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    Summary
    EudraCT Number:2022-003359-33
    Sponsor's Protocol Code Number:MABS06
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2023-06-24
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2022-003359-33
    A.3Full title of the trial
    Assess efficacy of intra-arterial autologous myogenic stem cell therapy for m.3243A>G mutation carriers
    Analyse van effectiviteit van intra-arteriële autologe myogene stamcel therapie voor m.3243A>G-mutatiedragers
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Assess efficacy of administration of body's-own muscle stem cell therapy
    Analyse van effectiviteit toediening lichaamseigen stamcellen via bloedbaan in arm van m.3243A>G mutatiedragers.
    A.4.1Sponsor's protocol code numberMABS06
    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 SponsorMUMC
    B.1.3.4CountryNetherlands
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportInterreg EMR
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMUMC
    B.5.2Functional name of contact pointTrial coordinator
    B.5.3 Address:
    B.5.3.1Street AddressP. Debyelaan 25
    B.5.3.2Town/ cityMaastricht
    B.5.3.3Post code6229HX
    B.5.3.4CountryNetherlands
    B.5.4Telephone number31433882918
    B.5.6E-mailflorence.vantienen@maastrichtuniversity.nl
    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 namemesoangioblasts
    D.3.2Product code MABs
    D.3.4Pharmaceutical form Concentrate for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraarterial use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNmesoangioblasts
    D.3.9.2Current sponsor codeMABS06
    D.3.9.3Other descriptive nameAutologous muscle precursor cells
    D.3.9.4EV Substance CodeSUB293266
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150000000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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
    Mitochondrial myopathy
    Mitochondriële myopathie
    E.1.1.1Medical condition in easily understood language
    muscle weakness due to the m.3243A>G mutation in the mitochondrial DNA
    spierzwakte veroorzaakt door de m.3243A>G mitochondriële DNA mutatie
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10027710
    E.1.2Term Mitochondrial myopathy
    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
    Assessment of muscle strength and fatigue in treated and untreated BB muscle will enable to assess the efficacy of MABs as ATMP to induce muscle regeneration.
    Analyse van de spierkracht en vermoeidheid in de behandelde en onbehandelde biceps brachii spier voor en na ATMP toediening om de effectiviteit van autologe MABs m.b.t. spierregeneratie te analyseren.
    E.2.2Secondary objectives of the trial
    Assess muscle mass, morphology, m.3243A>G mutation load and mitochondrial respiratory capacity in muscle biopsies of the treated and the untreated BB muscle.
    Analyse van spiermassa, spiermorfologie, m.3243A>G mutatiepercentage en mitochondriele respiratie capaciteit in spierbiopten van de behandelde en onbehandelde biceps brachii spier
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Written informed consent
    - Age: 18-64 year
    - Sex: male/female
    - Patients with the m.3243A>G mutation load of 50%-80% determined in skeletal muscle or derived from age-corrected calculation of blood m.3243A>G mutation load
    Schriftelijke toestemming
    Leeftijd 18-64 jaar
    Mannen en vrouwen
    Dragers van 50-80% m.3243A>G mutatie bepaald in spier of afgeleid van leeftijd-gecorrigeerde m.3243A>G mutatiepercentage in bloed
    E.4Principal exclusion criteria
    - Use of dabigatran, apixaban, edoxaban or rivaroxaban (DOACs) as anti-coagulants
    - Have a weekly alcohol intake of ≥ 35 units (men) or ≥ 24 units (women)
    - Current history of drug abuse
    - Deficient immune system or autoimmune disease
    - Significant concurrent illness
    - Ongoing participation in other clinical trials with intervention
    - Pregnant or lactating women
    - Psychiatric or other disorders likely to impact on informed consent
    - Patients unable and/or unwilling to comply with treatment and study instructions
    - A history of strokes with signs of extra-pyramidal or pyramidal syndrome
    - Allergy for contrast fluid
    - Peripheral signs of ischemia or vasculopathy
    - Claustrophobia
    - Metal implants
    - Any other factor that in the opinion of the investigator excludes the patient from the study
    - Gebruik van dabigatran, apixaban, edoxaban of rivaroxaban (DOAC's) als anticoagulantia
    - Een wekelijkse alcoholinname van ≥ 35 eenheden (mannen) of ≥ 24 eenheden (vrouwen)
    - Huidig drugsgebruik
    - Deficiënt immuunsysteem of auto-immuunziekte
    - Significante gelijktijdige ziekte
    - Doorlopende deelname aan andere klinische onderzoeken met interventie
    - Zwangere of zogende vrouwen
    - Psychische of andere stoornissen die van invloed kunnen zijn op geïnformeerde toestemming
    - Patiënten die de behandel- en onderzoeksinstructies niet kunnen en/of willen opvolgen
    - Een voorgeschiedenis van beroertes met tekenen van extrapiramidaal of piramidaal syndroom
    - Allergie voor contrastvloeistof
    - Perifere tekenen van ischemie of vasculopathie
    - Claustrofobie
    - Metalen implantaten
    - Elke andere factor die naar de mening van de onderzoeker de patiënt uitsluit van het onderzoek
    E.5 End points
    E.5.1Primary end point(s)
    Assessment of changes in muscle strength and muscle fatigue of treated and untreated biceps brachii muscle using dynamometer muscle force measurements
    Analyseren van veranderingen in spierkracht en vermoeidheid van de behandelde en onbehandelde biceps brachii spier door middel van dynamometer krachtmetingen.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At baseline and 4-6 weeks after the third ATMP administration.
    Voor 1e toediening (baseline) en 4-6 weken na de 3e ATMP toediening
    E.5.2Secondary end point(s)
    Assess muscle mass, morphology, m.3243A>G mutation load and mitochondrial respiratory capacity in muscle biopsies treated and untreated BB muscle.
    • Muscle mass
    o MRI T3 analysis to assess changes in muscle mass in both arms at baseline and one month after last administration.
    • Muscle morphology
    o eMHC+ immunostaining to assess percentage of new / regenerating muscle fibers in muscle biopsies from both arms at baseline and one month after last administration.
    o H&E staining to assess general morphology.
    o Distribution muscle fiber types using MHC-immunostaining
    • Mitochondrial mutation load and functioning
    o Determine COX and SDH as markers of mitochondrial respiratory functioning in muscle fibers.
    o Assess changes in m.3243A>G mutation load in new/regenerating muscle fibers isolated via laser microdissection.
    Beoordeel spiermassa, morfologie, m.3243A>G-mutatiebelasting en mitochondriale ademhalingscapaciteit in met spierbiopten behandelde en onbehandelde BB-spier.
    • Spiermassa
    o MRI T3-analyse om veranderingen in massa van biceps brachii spieren van beide armen te beoordelen voor 1e toediening en 4-6 weken na de laatste toediening.
    • Spiermorfologie
    o eMHC+-immunokleuring om het percentage nieuwe/regenererende spiervezels in spierbiopten van beide armen bij baseline en één maand na de laatste toediening te bepalen.
    o H&E-kleuring om de algemene morfologie te beoordelen.
    o Verdeling spiervezeltypes met MHC-immunokleuring
    • Mitochondriele mutatiepercentage en functioneren
    o Bepaal COX en SDH als markers van mitochondriale respiratie functie in spiervezels.
    o Analyseren van veranderingen in m.3243A>G-mutatiepercentage in nieuwe/regenererende spiervezels geïsoleerd via lasermicrodissectie.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At baseline and 4-6 weeks after the third ATMP administration.
    Voor 1e toediening (baseline) en 4-6 weken na de 3e ATMP toediening
    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 Yes
    E.6.4Safety No
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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 Yes
    E.8.1.7.1Other trial design description
    open-label intra-subject gecontroleerde studie
    open-label intra-subject controlled study
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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 No
    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
    laatste bezoek laatste proefpersoon
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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) No
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2023-06-24. Yes
    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 state20
    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)
    None
    Geen
    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 Decision2023-06-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-06-12
    P. End of Trial
    P.End of Trial StatusOngoing
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