In short
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.
At a glance
- 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)
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. It is a synthetic biochemical compound that comes in the form of a capsule and is taken orally.
Target Condition: Primary Mitochondrial Disease
OMT-28 is being investigated for the treatment of Primary Mitochondrial Disease (PMD). 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.
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
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. 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.
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
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
Study Objectives and Endpoints
The main objectives of this study are: 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. GDF-15 is a protein that can indicate the level of cellular stress and is often elevated in mitochondrial diseases. To assess the safety and tolerability of OMT-28 at a dose of 24 mg.
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.
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)
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.