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    Summary
    EudraCT Number:2021-003471-34
    Sponsor's Protocol Code Number:REN001-202
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-11-19
    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 ConcernedSpain - AEMPS
    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)
    ESTUDIO ABIERTO Y MULTICÉNTRICO PARA EVALUAR LA SEGURIDAD Y TOLERABILIDAD A LARGO PLAZO DE REN001 EN SUJETOS CON MIOPATÍA MITOCONDRIAL PRIMARIA (PMM)
    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.
    Un estudio multicéntrico no ciego para determinar la seguridad y tolerabilidad a largo plazo del fármaco del estudio REN001 en sujetos con miopatía mitocondrial primaria (PMM).
    A.3.2Name or abbreviated title of the trial where available
    Open label study of REN001 in PMM subjects
    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.3.4Pharmaceutical 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 INNREN001
    D.3.9.2Current sponsor codeREN001
    D.3.9.3Other descriptive nameSodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)
    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 No
    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
    Primary Mitochondrial Myopathy
    Miopatía mitocondrial 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
    Trastornos asociados con cambios en el material genético que se encuentran dentro del ADN de las mitocondrias o en genes fuera de las mitocondrias, que afectan principalmente al músculo esquelético
    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.
    Evaluar la seguridad y tolerabilidad a largo plazo de REN001 en sujetos con PMM.
    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.
    Evaluar a los pacientes con PMM que reciben tratamiento a largo plazo con REN001 en términos de síntomas asociados con PMM, resistencia al ejercicio, calidad de vida (QoL) y productividad laboral.
    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. Haber completado el tratamiento en el estudio STRIDE o haber estado participando en el estudio REN001-101 antes de que se suspendiera por la pandemia de la COVID-19 y, a juicio del investigador y del promotor, haber cumplido con los requisitos del estudio.
    2. Padecer PMM caracterizada principalmente por la intolerancia al ejercicio o el dolor muscular activo.
    3. Estar dispuesto y ser capaz de tragar cápsulas de gelatina de REN001.
    4. Medicamentos concomitantes (incluidos los suplementos) destinados al tratamiento de la PMM u otras comorbilidades que probablemente se mantengan estables durante la participación en el estudio cuando sea clínicamente posible.
    5. Documento de consentimiento informado, firmado y fechado que indique que se ha informado al sujeto sobre todos los aspectos correspondientes del estudio.
    6. Las mujeres no deben estar en edad fértil o deben aceptar utilizar métodos anticonceptivos altamente eficaces desde el inicio hasta aproximadamente 30 días después de la última dosis del medicamento del estudio. Los hombres con parejas que sean mujeres en edad fértil (WOCBP, por sus siglas en inglés) también deben utilizar métodos anticonceptivos desde el inicio hasta 14 semanas después de la última dosis del medicamento del estudio.
    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. Está previsto que se necesite un agonista del receptor activado por proliferadores peroxisomales (PPAR, por sus siglas en inglés) distinto de REN001 durante el estudio.
    2. Está previsto que se necesiten fármacos durante el estudio con un índice terapéutico estrecho y una absorción, distribución, metabolismo y excreción (ADME) mediada por la proteína resistente al cáncer de mama (BCRP, por sus siglas en inglés) (Consultar la Tabla 1).
    3. Intención de donar sangre o componentes sanguíneos durante el estudio o en el plazo de un mes tras la finalización del mismo.
    4. Dependencia actualmente a alguna droga. El uso de opiáceos/cannabis por cuestiones médicas es aceptable si se demuestra con prescripción o bajo criterio del investigador.
    5. Dependencia actual del alcohol.
    6. Cualquier condición médica, psiquiátrica o analítica que pueda aumentar el riesgo asociado a la participación en el estudio o interferir con la interpretación de los resultados del estudio y que, a juicio del investigador y del supervisor clínico, haga que el sujeto no sea apto para participar en este estudio.
    7. Mujeres embarazadas o en periodo de lactancia.
    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
    Seguridad y tolerabilidad de REN001 según lo evaluado por:
    • Número y gravedad de los efectos adversos (EA)
    • Número de EA que provoca la discontinuación del medicamento del estudio
    • Número de efectos adversos graves (EAG)
    • Número de efectos adversos de interés especial (EAIE)
    • Número de EA que producen la muerte
    E.5.1.1Timepoint(s) of evaluation of this end point
    throughout the study
    a lo largo del estudio
    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
    Valores absolutos, cambios respecto al inicio del estudio e incidencia de cambios potencialmente significativos en:
    • Pruebas analíticas de seguridad
    • Electrocardiogramas (ECG)
    • Signos vitales en posición supina
    • Evaluaciones oculares

    Valores absolutos y cambios respecto al inicio del estudio en:
    • Distancia caminada durante la prueba de marcha de 12 minutos (12-Minute Walk Test, 12MWT)
    • Puntuaciones totales y subescalas de la escala de impacto del cansancio modificada (MFIS, por sus siglas en inglés)
    • Puntuaciones de la impresión global del paciente de la gravedad (PGIC, por sus siglas en inglés) para el cansancio y los síntomas musculares
    • Puntuaciones de gravedad del dolor y de interferencia del dolor en el cuestionario breve del dolor (BPI, por sus siglas en inglés)
    • Cuestionario breve sobre el sistema informal de medición de resultados proporcionados por el paciente (PROMIS, por sus siglas en inglés) Evaluación funcional de la terapia de enfermedades crónicas (FACIT, por sus siglas en inglés) Puntuaciones de cansancio 13a
    • Puntuaciones de dominio del estudio de evaluación breve de salud de 36 ítems (SF-36) (recordatorio de 7 días)
    • Cuestionario sobre el deterioro de la actividad y la productividad laboral: Puntuaciones del problema de salud específico (WPAI:SHP)
    • Puntuaciones de la impresión global del cambio del paciente (PGIC, por sus siglas en inglés) (síntomas musculares y de cansancio).
    E.5.2.1Timepoint(s) of evaluation of this end point
    throughout the study
    a lo largo del estudio
    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
    Cambio desde el inicio hasta el final del tratamiento en los marcadores óseos y, cuando esté disponible, la densidad mineral ósea
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 EEA19
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Canada
    New Zealand
    Belgium
    Denmark
    France
    Germany
    Hungary
    Italy
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    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
    última visita del último paciente
    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 days0
    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 days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    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 state30
    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 final del estudio, la atención del sujeto volverá a la disposición establecida antes del inicio del ensayo clínico. Por lo general, será el médico tratante del sujeto en el hospital/clínica.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-02-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-08
    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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