OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION)

NCT05972954 | Completed Phase 2

• 1 other identifier: OMT28-C0203
• Study Type: interventional
• Target: 28
• Locations: 3 countries
• Sites: 9

Brief 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.

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

Outcome Measures

Primary Outcomes (2)

• Responder rate

  Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease

  12 weeks treatment vs. 12 weeks baseline

• Number of Treatment Emergent Adverse Events (TEAE)

  Compare the number of TEAEs during and between evaluation phases

  up to 28 weeks

Secondary Outcomes (4)

• Responder rate

  24 weeks treatment vs. 12 weeks baseline

• Change in plasma concentration of GDF-15 [ng/L]

  12 weeks treatment vs. 12 weeks baseline

• Pharmacokinetics: Ctrough [ng/ml]

  weeks 12, 16, 24 and 28

• Pharmacokinetics: Cmax [ng/ml]

  week 12 and week 24

Study Arms (1)

Treatment with OMT-28

EXPERIMENTAL

24mg OMT-28, oral capsule, for 12 weeks

Drug: OMT-28

Interventions

OMT-28 DRUG

once daily

Treatment with OMT-28

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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
• \. 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).

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Omeicos Therapeutics GmbH: lead

Study Sites (9)

Universitätsklinikum Bonn, Sektion Neuromuskuläre Erkrankungen, Klinik und Poliklinik für Neurologie, Venusberg-Campus 1, Gebäude 80, NPP

Bonn, 53127, Germany

Location

Medizinisch Fakultät der Martin-Luther-Universität Halle-Wittenberg Universitätsklinik und Poliklinik für Neurologie Universitätsklinikum , Ernst-Grube-Str. 40

Halle, 06120, Germany

Location

Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik, Klinikum der Universität München, Ludwig-Maximilians-Universität (LMU Klinikum), Ziemssenstr. 1a

Munich, 80336, Germany

Location

IRCCS Institute of Neurological Science of Bologna, University of Bologna, Department of Biomedical and Neuromotor Science (DIBINEM), Ospedale Bellaria Via Altura, 3

Bologna, 40139, Italy

Location

U.O.C. di Neurologia e Malattie Neuromuscolari

Messina, 98125, Italy

Location

IRCCS Istituto Neurologico Carlo Besta, SC Servizio Di Medicina Di Laboratorio - Genetica Medica E Neurogenetica, Via Celoria 11

Milan, 20133, Italy

Location

Azienda Ospedaliero Universitaria Pisana, P.O. Santa Chiara, U.O. Neurologia, Edificio 13, Via Roma 67

Pisa, 56126, Italy

Location

UOC di neurofisiopatologia

Roma, 8-00168, Italy

Location

Radboud University Nijmegen Medical Centre

Nijmegen, Gelderland, 6500 HB, Netherlands

Location

MeSH Terms

Conditions

Muscular Diseases; Cardiomyopathies; Mitochondrial encephalopathy; Acidosis, Lactic; MELAS Syndrome; Syndrome; MERRF Syndrome; Deafness; Noninsulin-dependent diabetes mellitus with deafness

Condition Hierarchy (Ancestors)

Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Heart Diseases; Cardiovascular Diseases; Acidosis; Acid-Base Imbalance; Metabolic Diseases; Nutritional and Metabolic Diseases; Mitochondrial Encephalomyopathies; Mitochondrial Myopathies; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Disease; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Myoclonic Epilepsies, Progressive; Epilepsies, Myoclonic; Epilepsy, Generalized; Epilepsy; Epileptic Syndromes; Hearing Loss; Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Sensation Disorders; Neurologic Manifestations; Signs and Symptoms

Study Officials

• Robert Fischer, MD

  Omeicos Therapeutics GmbH

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2023
• First Posted: August 2, 2023
• Study Start: May 22, 2023
• Primary Completion: May 14, 2025
• Study Completion: July 30, 2025
• Last Updated: January 22, 2026

Record last verified: 2025-06

Locations

IT (5); DE (3); NL (1)