Primary Mitochondrial DiseasePhase 2PMD-OPTIONNCT05972954
OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)
A Phase 2 study testing OMT-28 in people with Primary Mitochondrial Disease, run by Omeicos Therapeutics GmbH at 9 sites in 3 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Primary Mitochondrial Disease
- What is being tested?
- OMT-28
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 9 sitesin 3 countries
- Study dates
- 22 May 2023to 30 Jul 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
- 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).
Who cannot join
- 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
- Uncontrolled Diabetes mellitus according to investigator's assessment
- Stroke-like episodes or seizures occurred within last 6 months
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- 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.)
- Patients with a positive hepatitis panel and/or positive immunodeficiency virus test at screening
- Regular use of steroid, non-steroidal anti-inflammatory drug (NSAID), or colchicine within 30 days before screening
- Chronic use of Metformin
- Use of fish oil / omega-3 fatty acid supplements within two weeks before screening
- Drinking more than 9 standard cups of alcohol per week and/or more than 3 standard cups of alcohol per occasion
- Positive drug and alcohol screen (including opiates, methadone, cocaine, amphetamines [including ecstasy], cannabinoids, barbiturates, benzodiazepines)
- Any significant hepatic disease
- Receiving any investigational therapy or any approved therapy for investigational use within 30 days or 5 half-lives prior to screening (whichever is longer)
- Received any vaccines (including the booster vaccination for COVID-19) within two weeks prior to Visit 1
- 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
- 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
- Subjects who have previously been exposed to OMT-28, whether responder or non-responder.
- Any use of statins (HMG-CoA reductase inhibitors)
- Use of quinine, tacrolimus, mycophenolate mofetil, ciclosporin, serotine receptor-type 1 agonist, penicillin G, penicillamine (d-penicillamine), nicotinic acid (niacin), colchicine, isotretinoin, and amiodarone, peroxisome proliferator-activated receptor (PPAR) activators, AMP-activated protein kinase (AMPK) activators, sirtuin activators, steroids, cyclooxygenase inhibitors.
Trial locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
Italy5 sites
IRCCS Institute of Neurological Science of Bologna, University of Bologna, Department of Biomedical and Neuromotor Science (DIBINEM), Ospedale Bellaria Via Altura, 3
Bologna, 40139
U.O.C. di Neurologia e Malattie Neuromuscolari
Messina, 98125
IRCCS Istituto Neurologico Carlo Besta, SC Servizio Di Medicina Di Laboratorio - Genetica Medica E Neurogenetica, Via Celoria 11
Milan, 20133
Azienda Ospedaliero Universitaria Pisana, P.O. Santa Chiara, U.O. Neurologia, Edificio 13, Via Roma 67
Pisa, 56126
UOC di neurofisiopatologia
Roma, 8-00168
Germany3 sites
Universitätsklinikum Bonn, Sektion Neuromuskuläre Erkrankungen, Klinik und Poliklinik für Neurologie, Venusberg-Campus 1, Gebäude 80, NPP
Bonn, 53127
Medizinisch Fakultät der Martin-Luther-Universität Halle-Wittenberg Universitätsklinik und Poliklinik für Neurologie Universitätsklinikum , Ernst-Grube-Str. 40
Halle, 06120
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik, Klinikum der Universität München, Ludwig-Maximilians-Universität (LMU Klinikum), Ziemssenstr. 1a
Munich, 80336
Netherlands1 site
Radboud University Nijmegen Medical Centre
Nijmegen, Gelderland, 6500 HB
About this study
Official title: A Phase 2a Safety, Tolerability, and Pharmacodynamic Study of OMT-28 in PMD Patients With Myopathy and/or Cardiomyopathy and Inflammation (PMD-OPTION)
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:
Read more
- 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.
Read the detailed description
This is an open label, single-arm, multiple-phase and multicenter Phase 2a study to evaluate the efficacy, safety, and pharmacokinetics of a single OMT-28 dose (24 mg given once daily) in patients with Primary Mitochondrial Disease and clinical manifestation of myopathy and/or cardiomyopathy.
Patients are eligible if they have
- Primary Mitochondrial Disease with a documented mitochondrial transfer ribonucleic acid (tRNA) point mutation, including m3243A>G, m8344A>G, or single mitochondrial DNA (mtDNA) deletions,
- a clinically relevant myopathy and/or cardiomyopathy confirmed following standard guidelines,
- a blood plasma GDF-15 concentration > 1200 ng/L and < 10.000 ng/L at screening
Participation in the study is divided into 3 parts:
- Screening and baseline: 12 weeks
- Treatment: 24 weeks
- Safety follow-up: 4 weeks
Total duration: 40 weeks
Safety will be monitored throughout the study. Blood samples for safety, pharmacodynamics and pharmacokinetics will be collected at every of the 6 study visits. Exercise tests (6/12-minutes walking test, 5xSST), transthoracic echocardiography, patient reported outcomes (e.g. Fatigue Severity Scale and Patients' Global Impression of Change (PGIC) scale) will be assessed at prespecified visits. Patients will be provided with a diary to record timing of drug administration and clinical symptoms while not on site. Diaries will be reviewed and checked for compliance at each non-resident visit to the clinical site.
- Conditions
- Primary Mitochondrial Disease
- Being tested
- OMT-28
- Keywords
- Myopathy, Cardiomyopathy, Mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS) syndrome, Myoclonic epilepsy with ragged red fibers (MERRF), Maternally inherited diabetes and deafness (MIDD) syndrome, GDF-15
- Medical subject headings
- Muscular Diseases, Cardiomyopathies, Mitochondrial encephalopathy, Acidosis, Lactic, MELAS Syndrome, Syndrome, MERRF Syndrome, Deafness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OMT-28Drug
once daily
| Study group | Role | What they receive |
|---|---|---|
| Treatment with OMT-28 | Experimental | 24mg OMT-28, oral capsule, for 12 weeks Receives: Drug: OMT-28 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Responder rate
Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease
Measured: 12 weeks treatment vs. 12 weeks baseline
Number of Treatment Emergent Adverse Events (TEAE)
Compare the number of TEAEs during and between evaluation phases
Measured: up to 28 weeks
Secondary outcomes (4)
Responder rate
Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20%
Measured: 24 weeks treatment vs. 12 weeks baseline
Change in plasma concentration of GDF-15 [ng/L]
GDF-15 plasma concentration during evaluation phases, change in GDF-15 concentration during evaluations phases and comparison of GDF-15 concentration between evaluation phases
Measured: 12 weeks treatment vs. 12 weeks baseline
Pharmacokinetics: Ctrough [ng/ml]
Trough plasma concentrations (Ctrough) of OMT-28
Measured: weeks 12, 16, 24 and 28
Pharmacokinetics: Cmax [ng/ml]
Plasma concentration of OMT-28 approximately at Cmax and one further timepoint after single dose
Measured: week 12 and week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2023
- Study start22 May 2023
- Primary completion14 May 2025
- Study completion30 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Mitochondrial DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Omeicos Therapeutics GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert Fischer, MD, Study director, Omeicos Therapeutics GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05972954
- Sponsor's protocol ID
- OMT28-C0203
- Acronym
- PMD-OPTION
- Status last verified
- Jun 2025
- First submitted
- 27 Jun 2023
- Last updated
- 22 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Safety, Tolerability, and Pharmacodynamic Study of OMT-28 in PMD Patients With Myopathy and/or Cardiomyopathy and Inflammation (PMD-OPTION)
- ClinicalTrials.gov ID
- NCT05972954
- Acronym / sponsor ID
- PMD-OPTION / OMT28-C0203
- Conditions
- Primary Mitochondrial Disease
- Keywords
- Myopathy, Cardiomyopathy, Mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS) syndrome, Myoclonic epilepsy with ragged red fibers (MERRF), Maternally inherited diabetes and deafness (MIDD) syndrome, GDF-15
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Omeicos Therapeutics GmbHIndustry (pharma, biotech, devices)
- Study start
- 22 May 2023 (actual)
- Primary completion
- 14 May 2025 (actual)
- Study completion
- 30 Jul 2025 (actual)
- First posted
- 2 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is an open label, single-arm, multiple-phase and multicenter Phase 2a study to evaluate the efficacy, safety, and pharmacokinetics of a single OMT-28 dose (24 mg given once daily) in patients with Primary Mitochondrial Disease and clinical manifestation of myopathy and/or cardiomyopathy.
Patients are eligible if they have
- Primary Mitochondrial Disease with a documented mitochondrial transfer ribonucleic acid (tRNA) point mutation, including m3243A>G, m8344A>G, or single mitochondrial DNA (mtDNA) deletions,
- a clinically relevant myopathy and/or cardiomyopathy confirmed following standard guidelines,
- a blood plasma GDF-15 concentration > 1200 ng/L and < 10.000 ng/L at screening
Participation in the study is divided into 3 parts:
- Screening and baseline: 12 weeks
- Treatment: 24 weeks
- Safety follow-up: 4 weeks
Total duration: 40 weeks
Safety will be monitored throughout the study. Blood samples for safety, pharmacodynamics and pharmacokinetics will be collected at every of the 6 study visits. Exercise tests (6/12-minutes walking test, 5xSST), transthoracic echocardiography, patient reported outcomes (e.g. Fatigue Severity Scale and Patients' Global Impression of Change (PGIC) scale) will be assessed at prespecified visits. Patients will be provided with a diary to record timing of drug administration and clinical symptoms while not on site. Diaries will be reviewed and checked for compliance at each non-resident visit to the clinical site.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with OMT-28 | Experimental | Drug: OMT-2824mg OMT-28, oral capsule, for 12 weeks |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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
- 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).
Key exclusion criteria (22 listed)
- 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
- Uncontrolled Diabetes mellitus according to investigator's assessment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OMT-28once daily | Drug | Treatment with OMT-28 |
Primary outcome
Responder rate
Time frame: 12 weeks treatment vs. 12 weeks baseline
Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease
Number of Treatment Emergent Adverse Events (TEAE)
Time frame: up to 28 weeks
Compare the number of TEAEs during and between evaluation phases
4 secondary outcomes
Responder rate
Time frame: 24 weeks treatment vs. 12 weeks baseline
Change in plasma concentration of GDF-15 [ng/L]
Time frame: 12 weeks treatment vs. 12 weeks baseline
Pharmacokinetics: Ctrough [ng/ml]
Time frame: weeks 12, 16, 24 and 28
Pharmacokinetics: Cmax [ng/ml]
Time frame: week 12 and week 24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Fischer, MD study director
Omeicos Therapeutics GmbH
9 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing OMT-28 in people with Primary Mitochondrial Disease, run by Omeicos Therapeutics GmbH at 9 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Italy, Germany, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Responder rate (time frame: 12 weeks treatment vs. 12 weeks baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jan 2026.