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    Summary
    EudraCT Number:2017-001987-39
    Sponsor's Protocol Code Number:CDRB436X2X02B
    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:2017-10-02
    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 number2017-001987-39
    A.3Full title of the trial
    An open label, multi-center roll-over study to assess long-term safety in patients who are ongoing or have completed a prior global Novartis or GSK sponsored Tafinlar (dabrafenib) and/or Mekinist (trametinib) study and are judged by the investigator to benefit from continued treatment
    Estudio abierto, multicéntrico, de continuación, para evaluar la seguridad a largo plazo en pacientes que están participando o han finalizado un estudio global previo de Tafinlar (dafrafenib) y/o Mekinist (trametinib), patrocinado por Novartis o por GSK, y el investigador considera que se beneficiarán de la continuación del tratamiento.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This clinical study is designed to provide continued access to patients who have previously participated in a dabrafenib and/or trametinib study and who in the opinion of the Investigator, would benefit from continued treatment
    Se trata de un estudio multicéntrico, abierto, de continuación, diseñado para proporcionar acceso continuado a pacientes que hayan participado previamente en un estudio de tratamiento con dabrafenib y/o trametinib (estudio principal) y que hayan cumplido los requisitos para el objetivo principal, y que a criterio del investigador, podrían beneficiarse de la continuación del tratamiento.
    A.4.1Sponsor's protocol code numberCDRB436X2X02B
    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 SponsorNovartis Farmacéutica, S.A.
    B.1.3.4CountrySpain
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Farmacéutica, S.A.
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressGran Vía de les Corts Catalanes, 764
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08013
    B.5.3.4CountrySpain
    B.5.4Telephone number+3493 3064464
    B.5.5Fax number+3493 3064615
    B.5.6E-maileecc.novartis@novartis.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 Yes
    D.2.1.1.1Trade name Tafinlar
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameTafinlar
    D.3.2Product code DRB436
    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 INNdabrafenib
    D.3.9.2Current sponsor codeDRB436
    D.3.9.3Other descriptive nameDABRAFENIB MESYLATE
    D.3.9.4EV Substance CodeSUB45696
    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.IMP: 2
    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 Mekinist
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameMekinist
    D.3.2Product code TMT212
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNTRAMETINIB
    D.3.9.2Current sponsor codeTMT212
    D.3.9.3Other descriptive nameTRAMETINIB DIMETHYL SULFOXIDE
    D.3.9.4EV Substance CodeSUB119776
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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.IMP: 3
    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 Mekinist
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameMekinist
    D.3.2Product code TMT212
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNTRAMETINIB
    D.3.9.2Current sponsor codeTMT212
    D.3.9.3Other descriptive nameTRAMETINIB DIMETHYL SULFOXIDE
    D.3.9.4EV Substance CodeSUB119776
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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.IMP: 4
    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 Tafinlar
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameTafinlar
    D.3.2Product code DRB436
    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 INNdabrafenib
    D.3.9.2Current sponsor codeDRB436
    D.3.9.3Other descriptive nameDABRAFENIB MESYLATE
    D.3.9.4EV Substance CodeSUB45696
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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
    Patients with BRAF V600 mutation positive
    Pacientes con mutación BRAF V600 positiva
    E.1.1.1Medical condition in easily understood language
    Patients with BRAF V600 mutation positive
    Pacientes con mutación BRAF V600 positiva
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10027481
    E.1.2Term Metastatic melanoma
    E.1.2System Organ Class 100000018545
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate long term safety as assessed by the occurrence of AEs/SAEs.
    El objetivo principal es evaluar la seguridad a largo plazo, determinado por la aparición de AAs/AAGs
    E.2.2Secondary objectives of the trial
    The secondary objective of the study is to evaluate clinical benefit as assessed by the Investigator. Proportion of patients with clinical benefit as assessed by the Investigator will be summarized at scheduled visits using FAS.
    Evaluar el beneficio clínico, determinado por el investigador
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients eligible for inclusion in this study have to meet all of the following criteria:
    1. Patient is currently receiving treatment with dabrafenib/trametinib monotherapy or combination within a Novartis or former GSK sponsored study which has fulfilled the requirements for the primary objective.
    2. In the opinion of the Investigator would benefit from continued treatment.
    3. Patient has demonstrated compliance, as assessed by the Investigator, within the parent study protocol requirement(s).
    4. Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures.
    5. Written informed consent obtained prior to enrolling in the roll-over study and receiving study medication. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness.
    6. If the patient is a minor, the parent who signs the consent for the minor must be a legally recognized parent or guardian. Where deemed appropriate by the Investigator, and the child’s parent or guardian, the child will also be included in the all discussions about the trials and the minor’s assent if 7 years or older will be obtained.
    7. Does not require treatment with prohibited concomitant medications
    Los pacientes elegibles para inclusión en este estudio deberán cumplir todos los criterios siguientes:
    1. Pacientes que estén recibiendo actualmente tratamiento con dabrafenib/trametinib en monoterapia o en combinación dentro de un estudio patrocinado por Novartis o anteriormente GSK, que hayan cumplido los requisitos para el objetivo principal.
    2. A criterio del investigador deberían beneficiarse de la continuación del tratamiento.
    3. Pacientes que hayan demostrado cumplimiento, evaluado por el investigador, dentro del(los) requisito(s) del protocolo del estudio principal.
    4. Voluntad y capacidad para cumplir con las visitas programadas, planes de tratamiento y cualquier otro procedimiento del estudio.
    5. Consentimiento informado por escrito obtenido antes de la inclusión en el estudio de continuación y de recibir la medicación del estudio. Si el consentimiento no puede ser expresado por escrito, deberá ser testificado y documentado formalmente, a ser posible a través de un testigo de confianza independiente.
    6. Si el paciente es un menor, el padre/madre que firme el consentimiento para el menor deberá ser un padre/madre o tutor legalmente reconocido. Cuando el investigador y el padre/madre o tutor del(la) niño(a) lo consideren apropiado, el(la) niño(a) también participará en todas las discusiones sobre los ensayos y se obtendrá el asentimiento del menor si el(la) niño(a) tiene 7 años o más.
    E.4Principal exclusion criteria
    Patients eligible for this study must not meet any of the following criteria:
    1. Patient has been previously permanently discontinued from study treatment in the parent protocol due to any reason.
    2. Patient’s indication is commercially available and reimbursed in the local country.
    3. Patient has participated in a combination trial where dabrafenib and/or trametinib was dispensed in combination with another study medication.
    4. Patient currently has unresolved toxicities for which dabrafenib and/or trametinib dosing has been interrupted in the parent study.
    5. Pregnant or nursing (lactating) women Female patients who are lactating must discontinue nursing prior to the first dose of study treatment and must refrain from nursing throughout the treatment period and for 4 months following the last dose of study treatment.
    6. Concurrent treatment with other systemic anti-cancer therapies is not allowed (e.g., chemotherapy, immune, biologic, or targeted therapy), with the exception of radiotherapy.
    7. Female patient is of childbearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 150-days after stopping treatment. Highly effective contraception methods include:
    • Total abstinence (when this is in line with the preferred and usual lifestyle of the subject). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
    • Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
    • Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject.
    • Placement of a non-hormonal intrauterine device (IUD) or intrauterine system (IUS) with a documented failure rate of less than 1% per year.
    • Double-barrier contraception: condom and occlusive cap (diaphragm or cervical/vault caps) with a vaginal spermicidal agent (foam/gel/cream/suppository).
    Note: Hormonal-based methods (e.g., oral contraceptives) are not considered as highly effective methods of contraception due to potential drug-drug interactions with dabrafenib.
    8. Sexually active males unless they use a condom during intercourse while on treatment and for 150 days after stopping treatment should not father a child in this period. A condom is required to be used by vasectomized men as well during intercourse in order to prevent delivery of the drug via semen.
    9. Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (i.e. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential.
    Los pacientes elegibles para inclusión en este estudio no deberán cumplir ninguno de los criterios siguientes:
    1. Pacientes a los que se les haya suspendido definitivamente el tratamiento del estudio en el protocolo principal por cualquier motivo.
    2. La indicación del paciente está disponible comercialmente y es reembolsada en el país local.
    3. El paciente ha participado en un ensayo de combinación en el que dabrafenib y/o trametinib se administró en combinación con otra medicación del estudio.
    4. Paciente con toxicidades no resueltas actualmente por las que se le ha interrumpido la dosis de dabrafenib y/o trametinib en el estudio principal.
    5. Pacientes embarazadas o en periodo de lactancia. Las mujeres lactantes deberán suspender la lactancia antes de la primera dosis del tratamiento del estudio y deben abstenerse de amamantar durante todo el periodo de tratamiento y durante 4 meses después de la última dosis del tratamiento del estudio.
    6. No se permite tratamiento concomitante con otras terapias antineoplásicas sistémicas por ejemplo, quimioterapia, terapia inmunológica, biológica o dirigida), con la excepción de radioterapia.
    7. Mujeres físicamente fértiles, definidas como todas las mujeres fisiológicamente capaces de quedarse embarazadas, a no ser que estén utilizando métodos anticonceptivos altamente eficaces durante la dosificación y durante 150 días después de suspender el tratamiento. Los métodos anticonceptivos altamente eficaces incluyen:
    Abstinencia total (cuando esté en consonancia con el estilo de vida habitual y preferida de la paciente). La abstinencia periódica (por ejemplo, calendario, ovulación, métodos sintotérmicos, de postovulación) y el coitus interruptus no son métodos anticonceptivos aceptables.
    Esterilización femenina (que se le haya realizado una ooforectomía bilateral con o sin histerectomía), histerectomía total o ligadura de trompas por lo menos seis semanas antes de tomar el tratamiento del estudio. En caso de ooforectomía solo, sólo cuando el estado reproductor de la mujer haya sido confirmado con evaluación de seguimiento del nivel hormonal.
    Esterilización masculina (por lo menos 6 meses antes de la selección). La pareja varón vasectomizado debería ser la única pareja de dicha paciente.
    Colocación de un dispositivo intrauterino (DIU) o sistema intrauterino (SIU) no hormonal con una tasa de fallo documentada de menos de un 1% por año.
    Anticonceptivo de doble barrera: preservativo o capuchón oclusivo (diafragma o capuchón en bóveda/cervical) con un agente espermicida vaginal (gel/espuma/crema espermicida/supositorio vaginal).
    Nota: los métodos basados en hormonas (por ejemplo, anticonceptivos hormonales) no se consideran métodos anticonceptivos altamente eficaces debido a las posibles interacciones fármaco-fármaco con dabrafenib.
    8. Varones sexualmente activos, a menos que utilicen un preservativo durante el coito mientras reciban tratamiento y durante 150 días después de suspender el tratamiento y no deberían engendrar hijos durante este periodo. Los varones vasectomizados también deberán utilizar un preservativo durante el coito para evitar la liberación del fármaco vía fluido seminal.
    9. Las mujeres se considerarán postmenopáusicas y físicamente no fértiles si presentan amenorrea natural (espontánea) durante 12 meses con un perfil clínico apropiado (por ejemplo, edad apropiada, historial de síntomas vasomotores) o han sido sometidas a ooforectomía bilateral quirúrgica (con o sin histerectomía), histerectomía total o a ligadura de trompas por lo menos seis semanas antes. En el caso de ooforectomía sola, sólo cuando el estado reproductor de la mujer haya sido confirmado con evaluación de seguimiento del nivel hormonal será considerada físicamente no fértil.
    E.5 End points
    E.5.1Primary end point(s)
    Frequency and severity of AEs/SAEs
    Frecuencia y severidad de los AAs/AAGs
    E.5.1.1Timepoint(s) of evaluation of this end point
    Endpoints will be evaluated throughout the study.
    Las variables serán evaluadas durtante todo el transcurso del estudio.
    E.5.2Secondary end point(s)
    Proportion of patients with clinical benefit as assessed by the investigator at scheduled visits.
    Proporción de pacientes con beneficio clínico, determinado por el investigador en las visitas programadas
    E.5.2.1Timepoint(s) of evaluation of this end point
    Endpoints will be evaluated throughout the study.
    Las variables serán evaluadas durtante todo el transcurso 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 Yes
    E.6.6Pharmacokinetic No
    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
    Roll-over study
    Estudio de continuación
    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) Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Estudio de continuación
    Roll-over study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 EEA25
    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
    Austria
    Canada
    China
    Denmark
    France
    Germany
    Italy
    Korea, Republic of
    Netherlands
    Norway
    Poland
    Slovakia
    Spain
    Sweden
    Thailand
    Ukraine
    United Kingdom
    United States
    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
    The end of the study will occur after all patients in the study have completed their last assessment per protocol (llow-up visit that occurs 30 days after the patient’s last dose or until SAE follow up is resolved). Patients may continue until :
    End of treatment visit
     One of the premature patient withdrawal criteria is met
     5 years after the first patient’s first visit
     A local access program becomes available
     Current treatment becomes commercially available and reimbursed
    El final del estudio ocurrirá después del UVUP. La última evaluación para cada paciente es la visita de seguimiento a realizar 30 días después de la última dosis del tratamiento del estudio administrada al paciente o hasta la resolución de los seguimientos requeridos de AAG, lo que ocurra más tarde. Los pacientes puede continuar con el tratamiento del estudio hasta que se cumplan uno de los siguientes criterios, lo que ocurra primero: refierase al apartado 4.3 del protocol.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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 Yes
    F.1.1Number of subjects for this age range: 10
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 85
    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 No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 55
    F.4.2.2In the whole clinical trial 100
    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)
    Not different from the expected normal treatment of that condition
    Ninguno diferente del tratamiento habitual para esta condición.
    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 Decision2017-12-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-12-04
    P. End of Trial
    P.End of Trial StatusOngoing
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