OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) (NCT05972954) | Clinical Trial Compass
CompletedPhase 2
OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
Germany, Italy28 participantsStarted 2023-05-22
Plain-language summary
The goal of this clinical trial is to learn about the treatment effects of the investigational new drug OMT-28 in patients with Primary Mitochondrial Disease.
The main question\[s\] it aims to answer are:
* Is OMT-28 safe and well tolerated in this patient population?
* Does OMT-28 reduce Growth Differentiation Factor 15 (GDF-15) and other relevant blood markers of mitochondrial dysfunction and inflammation?
* Does OMT-28 improve symptoms of the disease, e.g. fatigue or exercise intolerance?
Participants will be asked to participate in 6 study visits at an experienced clinical center, including physical examinations and exercise tests, and take the study medication regularly once per day according to the protocol.
Researchers will compare for every participant the results after 3 months and 6 months of treatment with a preceding 3 month period of standard care treatment to investigate the effects of OMT-28 on clinical parameters and a number of blood parameters.
Who can participate
Age range18 Years – 60 Years
SexALL
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Inclusion criteria
✓. Documented mutation resulting in mitochondrial disease: mitochondrial tRNA point mutations, including m3243A\>G, m8344A\>G, and single mtDNA deletions
✓. 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\[8\])
✓. GDF-15 between 1,200 ng/L and 10,000 ng/L at screening
✓. Ability to perform the exercise tests
Exclusion criteria
✕. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study
✕. 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
✕. Subjects with a history of cancer in the last 5 years
✕. Hypertension defined as systolic BP \>160 mmHg or diastolic BP \>100 mmHg at screening
What they're measuring
1
Responder rate
Timeframe: 12 weeks treatment vs. 12 weeks baseline
2
Number of Treatment Emergent Adverse Events (TEAE)
✕. Uncontrolled Diabetes mellitus according to investigator's assessment
✕. Stroke-like episodes or seizures occurred within last 6 months
✕. Motoric abnormalities other than related to the mitochondrial disease interfering with the outcome parameters
✕. History or evidence of active tuberculosis (TB) infection, any co-disease with inflammatory condition (e.g., Inflammatory Bowel Disease (IBD) etc.)