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    Summary
    EudraCT Number:2021-003471-34
    Sponsor's Protocol Code Number:REN001-202
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-09-13
    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 number2021-003471-34
    A.3Full title of the trial
    An open-label, multi-centre study to evaluate the long-term safety and tolerability of REN001 In subjects with Primary Mitochondrial Myopathy (PMM)
    Studio in aperto, multicentrico per valutare la sicurezza e la tollerabilità a lungo termine di REN001 in soggetti con miopatia mitocondriale primaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An unblinded , multi-centre study to determine the long term safety and tolerability of the study drug REN001 in Primary Mitochondrial Myopathy (PMM) subjects.
    Studio in aperto, multicentrico per valutare la sicurezza e la tollerabilità a lungo termine di REN001 in soggetti con miopatia mitocondriale primaria
    A.3.2Name or abbreviated title of the trial where available
    Open label study of REN001 in PMM subjects
    Studio in aperto di REN001 in soggetti con PMM
    A.4.1Sponsor's protocol code numberREN001-202
    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 SponsorReneo Pharma LTD
    B.1.3.4CountryUnited Kingdom
    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 supportReneo Pharma Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationReneo Pharma Ltd
    B.5.2Functional name of contact pointLynn Purkins
    B.5.3 Address:
    B.5.3.1Street AddressInnovation House, Discovery Park, Ramsgate Road
    B.5.3.2Town/ citySandwich, Kent
    B.5.3.3Post codeCT13 9FF
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441304809360
    B.5.5Fax number+441304809360
    B.5.6E-maillpurkins@reneopharma.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/20/2311
    D.3 Description of the IMP
    D.3.1Product nameREN001
    D.3.2Product code [REN001]
    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.3.9.2Current sponsor codeREN001
    D.3.9.3Other descriptive nameSodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop-1- ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 Myopathy
    Miopatia mitocondriale primaria
    E.1.1.1Medical condition in easily understood language
    Group of disorders associated with changes in genetic material found within the DNA of mitochondria (mtDNA) or in genes outside the mitochondria (nuclear DNA), mainly affecting the skeletal muscle
    Gruppo di disturbi associati a cambiamenti nel materiale genetico del DNA dei mitocondri (mtDNA) o nei geni fuori dai mitocondri (DNA nucleare), che colpiscono principalmente il muscolo scheletrico
    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 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
    To evaluate long-term safety and tolerability of REN001 in subjects with PMM.
    Valutare la sicurezza e la tollerabilità a lungo termine di REN001 in soggetti affetti da miopatia mitocondriale primaria
    E.2.2Secondary objectives of the trial
    To assess patients with PMM who are receiving long-term treatment with REN001 in terms of PMM associated symptoms, exercise endurance, quality of life (QoL) and work productivity.
    Valutare i pazienti affetti da PMM che stanno ricevendo un trattamento a lungo termine con REN001 in termini di sintomi associati alla PMM, resistenza all'esercizio, qualità della vita (QoL) e produttività lavorativa.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completed treatment in the parent (STRIDE) study or was participating in Study REN001-101 when the study was stopped due to the COVID-19 pandemic, and in the opinion of the Investigator and the Sponsor have been compliant with the study requirements.
    2. Have PMM which continues to be primarily characterized by exercise intolerance or active muscle pain.
    3. Willing and able to swallow gelatin capsules.
    4. Concomitant medications (including supplements) intended for the treatment of PMM or other co-morbidities likely to remain stable throughout participation in the study where clinically possible.
    5. Signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
    6. Females should be either of non-child-bearing potential or must agree to use highly effective methods of contraception from baseline through to approximately 30 days after last dose of study drug. Males with partners who are WOCBP must also use contraception from baseline through to 14 weeks after last dose of study drug.
    1. Soggetti che hanno completato il trattamento nello studio STRIDE o che partecipavano allo Studio REN001-101 quando il suddetto studio è stato sospeso a causa della pandemia di COVID-19, e che si siano conformati ai requisiti dello studio a giudizio dello sperimentatore e del promotore.
    2. Soggetti affetti da PMM tuttora caratterizzata principalmente da intolleranza all’esercizio o da dolore muscolare attivo.
    3. Soggetti disponibili e in grado di deglutire le capsule di gelatina.
    4. Soggetti le cui farmacoterapie concomitanti (compresi gli integratori) per il trattamento della PMM o di altre comorbilità si manterranno probabilmente stabili durante l’intero periodo di partecipazione allo studio, ove clinicamente possibile.
    5. Rilascio del documento di consenso informato firmato e datato attestante che il soggetto è stato informato di tutti gli aspetti pertinenti dello studio.
    6. Le donne devono essere non in età fertile, o disposte a utilizzare metodi contraccettivi altamente efficaci dal basale fino a circa 30 giorni dopo l’ultima dose del farmaco oggetto di studio. Anche gli uomini con partner in età fertile devono impegnarsi a utilizzare contraccezione dal basale fino a 14 settimane dopo l’ultima dose del farmaco oggetto di studio.
    E.4Principal exclusion criteria
    1. Anticipated to need a PPAR agonist other than REN001 during the study.
    2. Anticipated to need drugs during the study with a narrow therapeutic index and Breast Cancer Resistant Protein (BCRP) mediated absorption, distribution, metabolism and excretion (ADME).
    3. Intent to donate blood, or blood components during the study or within one month after completion of the study.
    4. Current drug dependency. Use of opiates/cannabis for medical reasons is acceptable with prescription evidence or at the Investigator’s discretion.
    5. Current alcohol dependency.
    6. Any medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or interfere with the interpretation of study results and, in the judgment of the Investigator and Medical Monitor, would make the subject inappropriate for entry into this study. 7. Pregnant or nursing females.
    1. Soggetti con fabbisogno previsto, durante lo studio, di un agonista del recettore attivati di proliferatori perossisomiali (peroxisome proliferator-activated receptor, PPAR) diverso da REN001.
    2. Soggetti con fabbisogno previsto, nel corso dello studio, di farmaci con ristretto indice terapeutico e con assorbimento, distribuzione, metabolismo ed escrezione (Absorption, Distribution, Metabolism and Excretion, ADME) mediati dalla proteina resistente del carcinoma della mammella (Breast Cancer Resistant Protein, BCRP).
    3. Soggetti che intendono donare sangue o emocomponenti nel corso dello studio oppure entro un mese dopo il completamento dello studio.
    4. Soggetti attualmente tossicodipendenti. L’uso di oppiacei/cannabis per motivi medici è accettabile se comprovato da prescrizione medica o a discrezione dello sperimentatore.
    5. Soggetti attualmente alcoldipendenti.
    6. Qualunque disturbo medico o psichiatrico o condizione di laboratorio che possa accrescere il rischio legato alla partecipazione allo studio oppure interferire con l'interpretazione dei risultati dello studio e che, a giudizio dello sperimentatore e del Responsabile del monitoraggio medico, renderebbe il soggetto inidoneo alla partecipazione a questo studio
    7. Donne in gravidanza o che allattano con latte materno.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability of REN001 as assessed by:
    • Number and severity of adverse events (AE)
    • Number of AEs leading to study drug discontinuation
    • Number of serious adverse events (SAEs)
    • Number of adverse events of special interest (AESIs)
    • Number of AEs leading to death
    Sicurezza e tollerabilità di REN001 misurate tramite:
    • Numero e gravità degli eventi avversi (Adverse Events, AE)
    • Numero di AE che portano all’interruzione del farmaco in studio
    • Numero di eventi avversi seri (Serious Adverse Events, SAE)
    • Numero di eventi avversi di interesse speciale (Adverse Events of Special Interest, AESI)
    • Numero di AE che portano al decesso
    E.5.1.1Timepoint(s) of evaluation of this end point
    throughout the study
    durante tutto lo studio
    E.5.2Secondary end point(s)
    Absolute values, changes from baseline, and incidence of potentially clinically significant changes in:
    •Laboratory safety tests
    •Electrocardiograms (ECG)
    •Supine vital signs
    •Eye assessments

    Absolute values and changes from baseline in:
    •Distance walked during the 12-Minute Walk Test (12MWT)
    •Modified Fatigue Impact Scale (MFIS) total scores and sub-scale scores
    •Patient Global Impression of Severity (PGIS) scores for fatigue and muscle symptoms
    •Brief Pain Inventory (BPI) pain severity and pain interference scores
    •Patient Reported Outcomes Measurement Informal System (PROMIS) Short Form-Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue 13a scores
    •36 Item Short Form Health Survey (SF-36) domain scores (7-day recall)
    •Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) scores
    •PGIC (muscle and fatigue symptoms) scores
    Valori assoluti, variazioni rispetto al basale e incidenza di cambiamenti di potenziale significatività clinica in quanto segue:
    • Analisi di laboratorio per la sicurezza
    • Elettrocardiogrammi (ECG)
    • Parametri vitali in posizione supina
    • Valutazioni oculistiche

    Valori assoluti e variazioni rispetto al basale in quanto segue:
    • Distanza percorsa nel test del cammino di 12 minuti (12MWT)
    • Punteggi totali e punteggi delle sottoscale nella Scala modificata dell’impatto dell’ affaticamento (Modified Fatigue Impact Scale, MFIS)
    • Punteggi relativi all’ affaticamento e ai sintomi muscolari nella scala dell’Impressione globale della gravità da parte del paziente (Patient Global Impression of Severity, PGIS)
    • Punteggi relativi alla gravità e interferenza del dolore nel Breve inventario del dolore (Brief Pain Inventory, BPI).
    • Punteggi nella scala Sistema informativo per la misurazione degli esiti riferiti dal paziente (Patient Reported Outcomes Measurement Information System, PROMIS) Modulo breve -Valutazione funzionale della terapia delle malattie croniche-afffaticamento (Functional Assessment of Chronic Illness Therapy-Fatigue, FACIT-F) 13a
    • Punteggi dei domini del Questionario breve sulla condizione di salute a 36 voci (36 Item Short Form Health Survey, SF-36) (relativo ai precedenti 7 giorni)
    • Punteggi nel Questionario sulla compromissione della produttività lavorativa e delle attività: specifico problema di salute (Work Productivity and Activity Impairment Questionnaire:Specific Health Problem, WPAI:SHP)
    • Punteggi PGIC (sintomi muscolari e di affaticamento).
    E.5.2.1Timepoint(s) of evaluation of this end point
    throughout the study
    durante tutto lo studio
    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 No
    E.6.6Pharmacokinetic Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    Change from baseline to end of treatment in bone markers and, where available, bone mineral density
    Cambiamenti dal basale fino al termine del trattamento nei marcatori ossei e, ove disponibile, nella densità minerale ossea.
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    New Zealand
    Australia
    Canada
    Belgium
    Denmark
    France
    Germany
    Hungary
    Italy
    Spain
    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 years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days1
    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: 75
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 55
    F.4.2.2In the whole clinical trial 80
    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 study the subject’s care will revert to the arrangement in place prior to the start of the clinical trial. This will usually be the subject’s treating physician at the hospital/clinic
    Al termine dello studio le cure del soggetto torneranno alle disposizioni in vigore prima dell'inizio della sperimentazione clinica. Di solito si tratta del medico curante del soggetto presso l'ospedale/clinica.
    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 Decision2022-04-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-28
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2023-12-14
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