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Compensation: Varies

Number of Visits: Unknown

Duration: 48 weeks

180 Participants Needed

KL1333 for Mitochondrial Disease
(FALCON Trial)

Recruiting at 17 trial locations

Overseen ByCommunication Manager

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Abliva AB

Must not be taking: Idebenone

Disqualifiers: Neurodegenerative phenotypes, Cardiovascular disease, Gastrointestinal issues, others

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

This trial is testing KL1333, a new medicine, to see if it can help people with mitochondrial disease feel less tired and improve their physical abilities. The study targets people with primary mitochondrial disease because they often suffer from severe fatigue. KL1333 works by boosting energy production in cells.

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.

What data supports the effectiveness of the drug KL1333 for mitochondrial disease?

There is no direct evidence supporting the effectiveness of KL1333 for mitochondrial disease, but past trials with similar treatments like idebenone showed some improvement in specific patient subgroups, suggesting potential benefits in certain cases.¹ ² ³ ⁴ ⁵

Is KL1333 safe for humans?

The research does not provide specific safety data for KL1333, but a similar compound, KH176, was well tolerated and appeared safe in a study with mitochondrial disease patients, with no serious adverse events reported.¹ ² ³ ⁶ ⁷

How is the drug KL1333 different from other treatments for mitochondrial disease?

KL1333 is a novel treatment for mitochondrial disease, which is a condition with limited treatment options. Unlike other treatments that primarily offer supportive care, KL1333 is being studied for its potential to directly address the underlying issues of mitochondrial dysfunction.¹ ³ ⁴ ⁶ ⁷

Research Team

Amel Karaa, MD

Principal Investigator

Massachusetts General Hospital

Grainne Gorman, MD, PhD

Principal Investigator

Wellcome Centre for Mitochondrial Research, Newcastle University

Eligibility Criteria

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

Willingness and ability to complete electronic PROs

I have muscle weakness or struggle with physical activity due to mitochondrial myopathy.

I have been feeling extremely tired for more than 3 months.

See 8 more

Exclusion Criteria

I have a mitochondrial disease mainly affecting my nervous system.

I have a genetic mutation linked to mitochondrial disease.

I haven't taken any experimental drugs within the last 30 days.

See 13 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

8-12 weeks

Treatment

Participants receive either KL1333 or placebo twice daily for 48 weeks

48 weeks

Safety follow-up

Participants are monitored for safety and effectiveness after treatment

5 weeks

Treatment Details

Interventions

KL1333
Placebo

Trial Overview The FALCON study is testing the effectiveness of KL1333 on fatigue symptoms and daily life impact in PMD patients after 48 weeks compared to a placebo. It also measures lower extremity strength/endurance while assessing safety and tolerability of KL1333.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: KL1333Experimental Treatment1 Intervention

Twice daily

Group II: Matching PlaceboPlacebo Group1 Intervention

Twice daily

Find a Clinic Near You

Who Is Running the Clinical Trial?

Abliva AB

Lead Sponsor

Trials

Recruited

270+

Findings from Research

Primary mitochondrial diseases are common inherited metabolic disorders affecting about 1 in 4,300 live births, but there are currently no licensed disease-modifying therapies, and most management is supportive.

Recent advances in therapy development have led to small molecules moving from preclinical studies to early-phase human trials, indicating progress in finding effective treatments for these complex disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Moving towards clinical trials for mitochondrial diseases.Pitceathly, RDS., Keshavan, N., Rahman, J., et al.[2021]

Mitochondrial disease research has made significant progress, but effective treatments are still lacking due to reliance on anecdotal evidence rather than rigorous clinical trials.

The author emphasizes the importance of learning from early randomized controlled trials in mitochondrial diseases to improve future research and utilize new resources for patient-oriented studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Why are there no proven therapies for genetic mitochondrial diseases?Stacpoole, PW.[2021]