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    Summary
    EudraCT Number:2019-001619-21
    Sponsor's Protocol Code Number:OPTIMAB
    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:2019-06-11
    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 number2019-001619-21
    A.3Full title of the trial
    PILOT STUDY FOR THE OPTIMITZATION OF THE MAINTENANCE DOSE OF ECULIZUMAB ACCORDING TO THE WEIGHT IN ADULT PATIENTS WITH ATYPIC HEMOLYTIC UREMIC SYNDROME
    ESTUDIO PILOTO PARA LA OPTIMITZACIÓN DE LA DOSIS DE MANTENIMIENTO DE ECULIZUMAB SEGÚN EL PESO EN PACIENTES ADULTOS CON SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    OPTIMIZATION OF MAINTENANCE DOSE WITH ECULIZUMAB ACCORDING TO WEIGHT IN PATIENTS WITH ATYPIC HAEMOLITHIC URINE MUSCLE SYNDROME
    OPTIMIZACIÓN DE LA DOSIS DE MANTENIMIENTO CON ECULIZUMAB SEGÚN EL PESO EN PACIENTES CON SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO
    A.3.2Name or abbreviated title of the trial where available
    OPTIMAB
    OPTIMAB
    A.4.1Sponsor's protocol code numberOPTIMAB
    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 SponsorHOSPITAL UNIVERSITARI DE BELLVITGE- IDIBELL
    B.1.3.4CountrySpain
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportHOSPITAL UNIVERSITARI DE BELLVITGE-IDIBELL
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHOSPITAL UNIVERSITARI DE BELLVITGE-IDIBELL
    B.5.2Functional name of contact pointCAROLINA POLO
    B.5.3 Address:
    B.5.3.1Street AddressFEIXA LLARGA, S/N
    B.5.3.2Town/ cityL'HOSPITALET DE LLOBREGAT, BARCELONA
    B.5.3.3Post code08907
    B.5.3.4CountrySpain
    B.5.4Telephone number+34932607385
    B.5.6E-mailcpolo@idibell.cat
    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 Yes
    D.2.1.1.1Trade name SOLIRIS
    D.2.1.1.2Name of the Marketing Authorisation holderAlexion Europe SAS
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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.3.4Pharmaceutical form 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 INNECULIZUMAB
    D.3.9.1CAS number 219685-50-4
    D.3.9.4EV Substance CodeSUB25187
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1200
    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 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
    ATYPICAL HEMOLYTIC UREMIC SYNDROME
    SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO
    E.1.1.1Medical condition in easily understood language
    ATYPICAL HEMOLYTIC UREMIC SYNDROME
    SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10079841
    E.1.2Term Atypical hemolytic uremic syndrome
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To explore the safety of dose adjustment of Eculizumab according to body weight in patients with aHUS in remission and in the maintenance phase.
    Explorar la seguridad del ajuste de dosis de Eculizumab según peso corporal en pacientes con SHUa en remisión y en fase de mantenimiento.
    E.2.2Secondary objectives of the trial
    Analyze the estimated glomerular filtration variation.
    Study pharmacokinetic and pharmacodynamic parameters before and after treatment modification.
    Study changes in the quality of life of patients.
    Analizar la variación del filtrado glomerular estimado.
    Estudiar parámetros farmacocinéticos y farmacodinámicos antes y después de la modificación del tratamiento.
    Estudiar los cambios en la calidad de vida de los pacientes.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects must be 18 years of age or older and may be of both sexes and of any race.
    2. Subjects must have a diagnosis of aHUS documented in their clinical history, be receiving treatment with Eculizumab and be in remission of the disease for at least 12 months.
    3. The subjects must present a glomerular filtration according to CKD EPI greater than 30 ml / min at the time of inclusion.
    4. Subjects must be willing to give written informed consent for the trial and be able to do so. If a subject can not give their informed consent in writing independently, their legal representative can do so instead.
    5. Women of childbearing age (WOCBP) must perform a pregnancy test at the time of inclusion and accept the use of a medically acceptable method of contraception during the selection period and while receiving the medication specified in the protocol. Any woman who is physiologically capable of becoming pregnant, from menarche to postmenopausal, unless she is permanently sterile, is considered a woman of childbearing age. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal condition is defined as the absence of menstruation for 12 months without an alternative medical cause. A high level of follicle stimulating hormone (FSH) in the postmenopausal range can be used to confirm a postmenopausal state in women who do not use hormonal contraceptives or hormone replacement therapy. However, in the absence of 12 months of amenorrhea, a single measurement of FSH is insufficient.
    Only women of childbearing age who sign the contraceptive methods recommended by the Clinical Trial Facilitation Group (CTFG) can participate as highly effective contraceptive methods, that is, with a failure rate of less than 1% per year when used consistently and correct:
    • Combined hormonal contraception (containing estrogen and progestogen) associated with the inhibition of ovulation (oral, intravaginal or transdermal).
    • Progestin-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable)
    • Intrauterine device (IUD)
    • Intrauterine hormonal release system (SIU)
    • Bilateral tubal occlusion
    • Vasectomized partner (provided that the partner is the only sexual partner of the participant in the WOCBP trial and that the vasectomized partner has received a medical evaluation of the surgical success)
    • Sexual abstinence (defined as abstaining from sex during the entire period of risk associated with study treatments)
    1. Los sujetos deberán tener 18 años o más y podrán ser de ambos sexos y de cualquier raza.
    2. Los sujetos deberán tener un diagnóstico de SHUa documentado en su historia clínica, estar recibiendo tratamiento con Eculizumab y estar en remisión de la enfermedad desde al menos 12 meses.
    3. Los sujetos deberán presentar un Filtrado glomerular según CKD EPI superior a 30 ml/min en el momento de la inclusión.
    4. Los sujetos deberán estar dispuestos a otorgar su consentimiento informado por escrito para el ensayo y ser capaces de hacerlo. Si un sujeto no puede otorgar su consentimiento informado por escrito de forma independiente, podrá hacerlo su representante legal en su lugar.
    5. Las mujeres en edad fértil (WOCBP) deberán realizar un test de embarazo en el momento de la inclusión y aceptar el uso de un método anticonceptivo médicamente aceptable durante el periodo de selección y mientras reciban la medicación especificada en el protocolo. Se considera mujer en edad fértil toda aquella mujer fisiológicamente capaz de quedarse embarazada, desde la menarquia hasta convertirse en posmenopáusica, a menos que sea permanente estéril. Los métodos de esterilización permanentes incluyen histerectomía, salpingectomía bilateral y ooforectomía bilateral. Un estado posmenopáusico se define como ausencia de menstruación durante 12 meses sin una causa médica alternativa. Un nivel alto de hormona estimulante del folículo (FSH) en el rango posmenopáusico puede ser utilizado para confirmar un estado postmenopáusico en mujeres que no usan anticonceptivos hormonales o terapia de reemplazo hormonal. Sin embargo, en ausencia de 12 meses de amenorrea, una sola medición de FSH es insuficiente.
    Sólo podrán participar mujeres en edad fértil que signa los métodos anticonceptivos recomendados por el Clinical Trial Facilitation Group (CTFG) como métodos anticonceptivos altamente efectivos, es decir, con una tasa de fracaso de menos del 1% por año cuando se usa de manera consistente y correcta:
    • Anticoncepción hormonal combinada (que contiene estrógeno y progestágeno) asociada a la inhibición de la ovulación (oral, intravaginal o transdérmica).
    • Anticoncepción hormonal de progestágeno solo asociada con la inhibición de la ovulación (oral, inyectable o implantable)
    • Dispositivo intrauterino (DIU)
    • Sistema de liberación hormonal intrauterino (SIU)
    • Oclusión tubárica bilateral
    • Pareja vasectomizada (siempre que la pareja sea la única pareja sexual del participante en el ensayo WOCBP y que la pareja vasectomizada ha recibido una evaluación médica del éxito quirúrgico)
    • Abstinencia sexual (definida como abstenerse de tener relaciones sexuales durante todo el periodo de riesgo asociado a los tratamientos de estudio)
    E.4Principal exclusion criteria
    1. Subjects presenting a glomerular filtration according to CKD EPI less than 30 ml / min.
    2. Body weight greater than 120Kg.
    3. Pregnant women.
    4. Women in breastfeeding period.
    5. Institutionalized patients.
    1. Sujetos que presenten un Filtrado glomerular según CKD EPI inferior a 30 ml/min.
    2. Peso corporal superior a 120Kg.
    3. Mujeres embarazadas.
    4. Mujeres en periodo de lactancia.
    5. Pacientes institucionalizados.
    E.5 End points
    E.5.1Primary end point(s)
    Recurrence rate of the underlying disease (aHUS) due to lack of efficacy of the treatment with Eculizumab.
    Tasa de recurrencia de la enfermedad de base (SHUa) por falta de eficacia del tratamiento con Eculizumab.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At each study visit (every 15 days during the first 3 months and subsequently quarterly until 12 months from the start of the Study)
    En cada visita del estudio (cada 15 días durante los 3 primeros meses y posteriormente trimestrales hasta los 12 meses desde el inicio del Estudio)
    E.5.2Secondary end point(s)
    - Safety variables:
    • Renal function (serum creatinine and glomerular filtration rate estimated by the CKD-EPI formula), complete blood count and biochemistry with liver profile, 24h urine profile to assess the presence of proteinuria, urinary sediment and fundamental electrolytes (every 15 days the first 3 months and then quarterly).
    • Proportion of patients discontinuing treatment with Eculizumab due to adverse effects associated with the drug.

    - Pharmacokinetic variables: percentage of patients achieving a Cmin of Eculizumab between 35 and 50 mcg / dL and time until reaching this value of Cmin (by determining the concentration of free Eculizumab in serum).
    - Pharmacodynamic variables:
    • levels of complement activation (complement activity and membrane attack complex). At baseline and at 90, 180 and 360 days of follow-up.
    • Complement system blockade (complement activity, hemolytic capacity CH50 and serum depleted C5). At baseline and at 90, 180 and 360 days of follow-up.

    - Quality of Life of the Patient through the questionnaires SF36 and EQ-5D at baseline, at 180 days and at 360 days of follow-up.
    - Variables de seguridad:
    • Función renal (creatinina sérica y filtrado glomerular estimado por fórmula de CKD-EPI), hemograma completo y bioquímica con perfil hepático, perfil de orina de 24h para valorar la presencia de proteinuria, sedimento urinario y electrolitos fundamentales (cada 15 días los 3 primeros meses y posteriormente de forma trimestral).
    • Proporción de pacientes que discontinúan el tratamiento con Eculizumab debido a efectos adversos asociados al fármaco.

    - Variables farmacocinéticas: porcentaje de pacientes que alcanzan una Cmin de Eculizumab entre 35 y 50 mcg/dL y tiempo hasta alcanzar este valor de Cmin (mediante la determinación de la concentración de Eculizumab libre en suero).
    - Variables Farmacodiámicas:
    • niveles de activación del complemento (actividad del complemento y del complejo de ataque de la membrana). En el punto basal y a los 90, 180 y 360 días de seguimiento.
    • bloqueo del sistema de complemento (actividad del complemento, capacidad hemolítica CH50 y suero deplecionado C5). En el punto basal y a los 90, 180 y 360 días de seguimiento.

    - Calidad de Vida del Paciente mediante los cuestionarios SF36 y EQ-5D en el punto basal, a los 180 días y a los 360 días de seguimiento.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 90, 180 and 360 days of follow-up.
    A los 90, 180 y 360 días de seguimiento.
    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 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) 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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 concerned6
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months18
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 state10
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    NONE
    NINGUNO
    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 Decision2019-10-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-09-26
    P. End of Trial
    P.End of Trial StatusOngoing
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