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    Summary
    EudraCT Number:2015-000371-26
    Sponsor's Protocol Code Number:DFI12712
    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:2015-10-05
    Trial results View 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 number2015-000371-26
    A.3Full title of the trial
    A phase 2/3, multicenter, randomized, double-blinded, placebo-controlled, repeatdose, dose-comparison study to evaluate the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency
    Estudio en fase II/III, multicéntrico, aleatorizado, doble ciego, controlado con placebo, con dosis repetidas y de comparación de dosis para evaluar la eficacia, seguridad, farmacodinámica y farmacocinética de olipudasa alfa en pacientes con deficiencia de esfingomielinasa ácida
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients with Acid Sphingomyelinase Deficiency
    Eficacia, seguridad, farmacodinámica y farmacocinética de olipudasa alfa en pacientes con deficiencia de esfingomielinasa ácida
    A.3.2Name or abbreviated title of the trial where available
    ASCEND
    ASCEND
    A.4.1Sponsor's protocol code numberDFI12712
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02004691
    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 SponsorGenzyme Corporation
    B.1.3.4CountryUnited States
    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 supportGenzyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationsanofi-aventis, s.a.
    B.5.2Functional name of contact pointUnidad Estudios Clínicos
    B.5.3 Address:
    B.5.3.1Street Addressc/ Josep Pla nº2, 4ª planta
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08019
    B.5.3.4CountrySpain
    B.5.4Telephone number93 485 94 00
    B.5.6E-mailES-unidadestudiosclinicos@sanofi.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/3/01/056
    D.3 Description of the IMP
    D.3.1Product nameOlipudase alfa
    D.3.2Product code GZ402665
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    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 INNOlipudase alfa
    D.3.9.1CAS number 927883-84-9
    D.3.9.2Current sponsor codeGZ402665
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
    D.3.9.4EV Substance CodeSUB75088
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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) Yes
    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 Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)
    Pacientes con deficiencia de esfingomielinasa ácida (Enfermedad de Niemann-Pick Tipo B)
    E.1.1.1Medical condition in easily understood language
    Patients with Niemann-Pick Type B disease
    Pacientes con Enfermedad de Niemann-Pick Tipo B
    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 18.0
    E.1.2Level LLT
    E.1.2Classification code 10041515
    E.1.2Term Sphingomyelin lipidosis
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this phase 2/3 study is to evaluate the efficacy of different doses of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult patients with acid sphingomyelinase deficiency (ASMD) by assessing separately, changes in spleen volume as measured by abdominal magnetic resonance imaging (MRI) and infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide.
    El objetivo principal de este estudio en fase II/III es evaluar la eficacia de diferentes dosis de olipudasa alfa (esfingomielinasa ácida humana recombinante) administrada por vía intravenosa una vez cada 2 semanas durante un total de 52 semanas a pacientes adultos con deficiencia de esfingomielinasa ácida (acid sphingomyelinase deficiency, ASMD) mediante la evaluación por separado de cambios en 1) el volumen del bazo medido por RM abdominal y 2) enfermedad pulmonar infiltrativa medida por la prueba de la función pulmonar, capacidad de difusión pulmonar del monóxido de carbono (diffusing capacity of the lung for carbon monoxide, DLCO).
    E.2.2Secondary objectives of the trial
    To confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks.
    To characterize the effect of olipudase alfa on liver volume after 52 weeks of study drug administration.
    To characterize the effect of olipudase alfa on the splenomegaly-related symptom score after 52 weeks of study drug administration
    To characterize the effect of olipudase alfa on platelet count after 52 weeks of study drug administration.
    To characterize the effect of olipudase alfa after 52 weeks of study drug administration on a symptom-based composite score composed of 4 symptom domains (pain, fatigue, dyspnea, and abdominal complaints).
    Confirmar la seguridad de olipudasa alfa administrada por vía intravenosa una vez cada 2 semanas durante un total de 52 semanas.
    Caracterizar el efecto de olipudasa alfa a partir del volumen del hígado tras 52 semanas de administración del fármaco del estudio.
    Caracterizar el efecto de olipudasa alfa a partir de la puntuación de los síntomas relacionados con la esplenomegalia obtenida a partir de un Formulario de Evaluación de Síntomas de la Mielofibrosis modificado tras 52 semanas de administración del fármaco del estudio.
    Caracterizar el efecto de olipudasa alfa a partir del recuento de plaquetas tras 52 semanas de administración del fármaco del estudio.
    Caracterizar el efecto de olipudasa alfa tras 52 semanas de administración del fármaco del estudio a partir de una puntuación compuesta basada en los síntomas procedente de un Diario-e que consta de 4 dominios de síntomas (dolor, fatiga, disnea y molestias abdominales).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The patient is willing and able to provide signed written informed consent.
    The patient is male or female aged 18 years or older.
    The patient has documented deficiency of acid sphingomyelinase as measured in peripheral leukocytes, cultured fibroblasts, or lymphocytes; and a clinical diagnosis consistent with Niemann-Pick disease type B (NPD B).
    The patient has diffuse capacity of the lung for carbon monoxide ?70% of the predicted normal value.
    The patient has a spleen volume ?6 multiples of normal (MN) measured by MRI; patients who have had partial splenectomy will be allowed if the procedure was performed ?1 year before screening/baseline and the residual spleen volume is ?6 MN.
    Female patients of childbearing potential must have a negative serum pregnancy test for beta-human chorionic gonadotropin (?-HCG).
    Female patients of childbearing potential and male patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception.
    El paciente está dispuesto y capacitado para proporcionar un consentimiento informado por escrito.
    El paciente es hombre o mujer de 18 años o mayor.
    El paciente tiene deficiencia de esfingomielinasa ácida documentada medida por leucocitos periféricos, fibroblastos en cultivo o linfocitos, además de diagnóstico clínico coherente con la enfermedad de Niemann-Pick (ENP) tipo B.
    El paciente tiene capacidad de difusión pulmonar del monóxido de carbono (DLCO) 70 % del valor normal previsto.
    El paciente tiene un volumen del bazo 6 múltiplos de lo normal (MN) medido por RM; Se aceptarán los pacientes que hayan tenido
    una esplenectomía parcial siempre que el procedimiento se haya realizado 1 año antes del periodo de selección/basal y el volumen residual del bazo sea 6 MN.
    Las pacientes en edad fértil deben obtener un resultado negativo en la prueba de embarazo en suero para la gonadotropina coriónica humana-beta (beta-human chorionic gonadotropin, -HCG).
    Las pacientes en edad fértil y los pacientes varones deben comprometerse a practicar abstinencia real en consonancia con su estilo de vida preferido y habitual o a utilizar 2 métodos anticonceptivos eficaces aceptables.
    E.4Principal exclusion criteria
    The patient has received an investigational drug within 30 days before study enrollment.
    The patient has a medical condition, including significant intercurrent illness; significant cardiac disease (eg, clinically significant arrhythmia, moderate or severe pulmonary hypertension or valvular dysfunction, or <40% left ventricular ejection fraction by echocardiogram); active hepatitis B or hepatitis C, or infection with human immunodeficiency virus (HIV); cirrhosis (as determined by clinical evaluation or liver biopsy); malignancy diagnosed within the past 5 years (other than basal cell carcinoma), or any other extenuating circumstance that may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
    The patient has a platelet count <60,000/?L (based on the average of 2 samples obtained at least 12 hours but no longer than 24 hours apart).
    The patient has an international normalized ratio (INR) >1.5.
    The patient has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >250 IU/L or total bilirubin >1.5 mg/dL (except for patients with Gilbert's syndrome).
    The patient has had a major organ transplant (eg, bone marrow or liver).
    The patient is scheduled during the study for in-patient hospitalization including elective surgery and excluding the liver biopsies required per protocol.
    The patient, in the opinion of the investigator, is unable to adhere to the requirements of the study.
    The patient is unwilling or unable to abstain from the use of alcohol for 1 day before and 3 days after each study drug infusion. Testing for blood alcohol levels will not be required.
    The patient is unwilling or unable to avoid 10 days before and 3 days after the protocol scheduled liver biopsies that are required at screening/baseline and at week 52, the use of medications or herbal supplements that are potentially hepatotoxic (e.g., 3-hydroxy-3-methyl glutaryl coenzyme A reductase inhibitors, erythromycin, valproic acid, anti-depressants, kava, echinacea) and/or may cause or prolong bleeding (e.g., anti-coagulants, ibuprofen, aspirin, garlic supplements, ginkgo, ginseng).
    The patient requires medications that may decrease olipudase alfa activity (e.g., fluoxetine, chlorpromazine, tricyclic antidepressants [e.g., imipramine, or desipramine]).
    The patient requires use of invasive ventilatory support.
    The patient requires use of noninvasive ventilator support while awake for longer than 12 hours daily.
    The patient is breast-feeding.
    El paciente ha recibido un fármaco en investigación en el plazo de 30 días antes de la inclusión en el estudio.
    El paciente tiene una afección médica, como enfermedades intercurrentes significativas; cardiopatía significativa (p. ej., arritmia clínicamente significativa, hipertensión pulmonar o disfunción valvular moderada o severa, o fracción de eyección ventricular izquierda <40 % por ecocardiograma [ECO]); hepatitis B o hepatitis C activas, o infección por el virus de la inmunodeficiencia humana (VIH); cirrosis (determinada por evaluación clínica o biopsia hepática); neoplasia maligna diagnosticada en los últimos 5 años (distinta de carcinoma basocelular), o cualquier otra circunstancia atenuante que puede repercutir significativamente al cumplimiento del estudio, incluidas todas las evaluaciones y actividades de seguimiento prescritas.
    El paciente tiene un recuento de plaquetas <60 x 103/l (basado en la media de 2 muestras obtenidas en un intervalo mínimo de 12 horas y máximo de 24 horas).
    El paciente tiene un índice internacional normalizado (international normalized ratio, INR) >1,5.
    El paciente tiene niveles de alanina aminotransferasa (ALT) o aspartato aminotransferasa (AST) >250 UI/l o bilirrubina total >1,5 mg/dl (excepto pacientes con síndrome de Gilbert).
    El paciente se ha sometido a un trasplante de órgano principal
    (p. ej., médula o hígado).
    El paciente tiene prevista una hospitalización, incluida la cirugía programada, pero excluidas las biopsias hepáticas requeridas por el protocolo, durante el tiempo que dure el estudio.
    En opinión del investigador, el paciente no es capaz de cumplir los requisitos del estudio.
    El paciente no está dispuesto o no es capaz de no beber alcohol 1 día antes y 3 días después de cada infusión del fármaco del estudio. No será necesario llevar a cabo análisis de niveles de alcohol en sangre.
    El paciente no está dispuesto o no es capaz de evitar 10 días antes y 3 días después de las biopsias hepáticas establecidas por el protocolo, que se requieren en el periodo de selección/basal y en la semana 52, el uso de medicamentos o suplementos de herboristería que son potencialmente hepatotóxicos (p. ej., inhibidores de 3-hidroxi-3-metilglutaril-coenzima A reductasa, eritromicina, ácido valproico, antidepresivos, kava, equinácea) y/o que puedan causar hemorragias (p. ej., anticoagulantes, ibuprofeno, ácido acetilsalicílico, suplementos de ajo, ginkgo, ginseng).
    El paciente necesita medicamentos que pueden disminuir la actividad de olipudasa alfa (p. ej., fluoxetina, clorpromazina, antidepresivos tricíclicos como imipramina o desipramina).
    El paciente necesita ventilación invasiva.
    El paciente necesita ventilación no invasiva cuando está despierto durante más de 12 horas al día.
    La paciente está en periodo de lactancia.
    E.5 End points
    E.5.1Primary end point(s)
    - Percentage change in spleen volume.
    - Percentage change in diffusing capacity of the lung for carbon monoxide.
    Cambio porcentual en el volumen del bazo (en MN)
    Cambio porcentual en DLCO (en % previsto de lo normal)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to Week 52
    Desde el momento basal hasta la semana 52.
    E.5.2Secondary end point(s)
    - Percentage change in liver volume
    - Change in splenomegaly-related symptom score
    - Percentage change in platelet count
    - Change in symptom based composite score composed of of 4 symptom domains (pain, fatigue, dyspnea, and abdominal complaints)
    - Number of adverse events
    _Cambio porcentual en el volumen del hígado (en MN)
    _Cambio en la puntuación de los síntomas relacionados con la esplenomegalia
    _Cambio porcentual en los recuentos de plaquetas
    _Cambio en la puntuación compuesta basada en los síntomas que consta de 4 dominios de síntomas (dolor, fatiga, disnea y molestias abdominales)
    _Número de acontecimientos adversos
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Percentage change in liver volume - Time Frame: Baseline to Week 52
    - Change in splenomegaly-related symptom score - Time Frame: Baseline to Week 52
    - Percentage change in platelet count - Time Frame: Baseline to Week 52
    - Change in symptom based composite score composed of of 4 symptom domains (pain, fatigue, dyspnea, and abdominal complaints). - Time Frame: Baseline to Week 52
    - Number of adverse events - Time Frame: Baseline to approximately 5 years
    _Cambio porcentual en el volumen del hígado (en MN): Desde el momento basal hasta la semana 52.
    _Cambio en la puntuación de los síntomas relacionados con la esplenomegalia: Desde el momento basal hasta la semana 52.
    _Cambio porcentual en los recuentos de plaquetas: Desde el momento basal hasta la semana 52.
    _Cambio en la puntuación compuesta basada en los síntomas que consta de 4 dominios de síntomas (dolor, fatiga, disnea y molestias abdominales): Desde el momento basal hasta la semana 52.
    _Número de acontecimientos adversos: Desde el momento basal hasta aproximadamente 5 años
    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 Yes
    E.6.10Pharmacogenetic Yes
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 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
    Argentina
    Australia
    Brazil
    Chile
    France
    Germany
    Israel
    Italy
    Japan
    Netherlands
    Portugal
    Spain
    Tunisia
    Turkey
    United Kingdom
    United States
    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
    Last visit of the last subject
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.1.1Number 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: 34
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 35
    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)
    As part of the trial, all patients will receive active treatment in the extension treatment period (ETP) which will last up to 4 years. Subsequently, depending on trial results and whether a marketing authorisation for olipudase alfa is granted, the sponsor will decide to either transition patients to commercial treatment or continue patients on investigational treatment or to discontinue treatment.
    Como parte del estudio, todos los pacientes recibirán tratamiento activo en el período de tratamiento de extensión (ETP), que tendrá una duración de hasta 4 años. Posteriormente, en función de los resultados del ensayo y si se concede una autorización de comercialización de alfa olipudase, el Promotor decidirá que los pacientes hagan una transición al tratamiento comercial o continuar los pacientes en tratamiento en investigación o interrumpir el tratamiento.
    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 Decision2015-12-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-10
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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