RecruitingPHASE2INTERVENTIONAL

Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease

The FALCON study is a research trial exploring a new medicine called KL1333 for adults living with primary mitochondrial disease. The main goal is to find out if KL1333 can effectively lessen feelings of tiredness (fatigue) and boost physical strength and stamina over 48 weeks. Researchers will compare KL1333 with a 'placebo' – a tablet that looks the same but contains no active medicine. The study will also carefully check for any side effects and how well people tolerate the medicine. Participants will take a tablet twice a day. This research is important to understand if KL1333 could be a helpful treatment option for people with this condition.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Abliva AB

Enrolment target

180

Start

13 Dec 2022

Estimated completion

01 Nov 2027

Primary Mitochondrial Disease

What is this study about?

This research study, called FALCON, is looking into a new medication known as KL1333. It's designed for adults who have primary mitochondrial disease (PMD). PMD is a condition that affects how the body creates energy, which can lead to various symptoms, including significant tiredness and muscle weakness.

The main purpose of this study is to see if KL1333 can help people feel less tired and improve their physical abilities. The researchers will be comparing KL1333 to a 'placebo', which is a pill that looks identical to KL1333 but doesn't have any active medicine in it. This comparison helps scientists understand if any changes seen in participants are truly due to the medicine or just a result of being in a study. They also want to make sure KL1333 is safe and that people can take it without too many problems.

Taking part in this study could help doctors learn more about primary mitochondrial disease and potentially find new ways to manage the symptoms that affect daily life for people with PMD. The information gathered will be used to see if KL1333 could become a real treatment option in the future.

Key takeaways

The study is testing KL1333 for primary mitochondrial disease.
It aims to reduce fatigue and improve physical strength.
Participants will receive either KL1333 or a dummy pill (placebo).
The study involves regular appointments and lasts over a year.
Your general health will be monitored closely throughout.
Participation is voluntary, and you can withdraw anytime.

Who may be eligible?

To join this study, you need to be an adult, aged 18 or over. You must have a confirmed diagnosis of primary mitochondrial disease, caused by a specific genetic change. This condition should be affecting multiple parts of your body, for example, conditions like MELAS or KSS.

You should also have experienced ongoing tiredness (fatigue) for at least three months, and this tiredness should be at a moderate level or worse. Additionally, you need to have some muscle weakness, particularly in your upper legs, or find it difficult when exercising, like going up hills or stairs.

Finally, you should be generally stable health-wise, apart from your mitochondrial disease symptoms, and be able to attend all the study appointments. The study team will do some checks to make sure everyone joining meets these requirements.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a confirmed diagnosis of primary mitochondrial disease (PMD) with a known genetic cause?
Have you been experiencing moderate or worse tiredness (fatigue) for at least 3 months?
Do you have some muscle weakness or difficulty with exercise?
Are you generally stable in your health, apart from your PMD symptoms?
Are you able to attend all study appointments?

Answer every question to see your result.

What does participation involve?

If you decide to take part in the FALCON study, it will involve three main stages. First, there's a 'screening and baseline' stage, which could last between 8 to 12 weeks. During this time, the research team will do tests to see if the study is right for you and gather starting information about your health.

Next comes the 'treatment' stage, lasting 48 weeks. During this time, you will take either the study medicine (KL1333) or a placebo (a pill with no active medicine) twice a day. You won't know which one you are taking, and neither will the study team. For every five people taking part, three will receive KL1333 and two will receive the placebo.

After the treatment stage, there's a 'safety follow-up' period of 5 weeks. In total, your involvement in the study will last between 61 to 65 weeks (about 14 to 15 months). Throughout this time, you'll need to attend study appointments regularly and complete electronic questionnaires about your health.

Potential risks and benefits

By taking part, you might benefit from receiving KL1333, which could potentially improve your energy levels and physical abilities, although there's also a chance you'll receive the placebo. All new medicines have potential risks, and KL1333 might cause side effects, which the study will carefully monitor. You will receive regular medical check-ups throughout the study. Remember, joining a study is always voluntary, and you have the right to withdraw at any time without it affecting your usual medical care.

Locations (35)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of California, Irvine - ALS & Neuromuscular Center
Verified postcode
Orange, United States· Recruiting
The Regents of the University of California - San Diego
Verified postcode
San Diego, United States· Recruiting
Children's Hospital Colorado - Center for Cancer and Blood Disorders (CCBD) - Anschutz Medical Campus Location
Verified postcode
Aurora, United States· Recruiting
Rare Disease Research, LLC
Verified postcode
Atlanta, United States· Recruiting
Mayo Clinic
Verified postcode
Rochester, United States· Recruiting
Washington University School of Medicine - Center for Advanced Medicine (CAM) - Neuroscience Center
Verified postcode
St Louis, United States· Not yet recruiting
Columbia University Irving Medical Center
Verified postcode
New York, United States· Not yet recruiting
Akron Children's Hospital
Verified postcode
Akron, United States· Recruiting
UPMC Children's Hospital of Pittsburgh
Verified postcode
Pittsburgh, United States· Not yet recruiting
Baylor College of Medicine (BCM)
Verified postcode
Houston, United States· Recruiting
The University of Texas Health Science Center at Houston
Verified postcode
Houston, United States· Recruiting
Hopital Universitaire de Bruxelles (H.U.B)/ Academisch Ziekenhuis Brussel
Verified postcode
Brussels, Belgium· Recruiting

Common questions

What is primary mitochondrial disease?

It's a health condition that affects how your body makes energy, which can lead to symptoms like tiredness and muscle weakness.

What is a placebo?

A placebo is a tablet that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers compare the real medicine's effects.

Will I know if I'm getting the medicine or the placebo?

No, neither you nor the study team will know during the study. This is common practice to keep the results fair and accurate.

How long will I be in the study?

The total study will last between 61 to 65 weeks, which is about 14 to 15 months.

Can I leave the study at any time?

Yes, you are free to leave the study at any point if you change your mind, and this won't affect your regular medical care.

How to find out more

Communication Manager

clinicaltrialinfo@abliva.com +46462756220 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.