KL1333 for Mitochondrial Disease
(FALCON Trial)

Recruiting in Palo Alto (17 mi)

+17 other locations

Overseen byAmel Karaa, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Abliva AB

Prior Safety Data

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.

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.

+9 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.

+13 more

Participant Groups

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.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: KL1333Experimental Treatment1 Intervention

Twice daily

Group II: Matching PlaceboPlacebo Group1 Intervention

Twice daily