they need to function well. One of these changes is called m.3243A>G. This change can cause a wide range of symptoms, including tiredness, weak muscles, stomach or bowel problems, hearing loss, stroke-like episodes, diabetes, pain, difficulty thinking clearly (often called "brain fog") and more. Each cell has many copies of mitochondrial DNA, and sometimes only some of them are affected by a change. This study is testing a new medicine called sonlicromanol to see if it is safe and can help reduce tiredness, improve leg muscle strength, and improve balance. The study will also examine how the body absorbs and removes the medicine. If sonlicromanol shows benefits, it might eventually be approved for people with this condition.
About 150 participants in approximately 40 study sites worldwide will take part in this study.
The study is a double-blind, placebo-controlled study, meaning that each participant will be randomly assigned to receive either sonlicromanol or matching placebo.
The study participation will take approximately 14 months (up to 4 weeks of screening, 52 weeks of treatment and 2-week follow-up period). There will be several assessments (such as medical history, physical exams, vital signs, ECG, ECHO, blood/urine samples and questionnaires) to be completed at each visit.REC name
London - City & East Research Ethics Committee
REC reference
25/LO/0711
Date of REC Opinion
31 Oct 2025
REC opinion
Further Information Favourable Opinion