A clinical study of a new drug (OMT-28) for use in patients suffering from the genetic disease PMD including muscle disease and/or Heart muscle disease and inflammation (PMD-OPTION)

Phase 1

Conditions: Primary Mitochondrial Disease (PMD)
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Registration Number: EUCTR2022-003307-16-DE

Lead Sponsor: OMEICOS Therapeutics GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 32

Inclusion Criteria

1. Any gender, age 18 to 60 years
2. Documented mutation resulting in mitochondrial disease: mitochondrial tRNA point mutations, including m3243A>G, m8344A>G, and single mtDNA deletions
3. Diagnosis of Cardiomyopathy defined as LV hypertrophy and/or LVEF<50% and/or late gadolinium enhancement on cardiac MRI and/or Myopathy as defined by the International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adult (Mancuso et al. 2017)
4. GDF-15 between 1,200 to 10,000 pg/mL measured at screening
5. Ability to perform the exercise tests
6. Willing and able to provide a signed Informed Consent, as well as written documentation in accordance with country and local privacy requirements, e.g., written data protection consent
7. Able and willing to comply with the requirements of this study protocol
8. Both female patients, as well as, female partners of male patients who are of child- bearing potential must be willing to not become pregnant for the complete duration of the study (30 days after the last dose of study medication).
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 32
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study
2. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data
3. Subjects with a history of cancer in the last 5 years
4. Hypertension defined as systolic BP >160 mmHg or diastolic BP >100 mmHg at screening
5. Uncontrolled Diabetes mellitus according to investigator’s assessment
6. Stroke-like episodes or seizures occurred within last 6 months
7. Motoric abnormalities other than related to the mitochondrial disease interfering with the outcome parameters
8. History or evidence of active tuberculosis (TB) infection, any co-disease with inflammatory condition (e.g. Inflammatory Bowel Disease (IBD) etc.)
9. Patients with a positive hepatitis panel and/or positive immunodeficiency virus test at screening
10. Regular use of steroid, non-steroidal anti-inflammatory drug (NSAID), or colchicine within 30 days before screening
11. Chronic use of Metformin
12. Use of fish oil / omega-3 fatty acid supplements within 2 weeks before screening
13. Drinking more than 9 standard cups of alcohol per week and/or more than 3 standard cups of alcohol per occasion
14. Positive drug and alcohol screen (including opiates, methadone, cocaine, amphetamines [including ecstasy], cannabinoids, barbiturates, benzodiazepines)
15. Any significant hepatic disease (defined as the presence of at least one of the following: AST, ALT, GGT, total bilirubin, or alkaline phosphatase >3x upper limit normal)
16. Receiving any investigational therapy or any approved therapy for investigational use within 30 days or 5 half-lives prior to screening (whichever is longer)
17. Received any vaccines (including the booster vaccination for Covid-19) within two weeks prior to Visit 1
18. Females of childbearing potential (those who are not surgically sterilized or post- menopausal for at least 1 year) are excluded from participation in the study unless they agree to use adequate contraception as described in Appendix 11.4 of the protocol
19. Males (including sterilized subjects) and whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 30 days after the last dose of study drug. They must agree to immediately inform the investigator if his partner becomes pregnant during the study.
20. Subjects who have previously been exposed to OMT-28, whether responder or nonresponder
21. Any use of statins (HMG-CoA reductase inhibitors)
22. Use of quinine, tacrolimus, mycophenolate mofetil, ciclosporin, serotine receptortype 1 agonist, penicillin G, penicillamine (d-penicillamine), nicotinic acid (niacin), colchicine, isotretinoin, and amiodarone, PPAR activators, AMPK activators, Sirtuin activators, Steroids, COX-inhibitors