Sonlicromanol for Mitochondrial Disease
(KH176-301 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new medicine, sonlicromanol, to determine if it can reduce fatigue and improve physical abilities, such as balance and muscle strength, in people with mitochondrial disease. Participants will take either sonlicromanol or a placebo (a look-alike pill with no active ingredient) twice daily for 52 weeks. The trial aims to assess the safety and effectiveness of sonlicromanol over one year. Eligible participants have chronic fatigue and a specific genetic mutation linked to mitochondrial disease, impacting their daily life. As a Phase 3 trial, this research represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, you cannot participate if you have taken any investigational medical product (IMP) within 3 months before the study or plan to use one during the study.
Is there any evidence suggesting that sonlicromanol is likely to be safe for humans?
Research has shown that sonlicromanol is generally safe for people. One study found that participants who took 100 mg of sonlicromanol twice daily did not experience any serious side effects, suggesting the treatment's safety for humans. While additional information is always beneficial, current evidence indicates that people can use this treatment without major safety concerns.
If sonlicromanol receives approval for another condition, it could further support its safety. However, discussing any concerns with a doctor before joining a trial is always advisable.12345Why do researchers think this study treatment might be promising for mitochondrial disease?
Most treatments for mitochondrial disease aim to manage symptoms or support energy production, often using supplements like Coenzyme Q10 or vitamins. But Sonlicromanol is unique because it targets the underlying mitochondrial dysfunction directly. It acts by modulating oxidative stress and improving cellular energy metabolism, which could address the root cause of the disease rather than just alleviating symptoms. Researchers are excited about Sonlicromanol because it has the potential to offer more comprehensive benefits and improve the quality of life for patients with mitochondrial disease.
What evidence suggests that sonlicromanol might be an effective treatment for mitochondrial disease?
Research has shown that sonlicromanol, which participants in this trial may receive, might help individuals with mitochondrial disease by boosting energy production and reducing inflammation. Previous patients experienced positive changes in these areas, which are often challenging in mitochondrial conditions. Some studies have also reported improvements in alertness and mood, which are important for everyday life. However, not all studies found improvements in physical abilities like walking or balance. This indicates that while sonlicromanol could help with some symptoms, its effects on physical function remain under investigation.26789
Are You a Good Fit for This Trial?
The KHENERFIN study is for people with mitochondrial diseases like MELAS syndrome who have chronic fatigue. Participants must have a specific mitochondrial DNA mutation, be able to take oral medication and use digital apps, and commit to not changing their exercise routine during the trial. They can't join if they're unstable medically or don't meet certain physical strength criteria.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either sonlicromanol or placebo twice daily to assess efficacy and safety over a year
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Sonlicromanol
Trial Overview
This trial tests Sonlicromanol against a placebo over 52 weeks to see if it reduces fatigue and improves physical abilities in those with mitochondrial disease. Patients will take either the drug or placebo twice daily while researchers track changes in balance control, muscle strength, and overall quality of life.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Placebo Group
Administration of 90 mg sonlicromanol (equivalent to 100 mg sonlicromanol.HCl) twice daily
Administration of 100 mg matching placebo twice daily
Find a Clinic Near You
Who Is Running the Clinical Trial?
Khondrion BV
Lead Sponsor
Citations
Phase 2b program with sonlicromanol in patients ...
Sonlicromanol is a promising candidate, as it positively alters the key metabolic and inflammatory pathways associated with mitochondrial disease.
Phase 2b program with sonlicromanol in patients with ...
Sonlicromanol is a promising candidate, as it positively alters the key metabolic and inflammatory pathways associated with mitochondrial ...
NCT06451757 | KHENERFIN Study: A Trial to Evaluate the ...
The KHENERFIN study is investigating whether the study medicine, sonlicromanol, is able to improve symptoms of fatigue and the impact of fatigue on daily life, ...
Therapeutic effects of the mitochondrial ROS-redox ...
Furthermore, KH176 treatment was able to significantly improve rotarod and gait performance and reduced the degeneration of retinal ganglion ...
The KHENERGY Study: Safety and Efficacy of KH176 in ...
No significant improvements in gait parameters or other outcome measures were obtained, except for a positive effect on alertness and mood, ...
Safety and Efficacy of KH176 in Mitochondrial m.3243A>G ...
Twice daily oral 100‐mg KH176 was well tolerated. No serious adverse events were reported.
Study Details | NCT04846036 | The KHENERGYC Study
The current study will explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed ...
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medchemexpress.com
medchemexpress.com/sonlicromanol-hydrochloride.html?srsltid=AfmBOorFsKzOqufF-5eKPWpSy4FL8tJ5v7HKcrlUahP0kge2mZmkntDFSonlicromanol hydrochloride (KH176 ...
Ria de Haas, et al. Therapeutic effects of the mitochondrial ROS-redox modulator KH176 in a mammalian model of Leigh Disease. ... SAFETY DATA SHEET (SDS).
The KHENERGY Study: Safety and Efficacy of KH176 in ...
The results of the study provide first data on safety and efficacy of KH176 in patients with mitochondrial disease and will be instrumental in ...
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