INTRODUCING THE
FALCON STUDY
A Mito clinical study
Ask Nurse Martha if you may be eligible to join the FALCON study.
In the FALCON study,
researchers want to know…
- Does the study drug, napazimone (KL1333), in tablet form, improve fatigue symptoms?
- Does the study drug improve physical abilities?
- Does the study drug improve the ability of people living with mito to better carry out daily activities?
Napazimone (KL1333) is an investigational study drug. It has not been approved for use in the US by the FDA or by any health authority in any country.
Who may be eligible join the FALCON study?
- Adults ages 18 and up
- Diagnosed with primary mitochondrial disease due to a gene mutation or deletion in the mitochondrial DNA
- Experiencing chronic mitochondrial fatigue and myopathy, which is muscle weakness
- Able to meet other study requirements explained by the study doctor
What to expect
5 – 8 in-person study visits (3 may be at home)
Monthly telephone visits
Study-related treatment and assessment costs are covered for study participants
Study drug is an oral tablet given twice a day
May keep taking current supplements if stable on them
Many study sites across the U.S.
You may or may not benefit from joining this study. You do not have to join the study and you may quit at any time.
The FALCON study is divided into three parts
Screening
Many tests to see if you meet the criteria to join
Treatment
Participants will be randomly assigned, like picking straws, to receive either the study drug or a placebo, which has no active ingredients.
Neither the participants nor the study team will know who is receiving the study drug or placebo. Participants will not be able to change which one they are assigned.
Safety Follow-Up Visit
For every 5 study participants, 3 will receive napazimone (KL1333)
and 2 will receive the placebo, which has no active ingredients
What is already known about the study drug?
The study drug, napazimone (KL1333), has been studied in healthy people and in patients with Primary Mitochondrial Disease. The most common side effects noted include:
- Abdominal discomfort or pain
- Abdominal distension (bloating)
- Diarrhea
- Nausea
The risks, benefits, and effectiveness of napazimone (KL1333) are not fully known.
About clinical studies
- Clinical research studies, which are also called clinical trials, are conducted by researchers who are looking for better ways to prevent, diagnose, or treat a health condition
- The data collected from clinical studies is shared with regulatory organizations who decide if a new medicine is safe, works as intended, and can be approved
- Studies follow specific laws to protect the rights, safety, well-being and privacy of participants
To learn more about clinical research studies, visit:
CISCRP – a not for profit organization that works to
educate people about clinical trials
National Institutes of Health: Clinical Research Trials
and You – a government hub for information about clinical trials
Why join?
- Joining the FALCON study will help researchers learn if napazimone (KL1333) works as intended to help improve symptoms in people living with primary mitochondrial disease, also known as Mito.
- Joining a clinical trial is an important, personal decision. Talk to your doctor to understand if it is the right decision for you.
What else should I consider?
There is some risk involved in any clinical study. Possible risks for participants include:
- May experience a side effect or health problem
- May get the placebo
- The study drug may not work or may not work better than the standard treatment
- May require more time and have more doctor’s visits than their regular healthcare
Learn More
Talk to your doctor to find out if the FALCON study is an option for you
Visit ClinicalTrials.gov and search for NCT05650229
Study sponsor
Abliva is a Member of Pharming Group N.V.
Pharming Group N.V. is a biopharmaceutical company developing innovative medicines to serve the unserved rare disease patient.
