Mitochondrial Diseases study: Sonlicromanol
Official title KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
ClinicalTrials.gov ID: NCT06451757
What this study is testing
What is Sonlicromanol?
An investigational medicine being studied to see if it can help with fatigue and physical abilities in people with mitochondrial diseases.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether sonlicromanol can reduce fatigue and improve balance and leg strength in people with mitochondrial disease.
- Who it's for
- You may be able to join if you have a mitochondrial disease that causes fatigue, trouble balancing, or weak legs.
- What's involved
- You would take a tablet twice a day for a year. You will have regular check‑ins to track your fatigue, balance, and leg strength.
- Time commitment
- The study lasts one year, with visits every few months to monitor your progress and safety.
- Phase 3: a large, late-stage study
- Time commitment: about 52 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Signed Informed Consent
- Males and females aged ≥18 years with a multi-system primary mitochondrial disease.
- A confirmed mitochondrial DNA tRNALeu(UUR) m.3243A\>G mutation (m.3243A\>G PMD) plus an age adjusted heteroplasmy percentage ≥ 20% in white blood...
- Patient self-reported chronic fatigue for at least 3 months prior to the Screening Visit and recorded in the clinical patient files; AND
- Presence of fatigue (raw total score \>22), assessed by Neuro-QoL SFv1-F at Screening.
You likely can't join if
- Treatment with any IMP within 3 months (or 5 times the half-life of the IMP, whichever is longer) prior to screening or plans to use an IMP (other...
- Bone deformities, motor abnormalities or chronic ulcers that in the opinion of the PI may interfere with and/or confound the interpretation of the...
- Surgery of gastrointestinal tract that might interfere with drug absorption.
- Clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings) in the opinion of the investigator.
- Prior treatment cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within...
- Structural heart disease based on cardiac MRI or Echocardiography (e.g., clinically significant valve disease
See the full eligibility criteria
- Signed Informed Consent
- Males and females aged ≥18 years with a multi-system primary mitochondrial disease.
- A confirmed mitochondrial DNA tRNALeu(UUR) m.3243A\>G mutation (m.3243A\>G PMD) plus an age adjusted heteroplasmy percentage ≥ 20% in white blood cells [=blood heteroplasmy/0.977(age+12)].
- Patient self-reported chronic fatigue for at least 3 months prior to the Screening Visit and recorded in the clinical patient files; AND
- Presence of fatigue (raw total score \>22), assessed by Neuro-QoL SFv1-F at Screening.
- Presence of mitochondrial myopathy defined as: 5xSST at Screening and Baseline should be ≥ 11 seconds and participant must demonstrate the ability to complete the test at baseline (i.e., complete the test within 30...
- Treatment with any IMP within 3 months (or 5 times the half-life of the IMP, whichever is longer) prior to screening or plans to use an IMP (other than the study intervention) during the study.
- Bone deformities, motor abnormalities or chronic ulcers that in the opinion of the PI may interfere with and/or confound the interpretation of the subject's performance during the 5 times sit to stand test (5XSST).
- Surgery of gastrointestinal tract that might interfere with drug absorption. Or severe GI dysmotility, chronic vomiting, diarrhea, bouts of pseudo-obstruction which will impair appropriate IMP absorption in the opinion...
- Clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings) in the opinion of the investigator.
- Prior treatment cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening.
- Structural heart disease based on cardiac MRI or Echocardiography (e.g., clinically significant valve disease
- 1st degree atrioventricular (AV)-block
- 2nd degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
- Right bundle branch block.
- History of acute heart failure (within the last 3 months).
- Higher degree of AV-blocks (AVB II° or III°).
- Other exclusion criteria per protocol
The study team makes the final eligibility decision.
Where it is enrolling
- Houston, Texas, United States
- Kopenhagen, Region Sjælland, Denmark
- Paris, Paris, France
- München, Germany
- Milan, Italy
- Nijmegen, Gelderland, Netherlands
- London, Greater London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The study runs about 52 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Houston, Texas, United States; Kopenhagen, Region Sjælland, Denmark; Paris, Paris, France; München, Germany; Milan, Italy; Nijmegen, Gelderland, Netherlands and 1 more location. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.