KL1333 for Mitochondrial Disease
(FALCON Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called KL1333 for individuals with primary mitochondrial disease, which often leads to chronic fatigue and muscle weakness. The researchers aim to determine if KL1333 can reduce fatigue and improve daily activities while assessing its safety. Participants will receive either KL1333 or a placebo (a pill with no active medicine) for comparison. This trial suits those with confirmed mitochondrial disease who experience ongoing fatigue and muscle weakness and can manage daily tasks like climbing stairs. As a Phase 2 trial, it focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants.
Will I have to stop taking my current medications?
The trial requires participants to stop taking idebenone during the study. If you are on mitochondrial disease-focused vitamins or supplements like CoQ10, niacin, or L-arginine, you must have been on a stable dose for 3 months before the trial and continue that dose throughout the study.
Is there any evidence suggesting that KL1333 is likely to be safe for humans?
Research has shown that KL1333 is generally safe for people. In earlier studies, KL1333 was safe and well-tolerated. Some participants experienced stomach-related side effects, which were linked to the dosage. Evidence also suggests that KL1333 might improve muscle strength in individuals with primary mitochondrial disease (PMD). While this information is encouraging, the treatment is still under testing to confirm these results.12345
Why do researchers think this study treatment might be promising?
Unlike other treatments for mitochondrial disease, KL1333 is unique because it targets the underlying energy production issues within cells. Most current therapies focus on managing symptoms rather than addressing the root cause. KL1333 is designed to enhance the function of mitochondria, the cell's powerhouses, potentially leading to improved energy levels and overall function. This approach is exciting for researchers because it offers the possibility of a more effective and direct treatment, potentially transforming how mitochondrial diseases are managed.
What evidence suggests that KL1333 might be an effective treatment for mitochondrial disease?
Studies have shown that KL1333, which participants in this trial may receive, can help reduce tiredness and improve strength and stamina in people with primary mitochondrial disease. Previous patients reported feeling less fatigued and experiencing better muscle function after taking KL1333. This investigational treatment addresses the energy production problems caused by mitochondrial disease, which might explain its potential benefits. Early research is promising and suggests that KL1333 could significantly improve daily life for those affected by this condition.14678
Who Is on the Research Team?
Grainne Gorman, MD, PhD
Principal Investigator
Wellcome Centre for Mitochondrial Research, Newcastle University
Amel Karaa, MD
Principal Investigator
Massachusetts General Hospital
Are You a Good Fit for This Trial?
Adults over 18 with primary mitochondrial disease (PMD) characterized by muscle weakness or limited exercise tolerance, chronic fatigue, and a confirmed genetic diagnosis. Participants must be clinically stable, able to attend appointments, willing to maintain diet and therapy regimens, not pregnant, and agree to contraception requirements.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either KL1333 or placebo twice daily for 48 weeks
Safety follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- KL1333
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Abliva AB
Lead Sponsor