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    Summary
    EudraCT Number:2021-001379-18
    Sponsor's Protocol Code Number:QEL-001-CLN-01
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-12-20
    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 number2021-001379-18
    A.3Full title of the trial
    A single-arm, open-label, multi-centre, phase I/II study evaluating the safety and clinical activity of QEL-001, an autologous CAR T regulatory cell
    treatment targeting HLA-A2, in HLA-A2/ A28neg patients that have received an HLA-A2pos liver transplant
    Un estudio de fase I/II multicéntrico, abierto, de un solo brazo que evalúa la seguridad y actividad clínica del QEL-001, un tratamiento de células CAR-T reguladoras autólogas dirigido al HLA-A2, en pacientes cuyo HLA-A2/A28 es negativo y que recibieron un trasplante de hígado HLA-A2 positivo.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase I/II study of QEL-001 in A2-mismatch liver transplant patients
    Estudio de fase I/II del QEL-001 en pacientes de trasplante de hígado con disparidad de A2
    A.3.2Name or abbreviated title of the trial where available
    LIBERATE
    A.4.1Sponsor's protocol code numberQEL-001-CLN-01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05234190
    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 SponsorQuell Therapeutics Limited
    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 supportQuell Therapeutics Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationQuell Therapeutics Limited
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressTranslation and Innovation Hub, 84 Wood Lane
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW12 0BZ
    B.5.3.4CountryUnited Kingdom
    B.5.6E-mailQEL-001-CLN-01@quell-tx.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 nameQEL-001
    D.3.2Product code QEL-001
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot requested to date
    D.3.9.2Current sponsor codeQEL-001
    D.3.9.3Other descriptive nameAutologous T regulatory cells expressing an HLA-A2-specific chimeric antigen receptor
    D.3.9.4EV Substance CodeSUB218939
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number50 to 700
    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 No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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
    Prevention of liver transplant rejection
    Prevención de rechazo tras trasplante de hígado
    E.1.1.1Medical condition in easily understood language
    Prevention of liver transplant rejection
    Prevención de rechazo tras trasplante de hígado
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10024715
    E.1.2Term Liver transplant rejection
    E.1.2System Organ Class 10021428 - Immune system 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 the safety and tolerability of a single target dose of QEL-001
    Evaluar la seguridad y la tolerabilidad de una dosis única de QEL-001
    E.2.2Secondary objectives of the trial
    To assess the clinical activity of QEL-001 (Part 2 only)
    To assess safety-related events
    To determine absence or presence of exposure to replication-competent lentivirus (RCL)
    [Sólo parte 2] Evaluar la actividad clínica de QEL-001
    Para evaluar los acontecimientos relacionados con la seguridad
    Para determinar la ausencia o presencia de exposición a lentivirus competentes para replicación (RCL)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects (regardless of gender) must be 18 to 70 years of age inclusive, at the time of signing the informed consent.
    2. HLA A2/A28neg (including split antigens A68neg and A69neg) subjects who have received an A2pos liver transplant.
    3. Having received a liver transplant 12 months to 5 years prior to study entry with:
    a. No episodes of rejection in the previous 12 months (except early rejection taking place in the first 3 months post-transplantation.
    b. No previous episodes of rejection requiring lymphodepleting antibody therapy.
    4. Having alanine aminotransferase (ALT) <60 U/L and either alkaline phosphatase (ALP) <200 U/L or gamma-glutamyl transferase (GGT) <100 U/L.
    5. Having eGFR > or = 40 mL/min/1.73 m2 using the MDRD formula.
    6. Having an adequate bone marrow (BM) function without requiring ongoing blood product(s) or granulocyte colony-stimulating factor (GCSF) and meeting the following criteria:
    a. Absolute neutrophil count > or = 1.0 x 10e9/L
    b. Absolute lymphocyte count > or = 0.3 x 10e9/L
    c. Leucocyte count > or = 2.0 x 10e9/ L
    d. Haemoglobin > or = 80 g/L
    e. Platelet greater or equal to 100 x 10e9/L
    7. Having an Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1
    8. Able and willing to use a highly effective method of contraception (male subjects and female subjects with reproductive potential) from informed consent through and for at least 12 months.
    9. A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) at Screening, prior to conditioning with rATG and prior/post QEL-001 infusion.
    10. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
    11. Being on stable maintenance of immunosuppression for at least 12 weeks prior to study entry
    1. Los sujetos (independientemente del sexo) deben tener entre 18 y 70 años de edad, ambos inclusive, en el momento de firmar el consentimiento informado.
    2. Sujetos con HLA A2/A28neg (incluidos los antígenos divididos A68neg y A69neg) que hayan recibido un trasplante de hígado de A2pos.
    3. Haber recibido un trasplante de hígado entre 12 meses y 5 años antes de la entrada en el estudio con:
    a. Ningún episodio de rechazo en los 12 meses previos (excepto el rechazo precoz que se produce en los 3 primeros meses postrasplante).
    b. Ningún episodio previo de rechazo que requiera una terapia de anticuerpos que produzcan linfopenia.
    4. Tener alanina aminotransferasa (ALT) < 60 U/l y fosfatasa alcalina (ALP) < 200 U/l o gammaglutamil transferasa (GGT) < 100 U/l.
    5. Tener una filtración glomerular estimada (eGFR) > o = 40 ml/min/1,73 m2 según la fórmula MDRD.
    6. Tener una función adecuada de la médula ósea (BM) sin requerir producto(s) sanguíneo(s) en curso o factor estimulante de colonias de granulocitos (GCSF) y cumplir los siguientes criterios:
    a. Recuento absoluto de neutrófilos > o = 1,0 x 10e9/L
    b. Recuento absoluto de linfocitos > o = 0,3 x 10e9/L
    c. Recuento de leucocitos > o = 2,0 x 10e9/ L
    d. Hemoglobina > o = 80 g/L
    e. Plaquetas mayores o iguales a 100 x 10e9/L
    7. Tener un estado de rendimiento 0 o 1 del Eastern Cooperative Oncology Group (ECOG)
    8. Ser capaz y estar dispuesto a utilizar un método anticonceptivo altamente eficaz (sujetos masculinos y sujetos femeninos con potencial reproductivo) desde el consentimiento informado y durante al menos 12 meses
    9. Una WOCBP debe tener una prueba de embarazo altamente sensible negativa (orina o suero, según lo exijan las normativas locales) en el momento del cribado, antes del acondicionamiento con rATG y antes/después de la infusión de QEL-001
    10. Capaz de dar un consentimiento informado firmado que incluya el cumplimiento de los requisitos y restricciones enumerados en el formulario de consentimiento informado (ICF) y en este protocolo
    11. Estar en mantenimiento estable de la inmunosupresión durante al menos 12 semanas antes de la entrada en el estudio
    E.4Principal exclusion criteria
    1. Having any medical condition that, in the opinion of the principal investigator (PI), would interfere with safe completion of the trial including:
    a. Severe cardiac disease, severe respiratory disease with O2 blood saturation <92%.
    b. Any other major organ dysfunction.
    2. Having received prior non-liver solid organ or hematopoietic stem cell transplant.
    3. Have had any major surgical intervention in the last 3 months prior to study entry (such as open surgeries requiring general anaesthetic).
    4. History of autoimmune disease requiring systemic immunosuppression, systemic disease modifying agents or biologics within the last 24 months prior to study entry.
    5. History of autoimmune hepatitis, primary sclerosing cholangitis or primary biliary cholangitis.
    6. Having any evidence of recurrent hepatocellular carcinoma (HCC) following clinical assessment; for subjects with liver explant histology showing a RETREAT score greater than 0 the clinical assessment should include a thoraco-abdominal CT scan performed within 1 month prior to enrolment.
    7. History of HCC with high risk of recurrence defined as:
    a. For subjects who are <2 years post-transplant: a) RETREAT score greater than or equal to 3.
    b. For subjects who are 2-5 years post-transplant: a) RETREAT score greater than or equal to 4.
    c. Additional explant-based exclusion criteria: i) presence of diffuse HCC in explant; ii)
    presence of extrahepatic extension or macrovascular invasion; iii) diagnostics of
    cholangiocarcinoma; iv) subjects who have undergone a downstaging procedure pretransplantation.
    8. Having active primary or recurrent malignant disease or have been in remission from clinically significant malignancy for less than 5 years.
    a. Except subjects that have had a cervical carcinoma in situ that has been resected with no evidence of recurrence or metastatic disease for at least 3 years may participate in the study.
    b. Except subjects that have had basal cell or squamous epithelial skin cancers that have been completely resected with no evidence of recurrence for at least 3 years may participate in the study.
    9. Having urine protein to creatinine ratio > 25mg/mmol or > 50mg/mmol if the subject is already under dual therapy TAC/EVR.
    10. Having triglycerides > or = 3.95 mmol/L despite appropriate therapy.
    11. Having cholesterol > or = 7.8 mmol/L despite appropriate therapy.
    12. Having QTc > 450 msec for male subjects or QTc > 470 msec for female subjects or QTc > 480 msec in subjects with bundle branch block.
    13. Having any contraindications to receive rATG, EVR (notably subjects with rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption), TAC or rituximab.
    14. Receiving any other concurrent investigational agents within 3 months prior study entry.
    15. Having an active infection as defined in the study protocol.
    16. Having previous history of human immunodeficiency virus (HIV).
    17. Evidence of active or latent tuberculosis (TB). After anti-TB treatment, patients with history of active or latent TB may become eligible according to national guidelines.
    18. Recent history of reactivation of Cytomegalovirus (CMV) defined as positive DNA test within 6 months prior entry to the study.
    19. Females who are pregnant (i.e., positive blood or urine β human Chorionic Gonadotropin (beta- hCG) test) or lactating.
    20. Do not have:
    a. up-to-date vaccination status as per local immunization schedules/national guidelines (e.g., influenza and pneumococcal vaccination for >65 years old).
    b. completed vaccination against COVID-19 at least 2 weeks prior to study entry, in accordance with national guidelines. Subsequent COVID-19 ‘booster’ vaccination courses, as per national guideline, must be completed at least 2 weeks prior to study entry.
    21. Have known or suspected hypersensitivity or allergy to DMSO present in QEL-001 as excipients.
    22. Having a non-adequate bilateral venous access for leukapheresis, or not willing to undergo a central venous catheter insertion.
    23. Having contraindications to undergo a leukapheresis.
    24. Have any significant medical or social condition that, in the Investigator's opinion, may interfere with the subject’s optimal participation or compliance with the study procedure.
    25. Having history of liver cirrhosis or advanced fibrosis post-transplantation.
    26. Receiving prohibited medications that cannot be stopped at study entry.
    1. Tener cualquier condición médica que, en opinión del investigador principal, pueda interferir con la realización del ensayo con seguridad, incluyendo:
    a.Enfermedad cardíaca grave, enfermedad respiratoria grave con saturación de O2 en sangre <92%.
    b.Cualquier otra disfunción de órganos importantes.
    2. Haber recibido previamente un trasplante de un órgano sólido no hepático o de células madre hematopoyéticas.
    3. Haber tenido alguna intervención quirúrgica importante en los últimos 3 meses anteriores a la entrada en el estudio (como cirugías abiertas que requieran anestesia general).
    4. Antecedentes de enfermedades autoinmunes que requieran inmunosupresión sistémica, agentes modificadores de la enfermedad sistémica o productos biológicos en los últimos 24 meses anteriores a la entrada en el estudio.
    5. Antecedentes de hepatitis autoinmune, colangitis esclerosante primaria o colangitis biliar primaria.
    6. Tener cualquier evidencia de carcinoma hepatocelular recurrente tras la evaluación clínica; en el caso de los sujetos con histología de explante hepático que muestren una puntuación RETREAT superior a 0, la evaluación clínica debe incluir una tomografía computarizada toraco-abdominal realizada en el plazo de 1 mes antes de la inscripción.
    7. Antecedentes de HCC con alto riesgo de recurrencia definidos como:
    a.Para los sujetos cuyo transplante tienen <2 años : a)Puntuación RETREAT mayor o igual a 3.
    b.Para los sujetos cuyo transplante tiene entre 2 y 5 años: a)Puntuación de RETREAT mayor o igual a 4.
    c. Criterios de exclusión adicionales basados en el explante: i)presencia de CHC difuso en el explante; ii)presencia de extensión extrahepática o invasión macrovascular; iii)diagnóstico de colangiocarcinoma; iv)sujetos que hayan sido sometidos a un procedimiento de reducción de estadio antes del trasplante.
    8. Tener una neoplasia maligna activa primaria o recurrente o en remisión clínicamente significativa durante menos de 5 años.
    a. Excepto los sujetos que hayan tenido un carcinoma cervical in situ que haya sido resecado sin evidencia de recurrencia o enfermedad metastásica durante al menos 3 años podrán participar en el estudio.
    b. Excepto los sujetos que hayan tenido cánceres de piel de células basales o epiteliales escamosas que hayan sido completamente resecados sin evidencia de recidiva durante al menos 3 años podrán participar en el estudio.
    9. Tener una relación proteína/creatinina en orina > 25 mg/mmol o > 50mg/mmol si el sujeto ya está bajo terapia dual TAC/EVR.
    10. Tener triglicéridos > o = 3,95 mmol/L a pesar del tratamiento adecuado.
    11. Tener un colesterol > o = 7,8 mmol/L a pesar del tratamiento adecuado.
    12. Tener un QTc > 450 mseg en sujetos masculinos o un QTc > 470 mseg en sujetos femeninos o un QTc > 480 mseg en sujetos con bloqueo de rama.
    13. Tener alguna contraindicación para recibir rATG, EVR (especialmente los sujetos con problemas hereditarios raros de intolerancia a la galactosa, deficiencia total de lactasa o malabsorción de glucosa-galactosa), TAC o rituximab.
    14. Recibir cualquier otro agente de investigación concurrente en los 3 meses anteriores a la entrada en el estudio.
    15. Tener una infección activa como se define a continuación:
    a.Replicación activa de la hepatitis B o de la hepatitis C activa (HCV-ARN)
    b.Infección bacteriana, vírica o fúngica no controlada y potencialmente mortal
    16. Tener antecedentes del virus del VIH.
    17. Evidencia de tuberculosis activa o latente. Tras el tratamiento antituberculoso, los pacientes con antecedentes de tuberculosis activa o latente pueden ser elegibles según las directrices nacionales.
    18. Antecedentes recientes de reactivación del citomegalovirus definidos como prueba de ADN positiva en los 6 meses anteriores a la entrada en el estudio.
    19. Mujeres embarazadas o en periodo de lactancia.
    20. No tener:
    a. el no tener las vacunas actualizadas según los calendarios de vacunación locales/directrices nacionales .
    b. completada la vacunación contra la COVID-19 al menos 2 semanas antes de la entrada en el estudio, de acuerdo con las directrices nacionales. Los ciclos posteriores de "refuerzo" de COVID-19, según las directrices nacionales, deben completarse al menos 2 semanas antes de la entrada en el estudio.
    21. Tener hipersensibilidad o alergia conocida o sospechada al DMSO presente en el QEL-001 como excipiente.
    22. Tener un acceso venoso bilateral no adecuado para la leucaféresis, o no estar dispuesto a someterse a la inserción de un catéter venoso central.
    23. Tener contraindicaciones para someterse a una leucaféresis.
    24. Tener cualquier condición médica o social significativa que, en opinión del investigador, pueda interferir con la participación óptima del sujeto o el cumplimiento del procedimiento del estudio.
    25. Tener antecedentes de cirrosis hepática o fibrosis avanzada tras el trasplante.
    26. Recibir medicamentos prohibidos que no pueden ser suspendidos a la entrada del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence of protocol-defined Dose-Limiting Toxicities (DLTs) within 28 days post infusion.
    • Incidence and severity of Treatment Emergent Adverse Events (TEAE) including serious adverse events (SAE) according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
    • Incidencia de las toxicidades limitantes de la dosis (DLTs) definidas por el protocolo en los 28 días posteriores a la infusión.
    • Incidencia y gravedad de los acontecimientos adversos surgidos durante el tratamiento (TEAE), incluidos los acontecimientos adversos graves (SAE), de acuerdo con los Criterios terminológicos comunes para acontecimientos adversos (CTCAE) versión 5.0.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Assessed continuously throughout study.
    Evaluado continuamente a lo largo del estudio
    E.5.2Secondary end point(s)
    • Proportion of subjects that can be successfully withdrawn from IS defined as the percentage of subjects who have stable Liver Function Tests (LFTs) and are IS free for 2 months following Immunosuppression withdrawal.
    • Proportion of subjects that can be successfully withdrawn from IS defined as the percentage of subjects who have stable LFTs and are IS free for 1 year following Immunosuppression withdrawal.
    • Incidence and severity of infections from treatment to Week 82.
    • Proportion of subjects with detectable RCL assessed by polymerase chain reaction (PCR) to vesicular stomatitis virus G glycoprotein (VSV-G) protein [time frame: pre-treatment and Week 14, Week 26 and Week 54.
    • Proportion of subjects meeting criteria for operational tolerance defined as (1) successfully withdrawn from IS and remaining IS free for 1 year following Immunosuppression withdrawal; (2) having stable LFTs and (3) meeting histological criteria of operational tolerance.
    • Incidence rate composite efficacy failure from treatment to 1 year following Immunosuppression withdrawal. Composite efficacy failure endpoint is defined as AR, biopsy proven acute rejection (BPAR), reintroduction of IS or graft loss.
    • Proporción de sujetos a los que se les puede retirar con éxito la inmunosupresión, definida como el porcentaje de sujetos con pruebas de función hepática (LFT) estables y libres de inmunosupresión durante 2 meses tras la retirada de la inmunosupresión.
    • Proporción de sujetos que pueden dejar con éxito la IS definida como el porcentaje de sujetos que tienen las pruebas de función hepática estables y están libres de IS durante 1 año tras la retirada de la inmunosupresión.
    • Proporción de sujetos que cumplen los criterios de tolerancia operativa definidos como (1) que se retiren con éxito de la IS y que permanezcan libres de IS durante 1 año tras la retirada de la inmunosupresión; (2) que tengan unas pruebas de función hepática estables y (3) que cumplan los criterios histológicos de tolerancia operativa.
    • Tasa de incidencia de fracaso de la eficacia compuesta desde el tratamiento hasta 1 año después de la retirada de la inmunosupresión. El criterio de valoración combinado de fracaso de la eficacia se define como rechazo agudo (AR), rechazo agudo demostrado por biopsia (BPAR), reintroducción de IS o pérdida del injerto.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Incidence rate of composite efficacy failure will be assessed from treatment through week 82
    RCL detection is performed at Day 1, Week 14, Week 24, Week 52, and then LTFU month 6, month 12, month 24, month 36, month 48, month
    60, then yearly between years 6 to 15
    Infectious disease markers will be assessed at Screening, pre-treatment visits 3 and 4
    Se evaluará la tasa de incidencia del fracaso compuesto de la eficacia desde el tratamiento hasta la semana 82.
    La detección de RCL se realiza en el día 1, semana 14, semana 24, semana 52, y después en el LTFU mes 6, mes 12, mes 24, mes 36, mes 48, mes
    60, y luego anualmente entre los años 6 y 15
    Los marcadores de enfermedades infecciosas se evaluarán en el cribado y en las visitas previas al tratamiento 3 y 4.
    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 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) Yes
    E.7.1.1First administration to humans Yes
    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 Yes
    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.8.3 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 EEA6
    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
    Spain
    Belgium
    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
    The end of study is defined as the last subject’s visit (LPLV), which is the last visit of the LTFU which is the last subject’s Year 15 evaluation. A subject is considered to have completed the study if they have completed the last visit of the LTFU. Subjects that discontinue from the “Treatment and Primary follow-up" phase before W14 (for part 1) or before W82 (for part 2), will be enrolled into the LTFU as recommended by regulatory authority guidance and after consent has been obtained.
    Última visita del sujeto (UVUP), que es la última visita del seguimiento a largo plazo (SLP) que es la evaluación del último sujeto en el año 15. Se considera que un sujeto ha finalizado el estudio si ha completado la última visita del SLP. Los sujetos que abandonen la fase de "Tratamiento y seguimiento primario" antes de la semana 14 (parte 1) o antes de la semana 82 (parte 2), serán inscritos en el SLP según lo recomendado por la guía de la autoridad reguladora y después de haber consentido
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years16
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years16
    E.8.9.2In all countries concerned by the trial months6
    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: 32
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 42
    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)
    Subjects enrolled to the study will be subject to long-term follow for a period of up to 15 years following participation in Part 1 or Part 2 of the study. No further study intervention is anticipated following the end of the study and consequently subjects will revert to standard of care as directed by their treating physician.
    Los sujetos inscritos en el estudio serán objeto de seguimiento a largo plazo durante un período de hasta 15 años tras su participación en la Parte 1 o la Parte 2 del estudio. Una vez finalizado el estudio, no se prevé ninguna otra intervención y, por consiguiente, los sujetos volverán a seguir el tratamiento estándar que les indique el médico que les trate.
    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 Decision2023-04-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-04-13
    P. End of Trial
    P.End of Trial StatusOngoing
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