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    Summary
    EudraCT Number:2022-003307-16
    Sponsor's Protocol Code Number:OMT28-C0203
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2023-01-23
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2022-003307-16
    A.3Full title of the trial
    A Phase 2a Safety, Tolerability, and Pharmacodynamic Study of OMT-28 in PMD patients with myopathy and/or cardiomyopathy and inflammation (PMD-OPTION)
    Studio in fase 2a riguardante la sicurezza, la tollerabilità e la farmacodinamica di OMT-28 in pazienti affetti da malattia mitocondriale primaria (PMD) con miopatia e/o cardiomiopatia e infiammazione (PMD-OPTION)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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)
    Studio di fase 2a su sicurezza, tollerabilità e farmacodinamica di OMT-28 in pazienti con PMD con miopatia e/o cardiomiopatia e infiammazione (PMD-OPTION)
    A.3.2Name or abbreviated title of the trial where available
    PMD-OPTION
    PMD-OPTION
    A.4.1Sponsor's protocol code numberOMT28-C0203
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOMEICOS Therapeutics GmbH
    B.1.3.4CountryGermany
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportOMEICOS Therapeutics GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOMEICOS Therapeutics GmbH
    B.5.2Functional name of contact pointLuciana Summo
    B.5.3 Address:
    B.5.3.1Street AddressRobert-Rössle-Strasse 10 (Building 79)
    B.5.3.2Town/ city13125
    B.5.3.3Post codeBerlin
    B.5.3.4CountryGermany
    B.5.4Telephone number00493094894818
    B.5.5Fax number004903094894811
    B.5.6E-maill.summo@omeicos.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product name2-{[(8Z)-13-[(methylcarbamoyl)formamido]- tridec-8-en-1-yl]oxy}acetic acid
    D.3.2Product code [OMT-28]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOMT-28
    D.3.9.2Current sponsor codeOMT-28
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number24
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Mitochondrial Disease (PMD)
    Malattia mitocondriale primaria (PMD)
    E.1.1.1Medical condition in easily understood language
    Genetic disease called "Primary Mitochondrial Disease (PMD)" with myopathy and/or cardiomyopathy and inflammation
    Malattia genetica chiamata "Malattia Mitocondriale Primaria (PMD)" con miopatia e/o cardiomiopatia e infiammazione
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.0
    E.1.2Level PT
    E.1.2Classification code 10052641
    E.1.2Term Mitochondrial DNA mutation
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.0
    E.1.2Level PT
    E.1.2Classification code 10052641
    E.1.2Term Mitochondrial DNA mutation
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To determine the responder rate of patients with a between phase difference in GDF-15 of at least 20% decrease.
    - To assess safety and tolerability of OMT-28 at 24 mg
    - Determinare il tasso di risposta dei pazienti con una differenza di GDF-15 tra le fasi corrispondente a una riduzione di almeno il 20%.
    - Valutare la sicurezza e la tollerabilità di OMT-28 alla dose di 24 mg
    E.2.2Secondary objectives of the trial
    - 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.
    - Valutare e confrontare la variazione di GDF-15 prima e dopo la somministrazione di OMT-28.
    - Valutare la farmacocinetica (PK) di OMT-28 somministrato una volta al giorno nei pazienti affetti da PMD.
    E.2.3Trial contains a sub-study No
    E.3Principal 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 and/or Myopathy consistent with current US and European guidelines
    4. GDF-15 between 1,200 to 10,000 pg/mL measured at screening
    5. Ability to perform the exercise tests
    6. Received at least 2 doses of COVID-19 vaccine
    7. 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
    8. Able and willing to comply with the requirements of this study protocol
    9. 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).
    1. Qualsiasi sesso, età compresa tra 18 e 60 anni
    2. Mutazione documentata risultante nella malattia mitocondriale: mutazioni puntiformi del tRNA mitocondriale, tra cui m3243A>G, m8344A>G, e singole delezioni nello mtDNA
    3. Diagnosi di cardiomiopatia e/o miopatia coerente con le attuali linee guida statunitensi ed europee
    4. GDF-15 tra 1.200 ng/l e 10.000 ng/l allo screening
    5. Capacità di eseguire i test da sforzo
    6. Aver ricevuto almeno 2 dosi di vaccino per il COVID-19
    7. Disponibilità e capacità di fornire un consenso informato firmato, nonché la documentazione scritta in conformità ai requisiti locali e nazionali in materia di privacy, es. consenso scritto alla protezione dei dati
    8. Disponibilità e capacità di rispettare i requisiti di questo protocollo di studio
    9. Sia le pazienti di sesso femminile sia le partner donne dei pazienti di sesso maschile potenzialmente fertili devono essere disposte a non iniziare una gravidanza per tutta la durata dello studio (30 giorni dopo l'ultima dose del farmaco in studio).
    E.4Principal 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
    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 second/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.
    1. Gravidanza, allattamento o indisponibilità ad adottare misure anticoncezionali durante la partecipazione allo studio
    2. Presenza di una condizione o anormalità che, a giudizio dello sperimentatore, potrebbe compromettere la sicurezza del paziente o la qualità dei dati
    3. Soggetti con anamnesi di cancro negli ultimi 5 anni
    Ipertensione definita come pressione sistolica >160 mmHg o pressione diastolica >100 mmHg all'esame iniziale
    5. Diabete mellito non controllato in base alla valutazione dello sperimentatore
    6. Episodi assimilabili all'ictus o convulsioni verificatisi negli ultimi 6 mesi
    7. Anomalie motorie diverse da quelle correlate alla malattia mitocondriale che interferiscono con i parametri degli esiti
    8. Anamnesi o evidenza di infezione attiva da tubercolosi (TB), qualsiasi malattia concomitante con condizione infiammatoria (es. malattia infiammatoria intestinale (IBD) ecc.)
    9. Pazienti con un profilo di positività per l'epatite e/o un test positivo per il virus dell'immunodeficienza all'esame iniziale
    10. Uso regolare di steroidi, farmaci antinfiammatori non steroidei (FANS) o colchicina entro 30 giorni prima dell'esame iniziale
    11. Uso cronico di metformina
    12. Uso di olio di pesce / integratori di acidi grassi omega-3
    13. Consumo di più di 9 bicchieri standard di alcol a settimana e/o più di 3 bicchieri standard di alcol per volta
    14. Test positivo per droghe e alcol (inclusi oppiacei, metadone, cocaina, anfetamine [compresa l'ecstasy], cannabinoidi, barbiturici, benzodiazepine)
    15. Qualsiasi malattia epatica significativa (definita come presenza di almeno una delle seguenti condizioni: AST, ALT, GGT, bilirubina totale o fosfatasi alcalina >3 x limite superiore normale)
    16. Assunzione di una terapia sperimentale o di una terapia approvata per uso sperimentale entro 30 giorni o 5 emivite prima dell'esame iniziale (a seconda di quale sia il periodo più lungo)
    17. Aver ricevuto qualsiasi vaccino (inclusa la seconda dose/o booster del vaccino per COVD-19) nelle due settimane precedenti la visita 1
    18. Le donne in età fertile (non sottoposte a sterilizzazione chirurgica o in post-menopausa da almeno 1 anno) sono escluse dalla partecipazione allo studio a meno che non accettino di usare misure contraccettive adeguate come descritto nell'Allegato 11.4
    19. I pazienti di sesso maschile (inclusi i soggetti sterilizzati) e le cui partner sono in età fertile devono accettare di usare misure contraccettive maschili (preservativi) durante il periodo compreso tra la firma del modulo di consenso informato e 30 giorni dopo l'ultima dose del farmaco in studio. Devono accettare di informare immediatamente lo sperimentatore se la partner rimane incinta durante lo studio.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint:
    • Number of patients (responder rate) with a between phase difference in GDF-15 of at least 20% decrease.
    Primary Safety Endpoints:
    • Incidence, severity, seriousness, reported causality, and duration of TEAEs, clinically significant changes in safety laboratory, vital signs, and 12-lead ECG
    Endpoint primario di efficacia:
    • Numero di pazienti (tasso di risposta) con una differenza di GDF-15 tra le fasi corrispondente a una riduzione di almeno il 20%.
    Endpoint primari di sicurezza:
    • Incidenza, entità, gravità, causalità riferita e durata degli eventi avversi emergenti dal trattamento, variazioni clinicamente significative nei test di laboratorio sulla sicurezza, nei parametri vitali e nell'ECG a 12 derivazioni
    E.5.1.1Timepoint(s) of evaluation of this end point
    Visit 1 Screening
    Visit 2 Baseline/Start of Treatment
    Visit 3
    Visit 4 End of Treatment
    Visit 5 Follow-up Visit
    Visita 1 Screening
    Visita 2 Baseline/inizio del trattamento
    Visita 3
    Visita 4 Fine del trattamento
    Visita 5 Visita di follow-up
    E.5.2Secondary 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.
    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).; Endpoint secondari di efficacia:
    • GDF-15 durante le fasi di valutazione, variazione del GDF-15 durante le fasi di valutazione e confronto del GDF-15 tra le fasi di valutazione.
    Endpoint farmacocinetici secondari:
    • Concentrazioni plasmatiche minime (Ctrough) di OMT-28 dalla visita 2 alla visita 5.
    • Concentrazione plasmatica di OMT-28 approssimativamente a Cmax e in un altro momento dopo la singola dose (visita 2) e allo stato stazionario (visita 4).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Visit 1 Screening
    Visit 2 Baseline/Start of Treatment
    Visit 3
    Visit 4 End of Treatment
    Visit 5 Follow-up Visit
    Visita 1 Screening
    Visita 2 Baseline/inizio del trattamento
    Visita 3
    Visita 4 Fine del trattamento
    Visita 5 Visita di follow-up
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 22
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    At the end of the clinical trial, participants will not continue to receive the study drug and will be followed up and treated according to local clinical practice.
    Al termine della sperimentazione clinica, i partecipanti non continueranno a ricevere il farmaco in studio e saranno seguiti e trattati in accordo alla pratica clinica locale.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2023-04-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-01-18
    P. End of Trial
    P.End of Trial StatusOngoing
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