Not recruitingRare disease

Study on the Safety and Effects of OMT-28 for Patients with Primary Mitochondrial Disease and Muscle or Heart Problems

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Primary Mitochondrial Disease, which can lead to muscle problems, known as myopathy, and heart issues, referred to as cardiomyopathy. The trial will test a new treatment called OMT-28, which is taken as a capsule. The purpose of the study is to see how safe and tolerable this treatment is for patients, as well as to understand its effects on the body.

Participants in the study will take the OMT-28 capsule for a period of 12 weeks. During this time, researchers will monitor the patients to see if there is a significant improvement in their condition, specifically looking for a decrease in a marker called GDF-15, which is related to inflammation. The study will also keep track of any side effects or changes in health that might occur while taking the treatment.

The trial aims to gather important information about how OMT-28 works in people with Primary Mitochondrial Disease and whether it can help reduce symptoms associated with muscle and heart problems. This research is an important step in finding new ways to manage and treat this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and performing necessary tests to ensure the patient meets the criteria for participation.
Step 2
Medication administration
The patient begins taking the study medication, OMT-28, which is provided in capsule form. The dosage is 24 mg, taken orally.
The medication is administered daily for a duration of 12 weeks.
Step 3
Monitoring and evaluation
Throughout the 12-week period, regular monitoring is conducted to assess the patient's response to the medication.
This includes measuring levels of GDF-15, a marker used to evaluate the effectiveness of the treatment, aiming for a decrease of at least 20%.
Step 4
Safety assessments
Safety assessments are performed to monitor any adverse effects. This involves checking vital signs, conducting laboratory tests, and performing 12-lead ECGs.
The incidence, severity, and duration of any side effects are recorded and analyzed.
Step 5
Final evaluation
At the end of the 12-week treatment period, a final evaluation is conducted to determine the overall effectiveness and safety of the medication.
The primary endpoint is the number of patients showing a significant decrease in GDF-15 levels.

Who can join the trial?

8 criteria

Any gender, age 18 to 60 years
Must have a documented genetic change (mutation) that causes a mitochondrial disease. This includes specific changes like mitochondrial tRNA point mutations (such as m3243A>G, m8344A>G) and single mtDNA deletions.
Must have a diagnosis of Cardiomyopathy, which is a heart condition. This can be shown by LV hypertrophy (thickening of the heart muscle), LVEF<50% (a measure of how well the heart pumps), or late gadolinium enhancement on a heart MRI. Alternatively, must have Myopathy, a muscle condition, as defined by specific guidelines.
Must have a GDF-15 level between 1,200 to 10,000 pg/mL, which is a specific blood test result, measured at the start of the study.
Must be able to perform exercise tests.
Must be willing and able to sign an Informed Consent, which is a document that explains the study and confirms your agreement to participate. This includes agreeing to data protection requirements.
Must be able and willing to follow the study's rules and requirements.
Female patients and female partners of male patients who can have children must agree not to become pregnant during the study and for 30 days after the last dose of the study medication.

Who cannot join the trial?

3 criteria

Patients who are not diagnosed with Primary Mitochondrial Disease cannot participate. This is a condition where the mitochondria, the energy-producing parts of cells, do not work properly.
Patients who are younger than 3 years old cannot participate.
Patients who are part of a vulnerable population, meaning they might need special protection or care, cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

A.O.U. Policlinico G. Martino Di Messina
Verified
Messina, Italy
Azienda Unita Sanitaria Locale Di Bologna
Verified
Bologna, Italy
Stichting Radboud University Medical Center
Responsive
Nijmegen, The Netherlands

Trial locations

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Investigated drugs

OMT-28 is a medication being studied for its effects on patients with myopathy and/or cardiomyopathy, which are conditions affecting the muscles and heart. The trial aims to see if this medication can reduce inflammation and improve muscle and heart function. Researchers are also looking at how safe and tolerable the medication is for patients over a period of time.

2-{[(8Z)-13-[(Methylcarbamoyl)Formamido]-Tridec-8-En-1-Yl]Oxy}Acetic Acid

What is already known about the treatment

OMT-28 – OMT-28 is administered orally and is currently being studied in clinical trials for its safety, tolerability, and effects in patients with Primary Mitochondrial Disease, particularly those with muscle and heart issues and inflammation. It is in the investigational stage, with ongoing research to determine its effectiveness and safety profile. The main therapeutic indication for OMT-28 is to address symptoms associated with mitochondrial dysfunction. At the molecular level, OMT-28 is believed to work by modulating pathways involved in mitochondrial energy production, although the exact mechanism is still under investigation. It is classified pharmacologically as a mitochondrial modulator.

Investigated diseases

Primary Mitochondrial Disease – This is a group of disorders caused by dysfunctional mitochondria, the parts of cells responsible for energy production. These diseases can affect multiple systems in the body, leading to a wide range of symptoms. Commonly, they impact organs and tissues with high energy demands, such as the brain, muscles, and heart. Symptoms may include muscle weakness, neurological problems, and fatigue. The progression of the disease varies widely among individuals, with some experiencing mild symptoms and others facing more severe challenges. The variability in symptoms and progression makes it a complex condition to understand and manage.