OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
- Conditions
- Primary Mitochondrial Disease
- Registration Number
- NCT05972954
- Lead Sponsor
- Omeicos Therapeutics GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- All
- Target Recruitment
- 32
Inclusion Criteria:<br><br> 1. Documented mutation resulting in mitochondrial disease: mitochondrial tRNA point<br> mutations, including m3243A>G, m8344A>G, and single mtDNA deletions<br><br> 2. Diagnosis of Cardiomyopathy defined as LV hypertrophy and/or LVEF<50% and/or late<br> gadolinium enhancement on cardiac MRI and/or Myopathy as defined by the<br> International Workshop: Outcome measures and clinical trial readiness in primary<br> mitochondrial myopathies in children and adult (Mancuso et al. 2017[8])<br><br> 3. GDF-15 between 1,200 ng/L and 10,000 ng/L at screening<br><br> 4. Ability to perform the exercise tests<br><br> 6. Willing and able to provide a signed Informed Consent, as well as written<br> documentation in accordance with country and local privacy requirements, e.g.,<br> written data protection consent 7. Able and willing to comply with the requirements<br> of this study protocol 8. Both female patients, as well as, female partners of male<br> patients who are of child-bearing potential must be willing to not become pregnant<br> for the complete duration of the study (30 days after the last dose of study<br> medication).<br><br>Exclusion Criteria:<br><br> 1. Pregnant, breastfeeding, or unwilling to practice birth control during participation<br> in the study<br><br> 2. Presence of a condition or abnormality that in the opinion of the Investigator would<br> compromise the safety of the patient or the quality of the data<br><br> 3. Subjects with a history of cancer in the last 5 years<br><br> 4. Hypertension defined as systolic BP >160 mmHg or diastolic BP >100 mmHg at screening<br><br> 5. Uncontrolled Diabetes mellitus according to investigator's assessment<br><br> 6. Stroke-like episodes or seizures occurred within last 6 months<br><br> 7. Motoric abnormalities other than related to the mitochondrial disease interfering<br> with the outcome parameters<br><br> 8. History or evidence of active tuberculosis (TB) infection, any co-disease with<br> inflammatory condition (e.g., Inflammatory Bowel Disease (IBD) etc.)<br><br> 9. Patients with a positive hepatitis panel and/or positive immunodeficiency virus test<br> at screening<br><br> 10. Regular use of steroid, non-steroidal anti-inflammatory drug (NSAID), or colchicine<br> within 30 days before screening<br><br> 11. Chronic use of Metformin<br><br> 12. Use of fish oil / omega-3 fatty acid supplements within two weeks before screening<br><br> 13. Drinking more than 9 standard cups of alcohol per week and/or more than 3 standard<br> cups of alcohol per occasion<br><br> 14. Positive drug and alcohol screen (including opiates, methadone, cocaine,<br> amphetamines [including ecstasy], cannabinoids, barbiturates, benzodiazepines)<br><br> 15. Any significant hepatic disease<br><br> 16. Receiving any investigational therapy or any approved therapy for investigational<br> use within 30 days or 5 half-lives prior to screening (whichever is longer)<br><br> 17. Received any vaccines (including the booster vaccination for COVID-19) within two<br> weeks prior to Visit 1<br><br> 18. Females of childbearing potential (those who are not surgically sterilized or<br> post-menopausal for at least 1 year) are excluded from participation in the study<br> unless they agree to use adequate contraception as described in Appendix 11.4<br><br> 19. Males (including sterilized subjects) and whose female partners have child-bearing<br> potential, must agree to use male contraception (condoms) during the period from the<br> time of signing the informed consent form (ICF) through 30 days after the last dose<br> of study drug. They must agree to immediately inform the investigator if his partner<br> becomes pregnant during the study<br><br> 20. Subjects who have previously been exposed to OMT-28, whether responder or<br> non-responder.<br><br> 21. Any use of statins (HMG-CoA reductase inhibitors)<br><br> 22. Use of quinine, tacrolimus, mycophenolate mofetil, ciclosporin, serotine<br> receptor-type 1 agonist, penicillin G, penicillamine (d-penicillamine), nicotinic<br> acid (niacin), colchicine, isotretinoin, and amiodarone, peroxisome<br> proliferator-activated receptor (PPAR) activators, AMP-activated protein kinase<br> (AMPK) activators, sirtuin activators, steroids, cyclooxygenase inhibitors.
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Responder rate;Number of Treatment Emergent Adverse Events (TEAE)
- Secondary Outcome Measures
Name Time Method Responder rate;Change in plasma concentration of GDF-15 [ng/L];Pharmacokinetics: Ctrough [ng/ml];Pharmacokinetics: Cmax [ng/ml]