RecruitingPhase 2

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

United States, Australia, Belgium180 participantsStarted 2022-12-13

Plain-language summary

The primary objective of the FALCON study is to evaluate the efficacy of KL1333 on selected disease manifestations of primary mitochondrial disease (PMD) following 48 weeks of treatment. This objective involves evaluating the efficacy of KL1333 versus placebo on fatigue symptoms and impacts on daily living as well as on functional lower extremity strength and endurance. Additionally, the study evaluates the safety and tolerability of KL1333.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. m.3243A\>G associated MELAS-MIDD spectrum disorders,
. single large scale mtDNA deletion associated KSS-CPEO spectrum disorders,
. other multisystemic mtDNA-related disease (including MERRF).
. Not a woman of childbearing potential (WOCBP)
. WOCBP must agree not to try and become pregnant and use a highly effective method of contraception from the time of informed consent through at least 36 days (\~5 half-lives of KL1333 plus 30 days) after the last dose of investigational medicinal product (IMP) administration.

Exclusion criteria

. stroke-like episodes within the last 6 months

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Since this is a Phase 2 trial for KL1333, what do we actually know so far about its safety profile, and how does the earlier-stage nature of this study affect the balance of potential risks versus potential benefits for someone in my situation?
2This trial measures fatigue using a self-reported questionnaire and a 30-second sit-to-stand test — are those the kinds of symptoms and physical limitations I'm currently experiencing, and do you think those outcomes reflect what matters most to me personally?
3Since this trial is still recruiting, how long would I likely be enrolled, and how frequently would I need to come in for visits — and is that realistic given my current energy levels and daily functioning with mitochondrial disease?
4Are there any standard-of-care treatments for primary mitochondrial disease that I should consider trying first before exploring an experimental option like this Phase 2 study?
5Given that primary mitochondrial disease covers a range of underlying causes and severities, do I have the specific type or profile of mitochondrial disease that this trial is designed to study, and would my medical history make me a reasonable candidate to even discuss with the trial team?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in-patient-reported fatigue symptoms and impacts on daily living measured by Patient-Reported Outcomes Measurement Information System (PROMIS®) Fatigue PMD Short Form

Timeframe: Baseline and 48 Weeks

Change in 30 Second Site-to-Stand Test.

Timeframe: Baseline and 48 Weeks

Trial details

NCT IDNCT05650229

SponsorPharming Technologies B.V.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-10

Contact for this trial

Communication Manager

+31 71 524 7400 medinfo@pharming.com

View on ClinicalTrials.gov More Primary Mitochondrial Disease trials