Primary Mitochondrial Disease (PMD)
Conditions
Interventions
Product Name: OMT-28
Product Code: OMT-28
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: not available
Current Sponsor code: OMT-28
Other descriptive name: 2-{[(8Z)-13-[(methylcarbamoyl)forma
Sponsors
OMEICOS Therapeutics GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
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=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To determine the responder rate of patients with a between phase difference in GDF-15 of at least 20% decrease after 12 weeks of treatment. - To assess safety and tolerability of OMT-28 at 24 mg;Secondary Objective: - To determine the responder rate of patients with a between phase difference in GDF-15 of at least 20 % decrease after 24 weeks of treatment. - To evaluate and compare the change in GDF-15 before and after the administration of OMT-28. - To assess the pharmacokinetics (PK) of OMT-28 administered once daily in patients with PMD.;Primary end point(s): Primary Efficacy Endpoint: • Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease after 12 weeks of treatment. Primary Safety Endpoints: • Incidence, severity, seriousness, reported causality, and duration of TEAEs, clinically significant changes in safety laboratory, vital signs, and 12-lead ECG;Timepoint(s) of evaluation of this end point: Visit 1 Screening Visit 2 Baseline/Start of Treatment Visit 3 Visit 4 Visit 5 End of Treatment Visit 6 Follow-up Visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Efficacy Endpoints: • GDF-15 during evaluation phases, change in GDF-15 during evaluations phases and comparison of GDF-15 between evaluation phases. • Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease after 24 weeks of treatment. Secondary Pharmacokinetic Endpoints: • Trough plasma concentrations (Ctrough) of OMT-28 at Visit 2 to Visit 5. • Plasma concentration of OMT-28 approximately at Cmax and one further timepoint after single dose (Visit 2) and at steady state (Visit 4).;Timepoint(s) of evaluation of this end point: Visit 1 Screening Visit 2 Baseline/Start of Treatment Visit 3 Visit 4 Visit 5 End of Treatment Visit 6 Follow-up Visit | — |
Countries
Germany, Italy
Contacts
Public ContactLuciana Summo
OMEICOS Therapeutics GmbH
Outcome results
None listed