KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
The KHENERFIN study is testing a new medicine called sonlicromanol for people living with mitochondrial diseases. These conditions can cause extreme tiredness and affect physical abilities. The study wants to find out if sonlicromanol can help reduce feelings of tiredness (fatigue), make it easier to do daily activities, and improve strength and balance. Participants will take either sonlicromanol or a placebo (a tablet that looks the same but has no active medicine) twice a day for 52 weeks. Neither the patient nor their doctors will know who is getting which tablet. Researchers will also be checking how safe the medicine is. This is a Phase 3 study, meaning it's one of the final steps before a medicine might become available.
At a glance
What is this study about?
The KHENERFIN study is looking into a new medicine called sonlicromanol. This medicine is designed for people who have certain types of mitochondrial diseases. Mitochondria are like tiny powerhouses inside our cells that give us energy. When they don't work properly, it can lead to various health problems, including extreme tiredness (fatigue) and difficulties with physical activities.
This study aims to discover if sonlicromanol can make a real difference in the lives of people with mitochondrial disease. Specifically, it wants to see if the medicine can help reduce tiredness and its impact on daily life. It also hopes to improve muscle strength and balance, which are important for everyday movements and staying active.
To find this out, researchers will compare the effects of sonlicromanol with a 'placebo'. A placebo is a dummy pill that looks exactly like the real medicine but contains no active ingredients. This helps researchers understand if any improvements are due to the new medicine itself or other factors. The study will also keep a close eye on any side effects to make sure sonlicromanol is safe.
Key takeaways
- The study tests a new medicine, sonlicromanol, for mitochondrial diseases.
- It aims to reduce fatigue and improve physical strength and balance.
- Participants will take sonlicromanol or a placebo twice daily for one year.
- Neither patients nor doctors will know which treatment is being given.
- The study involves regular clinic visits and lasts about 60 weeks.
- Participants must have a specific genetic mutation and ongoing fatigue.
Who may be eligible?
This study is looking for adults aged 18 or older who have a specific type of mitochondrial disease. You would need to have a confirmed genetic change (mutation called m.3243A>G) in your mitochondrial DNA. This mutation would also need to be present at a certain level in your blood, urine, cheek swab, or muscle.
You also need to be experiencing ongoing tiredness (chronic fatigue) that has lasted for at least three months and is not caused by other health problems. This tiredness would be checked using a specific questionnaire. Additionally, you would need to show some weakness in your muscles, measured by how quickly you can do a 'sit to stand' test.
There are some reasons why you might not be able to join. For example, if you've recently taken part in another drug trial, have certain bone problems, severe digestive issues, or serious heart or breathing conditions. Your study doctor will review all your medical information to see if this study is right for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have a confirmed mitochondrial disease with the m.3243A>G genetic change?
- Have you been experiencing chronic tiredness (fatigue) for at least 3 months?
- Is your fatigue not caused by another medical condition?
- Can you do a 'sit to stand' test within a reasonable time?
- Are you able to commit to taking tablets daily for a year and attending regular clinic visits?
What does participation involve?
If you are eligible, you would start with a screening period lasting up to 4 weeks. During this time, the study team will do checks to make sure you can safely take part. If you join, you will be randomly assigned (like tossing a coin) to receive either the active medicine, sonlicromanol, or the placebo. You have an equal chance of receiving either.
You would take your assigned study tablets twice a day for 52 weeks (about one year). Neither you nor your study doctor will know whether you are taking sonlicromanol or the placebo. Throughout this year, you'll have regular visits to the clinic so the team can monitor your health, how you're feeling, and how the medicine is affecting you.
About two weeks after you take your last dose of the study medicine, you'll have a final follow-up visit. The total time you would be involved in the study, from screening to the final follow-up, would be about 60 weeks (just over a year).
Potential risks and benefits
Locations (10)
- Massachusetts General HospitalVerified postcodeBoston, United States· Not yet recruiting
- Cleveland Clinic Neurological Institute Mellen CenterVerified postcodeCleveland, United States· Not yet recruiting
- The University of Texas Health Science Center at HoustonVerified postcodeHouston, United States· Not yet recruiting
- Rigshospitalet, University of CopenhagenUnverifiedKopenhagen, Denmark· Not yet recruiting
- CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie ObstetriqueVerified postcodeBordeaux, France· Not yet recruiting
- Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-EstVerified postcodeParis, France· Not yet recruiting
- Klinikum der Universität München Friedrich-Baur-InstitutVerified postcodeMünchen, Germany· Not yet recruiting
- Fondazione IRCCS Istituto Neurologico Carlo BestaVerified postcodeMilan, Italy· Not yet recruiting
- Radboud University Medical CenterVerified postcodeNijmegen, Netherlands· Recruiting
- University College London Hospitals NHS Foundation Trust National Hospital for Neurology and NeurosurgeryVerified postcodeLondon, United Kingdom· Recruiting
Common questions
What is mitochondrial disease?
Mitochondrial diseases are conditions where the 'powerhouses' (mitochondria) inside your cells don't work properly, leading to a lack of energy and various health problems.
What is sonlicromanol?
Sonlicromanol is the new medicine being tested in this study to see if it can help people with mitochondrial diseases feel less tired and improve their physical abilities.
What is a 'placebo'?
A placebo is a tablet that looks exactly like the study medicine but doesn't have any active ingredients. It's used to fairly compare the effects of the real medicine.
Will I know if I'm getting the real medicine or the placebo?
No, neither you nor your study doctor will know whether you are taking sonlicromanol or the placebo. This helps make the study results as accurate as possible.
How long will I be in the study?
From the initial checks to the final follow-up visit, you'd be involved in the study for about 60 weeks (just over a year).
How to find out more
Jasper Levink, MSc.
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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