At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Sonlicromanol (drug), Placebo (drug)
- How long the study runs
- Study runs about 29 months (dates as stated)
- About the drug or intervention
- Sonlicromanol — drug: Administration of 90 mg sonlicromanol (100 mg sonlicromanol.HCl) twice daily during 52 weeks · Placebo — drug: Administration of 100 mg placebo twice daily during 52 weeks
- Patient visit burden
- Not specified by the sponsor
In plain English
This study, run by Khondrion BV, is testing a medicine called sonlicromadol—spelled sonlicromanol in the registry—for its effectiveness and safety in adults with primary mitochondrial diseases. It focuses on people with a specific genetic change called the m.3243A>G mutation, which can cause conditions such as maternally inherited diabetes and deafness (MIDD) and MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes).
Who can take part
- Adults aged 18 or over with a multi-system primary mitochondrial disease
- A confirmed m.3243A>G mutation with a heteroplasmy level of 20% or more, measured in blood, urine, cheek swab or muscle before joining
- Chronic fatigue lasting at least 3 months that is not explained by another cause, recorded in medical files and shown on a screening questionnaire (Neuro-QoL fatigue score above 22)
- Muscle weakness (mitochondrial myopathy), shown by being able to complete a 'sit to stand' test 5 times within 30 seconds, taking at least 11 seconds
- Signed informed consent
- Other inclusion rules apply as set out in the study protocol
Who may not be able to
- Taking another investigational medicine within the last 3 months, or planning to during the study
- Bone problems, movement problems or long-term ulcers that could affect the sit-to-stand test
- Stomach or bowel surgery, or severe digestive problems, that could stop the medicine being absorbed properly
- Significant lung disease or heart disease
- Certain heart procedures within the last 3 months
- A heart rhythm measure (QTcF) above 450 milliseconds in men or 470 milliseconds in women
- Certain structural heart problems, heart failure (class II or above), or poorly controlled heart conditions
- Family history of unexplained fainting, certain inherited heart rhythm syndromes, or sudden death before age 60
- Certain heart rhythm problems on an ECG (heart tracing), such as atrial fibrillation or higher-grade heart block
- Acute heart failure within the last 3 months
- Other exclusion rules apply as set out in the study protocol
What taking part involves
- • Taking sonlicromanol, the study medicine being tested for effectiveness and safety
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team about how long the study lasts, how many visits are needed and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years and over
- Who
- All
- Number of participants
- 220
- Started
- 2026-04-14
- Last checked
- 2026-06
Plain English Summary
What is this study?
- • Testing a new treatment for mitochondrial diseases
- • Phase3 - 220 participants
- • The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of people like balance control and lower limb skeletal muscle strength in people with mitochondrial disease
Who can take part?
- • Ages 18 Years and over
- • Diagnosed with mitochondrial diseases
Where?
- • London - University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of people like balance control and lower limb skeletal muscle strength in people with mitochondrial disease. In this study, the effects of sonlicromanol are compared against a placebo, a tablet identical in appearance and taste but without the active drug. Participants take either sonlicromanol or placebo twice daily for a treatment duration of 52 weeks. In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on secondary and exploratory outcomes, as well as its safety and tolerability after one year of treatment.
More detail
The KHENERFIN study is investigating the medicine sonlicromanol. The study aims to see if sonlicromanol can reduce symptoms of fatigue and reduce the impact of fatigue on daily life. The study also investigates if sonlicromanol improves physical abilities like balance control and lower limb skeletal muscle strength in people with mitochondrial disease. In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcomes. It also assesses the safety and tolerability of sonlicromanol. This study is a placebo controlled, double blind study; the effects of sonlicromanol will be compared with a placebo (study medication that looks like the actual study medicine but contains no active medicine). Neither the participants nor the study team know who is receiving the study medicine or placebo. Participants cannot change their assigned rreatment. During the screening period, which lasts a maximum of 4 weeks, it is assessed whether the potential participant meets all requirements to participate in the study. Patients who complete the screening phase and are enrolled in the study are randomly (by chance) assigned to receive either the study medicine sonlicromanol or placebo (no active medication). Participants have an equal chance of receiving either sonlicromanol or a placebo. A final follow-up visit is scheduled 2 weeks after taking the last dose of study medication. Total study duration is approximately 60 weeks. Sonlicromanol will be supplied in tablet form, containing 90 mg of sonlicromanol (equivalent to 100 mg of sonlicromanol.HCl), with the tablets embossed accordingly or provided as a placebo. The study medication must be taken twice daily during the treatment period of 52 weeks. Up to 220 subjects with a confirmed mitochondrial DNA tRNALeu(UUR) 3243A\>G mutation will be randomly assigned in a 1:1 ratio to receive either sonlicromanol or placebo.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years and over
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Inclusion criteria
- ✓Signed agreement to take part
- ✓Males and females aged ≥18 years with a multi-system primary mitochondrial disease.
- ✓Presence of chronic fatigue (not attributable to other etiologies than PMD):
- ✓Patient self-reported chronic fatigue for at least 3 months prior to the Screening Visit and recorded in the...
See the full criteria
Where Is This Study? (1 UK site)
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
London WC1N 3BG, United Kingdom
How to Get in Touch
Jasper Levink, MSc.
Sponsor contactCONTACT
G. Ruiterkamp, MSc.
Sponsor contactCONTACT
