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

This article discusses the ongoing clinical trials of a drug called OMT-28, also known as 2-{[(8Z)-13-[(Methylcarbamoyl)Formamido]-Tridec-8-En-1-Yl]Oxy}Acetic Acid. This medication is being studied for its potential benefits in treating patients with Primary Mitochondrial Disease (PMD) who experience muscle or heart problems. The trials aim to assess the safety, tolerability, and effectiveness of OMT-28 in improving the condition of PMD patients.

Table of Contents

What is OMT-28?

OMT-28, also known as 2-{[(8Z)-13-[(METHYLCARBAMOYL)FORMAMIDO]-TRIDEC-8-EN-1-YL]OXY}ACETIC ACID, is a new medication being studied for the treatment of primary mitochondrial disease[1]. It is a synthetic biochemical compound that comes in the form of a capsule and is taken orally[1].

Target Condition: Primary Mitochondrial Disease

OMT-28 is being investigated for the treatment of Primary Mitochondrial Disease (PMD)[1]. PMD is a group of genetic disorders that affect the mitochondria, which are tiny structures inside our cells that produce energy. When mitochondria don’t work properly, it can lead to various health problems, particularly affecting organs that require a lot of energy, such as muscles and the heart[1].

Specifically, this study focuses on PMD patients who have:

  • Myopathy: A condition causing muscle weakness and fatigue
  • Cardiomyopathy: A heart muscle disease that can lead to heart failure
  • Inflammation associated with these conditions[1]

Clinical Trial Details

The clinical trial for OMT-28 is a Phase 2a study, which means it’s an early stage of testing in humans to evaluate the drug’s effectiveness and safety[1]. The study is called “PMD-OPTION” and aims to assess how well OMT-28 works and how safe it is for patients with primary mitochondrial disease[1].

Eligibility Criteria

To participate in this study, patients must meet certain criteria. Some key inclusion criteria are:

  • Age between 18 and 60 years
  • Documented genetic mutation causing mitochondrial disease
  • Diagnosis of cardiomyopathy or myopathy
  • Ability to perform exercise tests
  • Willingness to follow the study protocol[1]

There are also several exclusion criteria, including:

  • Pregnancy or breastfeeding
  • Recent use of certain medications like steroids or metformin
  • Significant liver disease
  • Recent stroke-like episodes or seizures
  • Uncontrolled diabetes or hypertension[1]

Study Objectives and Endpoints

The main objectives of this study are:

  1. To determine how many patients respond to the treatment, as measured by a decrease in a blood marker called GDF-15. A response is defined as at least a 20% decrease in GDF-15 levels after 12 weeks of treatment[1]. GDF-15 is a protein that can indicate the level of cellular stress and is often elevated in mitochondrial diseases.
  2. To assess the safety and tolerability of OMT-28 at a dose of 24 mg[1].

Dosage and Administration

In this study, OMT-28 is given as a 24 mg capsule to be taken orally. The maximum daily dose is 24 mg, and the treatment period is expected to last up to 6 months[1].

Safety Considerations

As with any clinical trial, patient safety is a top priority. The study will closely monitor:

  • Any side effects that occur during the treatment
  • Changes in laboratory test results
  • Changes in vital signs
  • Changes in heart activity (as measured by ECG)[1]

It’s important to note that this is an investigational drug, which means it has not yet been approved for general use. The purpose of this clinical trial is to gather more information about its effectiveness and safety[1].

Aspect Details
Drug Name OMT-28 (2-{[(8Z)-13-[(Methylcarbamoyl)Formamido]-Tridec-8-En-1-Yl]Oxy}Acetic Acid)
Trial Phase Phase 2a
Target Condition Primary Mitochondrial Disease with myopathy and/or cardiomyopathy
Main Objective Determine responder rate based on GDF-15 decrease and assess safety/tolerability
Dosage 24 mg
Treatment Duration 12 weeks for primary assessment, up to 6 months total
Key Inclusion Criteria Age 18-60, documented mitochondrial mutation, cardiomyopathy or myopathy diagnosis
Key Exclusion Criteria Pregnancy, certain medication use, significant liver disease, uncontrolled diabetes
Primary Endpoints GDF-15 decrease, safety assessments (adverse events, lab tests, vital signs, ECG)

FAQ

  • What is the main purpose of the OMT-28 clinical trial? The main purpose of the OMT-28 clinical trial is to determine how many patients respond positively to the treatment, as measured by a decrease in a specific blood marker called GDF-15. The trial also aims to assess the safety and tolerability of OMT-28 at a 24 mg dose.
  • Who can participate in this clinical trial? Participants must be between 18 and 60 years old, have a documented mutation causing mitochondrial disease, and be diagnosed with either cardiomyopathy (heart muscle disease) or myopathy (muscle disease). They must also have specific levels of GDF-15 in their blood and be able to perform exercise tests.
  • How long does the treatment last in this trial? The primary assessment of the drug's effectiveness is done after 12 weeks of treatment. However, the total treatment period can last up to 6 months.
  • Are there any restrictions for participants during the trial? Yes, there are several restrictions. Participants cannot be pregnant or breastfeeding, cannot use certain medications like steroids or metformin, and must limit alcohol consumption. They also cannot have certain health conditions like significant liver disease or uncontrolled diabetes.
  • How is the effectiveness of OMT-28 measured in this trial? The primary measure of effectiveness is the number of patients who show at least a 20% decrease in their GDF-15 levels after 12 weeks of treatment. The trial also monitors for any side effects and changes in various health indicators to ensure the drug's safety.

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

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

    Not recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Germany Italy The Netherlands

Glossary

  • Primary Mitochondrial Disease (PMD): A group of genetic disorders that affect the mitochondria, which are structures in cells that produce energy. These diseases can affect various body systems, particularly those that require a lot of energy like muscles and the heart.
  • OMT-28: The study drug being tested in this clinical trial, also known by its chemical name 2-{[(8Z)-13-[(Methylcarbamoyl)Formamido]-Tridec-8-En-1-Yl]Oxy}Acetic Acid.
  • GDF-15: Growth Differentiation Factor 15, a protein in the blood that is being used as a marker to measure the effectiveness of OMT-28 in this study.
  • Cardiomyopathy: A group of diseases that affect the heart muscle, making it harder for the heart to pump blood to the rest of the body.
  • Myopathy: A general term referring to diseases of the muscle that cause weakness or a loss of muscle function.
  • Phase 2a Clinical Trial: An early stage of clinical research that aims to test the effectiveness and safety of a new drug in a small group of patients with the target condition.
  • Responder Rate: The percentage of patients who show a positive response to the treatment, in this case, measured by a decrease in GDF-15 levels.
  • Mitochondrial tRNA Point Mutations: Specific changes in the genetic material of mitochondria that can lead to mitochondrial diseases.
  • LVEF: Left Ventricular Ejection Fraction, a measurement of how much blood the left ventricle pumps out with each contraction. It's used to assess heart function.
  • Pharmacodynamic: The study of how a drug affects the body, including its mechanism of action and the relationship between drug concentration and effect.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-omt-28-for-patients-with-primary-mitochondrial-disease-and-muscle-or-heart-problems/