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    Summary
    EudraCT Number:2015-002739-17
    Sponsor's Protocol Code Number:AN-EPI3331
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-11-03
    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 number2015-002739-17
    A.3Full title of the trial
    A Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
    Estudio fase III, aleatorizado, abierto, con evaluador enmascarado y comparador activo para evaluar la no inferioridad en la eficacia y seguridad de liprotamasa en sujetos con insuficiencia pancreática exocrina debida a fibrosis quística.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin
    Estudio del tratamiento con unidades equivalentes de liprotamasa oral de origen no porcino
    A.3.2Name or abbreviated title of the trial where available
    SOLUTION Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin
    SOLUTION Estudio del tratamiento con unidades equivalentes de liprotamasa oral de origen no porcino
    A.4.1Sponsor's protocol code numberAN-EPI3331
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02279498
    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 SponsorANTHERA Pharmaceuticals, Inc.
    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 supportAnthera Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLinical Spain, S.L.U.
    B.5.2Functional name of contact pointMar Torre Gómez
    B.5.3 Address:
    B.5.3.1Street AddressCalle Rosa de Lima, 1-bis. Edificio Alba
    B.5.3.2Town/ cityLas Matas / Madrid
    B.5.3.3Post code28290
    B.5.3.4CountrySpain
    B.5.4Telephone number+34913726059
    B.5.5Fax number+34913726060
    B.5.6E-mailupmmadrid@linical.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/3/04/222
    D.3 Description of the IMP
    D.3.1Product nameliprotamase
    D.3.4Pharmaceutical form Capsule
    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 INNNot assigned
    D.3.9.1CAS number 9001-62-1
    D.3.9.3Other descriptive nameLIPASE
    D.3.9.4EV Substance CodeSUB12545MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10000 to 40000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPROTEASE
    D.3.9.1CAS number 9074-07-1
    D.3.9.4EV Substance CodeSUB15036MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10000 to 40000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAmylase
    D.3.9.1CAS number 9000-90-2
    D.3.9.3Other descriptive nameAMYLASE
    D.3.9.4EV Substance CodeSUB12892MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1500 to 6000
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 PANCREAZE®
    D.2.1.1.2Name of the Marketing Authorisation holderJanssen Pharmaceuticals, Inc
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 namePANCREAZE®
    D.3.4Pharmaceutical form Capsule
    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 INNNot assigned
    D.3.9.1CAS number 8049-47-6
    D.3.9.3Other descriptive namePANCREATIN
    D.3.9.4EV Substance CodeSUB12545MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10500 to 21000
    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
    Pancreatic exocrine Insufficiency due to Cystic Fibrosis
    Insuficiencia Pancreática Exocrina debida a Fibrosis Quística
    E.1.1.1Medical condition in easily understood language
    Maldigestion of dietary macronutrients (pancreas not producing enough
    enzymes for digestion of fat, sugars and proteins) in Cystic Fibrosis
    Mala digestión de los macronutrientes alimenticios (el páncreas no produce las suficientes enzimas para la digestión de la grasa, azúcares y proteínas) en la Fibrosis Cística.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    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 study is to demonstrate the non-inferiority of the non-porcine enzyme product, liprotamase, to Pancreaze for the change in coefficient of fat absorption (CFA) from screening to the end of the treatment period in subjects ?7 years of age with EPI due to cystic fibrosis (CF).
    El objetivo principal de este estudio es demostrar la no inferioridad del producto enzimático no porcino, liprotamasa, en comparación con Pancreaze en relación con el cambio en el coeficiente de absorción de grasas (CAG) desde la selección hasta el final del periodo de tratamiento en sujetos ?7 años de edad con insuficiencia pancreática exocrina (IPE) debida a fibrosis quística (FQ).
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are to evaluate and compare effects of liprotamase and Pancreaze on measures of digestion including the coefficient of nitrogen absorption (CNA), stool weight, symptoms of malabsorption.
    The exploratory objectives of the study is to evaluate the impact on health related quality of life at the end of the treatment period and the extension period as measured by the Cystic Fibrosis Questionnaire ? Revised (2002; CFQ-R).

    Safety will be evaluated throughout the study based on adverse events (AEs) including serious AEs (SAEs), blood chemistry, hematology, urinalysis, physical examinations, vital signs, and measurement of total cholesterol, albumin, prealbumin and fat-soluble vitamin levels.
    Los objetivos secundarios del estudio son evaluar y comparar los efectos de liprotamasa y Pancreaze sobre las medidas de la digestión, incluido el coeficiente de absorción de nitrógeno (CAN), el peso de las heces y los síntomas de malabsorción.
    Los objetivos exploratorios del estudio son evaluar el impacto sobre la calidad de vida relacionada con la salud al final del periodo de tratamiento y del periodo de extensión según se determina mediante el Cuestionario de Fibrosis Quística - Revisado (2002, CFQ-R por sus siglas en inglés).

    La seguridad se evaluará a lo largo de estudio en función de los acontecimientos adversos (AA) incluyendo los AA graves (AAG), análisis bioquímico de la sangre, hematología, análisis de orina, exploraciones físicas, constantes vitales y medidas de colesterol total, albúmina, prealbúmina y niveles de vitaminas liposolubles.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Cystic Fibrosis based on the disease diagnostic criteria:
    ? Two clinical features consistent with CF, and
    ? Either:
    a. genotype with 2 identifiable mutations consistent with CF, or
    b. sweat chloride >60 mEq/L by quantitative pilocarpine iontophoresis.
    2. Males or females ? 7 years of age at the time of screening.
    3. Have fecal elastase <100 mcg/g stool measured during screening unless previously documented.
    4. Have CFA during screening of ?80% while receiving PERT therapy.
    5. Are clinically stable with no evidence of acute upper or lower respiratory tract infection in the last 10 days prior to screening.
    6. Are of fair or better nutritional status as defined by:
    ? BMI ?16.0 kg/m2 for female subjects ?20 years of age, or
    ? BMI ?16.5 kg/m2 for male subjects ?20 years of age, or
    ? BMI ?25th percentile for subjects 7 to <20 years of age.
    ? Weight for all age groups has remained stable (no more than a 5% decline) during the 90 days prior to screening.
    7. Are able to take pancreatic enzyme supplementation in the form of capsules.
    8. Have been using the same pancreatic enzyme treatment for the past 30 days without change in dosing regimen.
    9. Are able to perform testing and procedures required for the study, as judged by the Investigator.
    10. If adult, have given written informed consent or assent as required by Anthera or its designee and the Institutional Review Board/Independent Ethic Committee (IRB/IEC). If not adult, have parent or legal guardian who has given written informed consent approved by Anthera or its designee and the IRB/IEC governing the site.
    1. Fibrosis quística según los criterios diagnósticos de la enfermedad:
    ? Dos características clínicas indicativas de FQ, y
    ? Bien:
    a. genotipo con dos mutaciones identificables indicativas de FQ, o bien
    b. cloruro en el sudor >60 mEq/l determinado mediante iontoforesis cuantitativa de pilocarpina.
    2. Varones o mujeres ?7 años de edad en el momento de la selección.
    3. Tener una concentración de elastasa fecal <100 µg/g de heces determinada durante la selección, a menos que se haya documentado previamente.
    4. Tener un CAG durante la selección de ?80 % mientras se está recibiendo terapia TSEP.
    5. Estar clínicamente estable sin indicios de infección aguda de las vías respiratorias altas o bajas en los últimos 10 días antes de la selección.
    6. Tener un estado nutricional aceptable o mejor según lo definido por:
    ? IMC ?16,0 kg/m2 para mujeres ?20 años de edad, o
    ? IMC ?16,5 kg/m2 para varones ?20 años de edad, o
    ? IMC ? al percentil 25 para sujetos de 7 a <20 años de edad.
    ? El peso para todos los grupos de edad ha permanecido estable (disminución no superior al 5 %) durante los 90 días previos a la selección.
    7. Ser capaz de tomar un suplemento de enzimas pancreáticas en forma de cápsulas.
    8. Haber estado usando el mismo tratamiento con enzimas pancreáticas durante los últimos 30 días sin cambios en la pauta posológica.
    9. Ser capaz de realizar las pruebas y procedimientos necesarios para el estudio, según el criterio del investigador.
    10. En el caso de adultos, haber dado el consentimiento informado por escrito o el asentimiento según los requisitos de Anthera o su representante y el Comité de Revisión Institucional/Comité Ético de Investigación Clínica (IRB/CEIC). En el caso de menores de edad, tener un progenitor o tutor que haya dado su consentimiento informado por escrito aprobado por Anthera o su representante y el IRB/CEIC que tutela al centro.
    E.4Principal exclusion criteria
    1. Presently using Pancreaze as enzyme preparation for treatment of EPI. Patients are not required to be Pancreaze naïve but should not have received Pancreaze within 30 days of screening.
    2. Women who are breast-feeding during the study.
    3. A history or diagnosis of fibrosing colonopathy (FC).
    4. History of distal intestinal obstruction syndrome (DIOS) in the 6 months prior to the screening visit.
    5. Any chronic diarrheal illness unrelated to pancreatic insufficiency (eg, infectious gastroenteritis, lactose intolerance, sprue or inflammatory bowel disease).
    6. A history of liver transplant, lung transplant, or significant surgical resection of the bowel. Significant resection of the bowel is defined as any resection of the terminal ileum or ileocecal valve. Patients who have had qualitative, long term changes in nutritional status after any other bowel resection (e.g., increased or new need for pancreatic enzyme supplementation compared to pre operative status in order to maintain the same nutritional status) should also be excluded.
    7. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level ?5 times upper limit of normal (ULN), or total bilirubin level ?1.5 times ULN at the screening visit.
    8. Signs and/or symptoms of liver cirrhosis or portal hypertension (eg, splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF.
    9. Forced expiratory volume in 1 second (FEV1) <30% of the predicted value.
    10. Known hypersensitivity to food additives or Pancreaze.
    11. Use of an enzyme preparation in excess of 10,000 lipase units/kg/day at screening or 2500 lipase units/kg per meal.
    12. Uncontrolled hyperglycemia or uncontrolled CF-related diabetes.
    13. Uncontrolled hyperuricemia or hyperuricosuria as determined by the principal Investigator (PI).
    14. Feeding via an enteral tube.
    15. Routine use of anti-diarrheals, anti-spasmodics, or cathartic laxatives, or a change in chronic osmotic laxative (eg, polyethylene glycol) regimen in the previous 3 months.
    16. Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interests of the patient.
    17. Unlikely to complete the study, as determined by the Investigator.
    18. Currently enrolled in, or have discontinued within the last 30 days (prior to the screening visit) from a clinical trial involving use of an investigational drug, biologic or device, or are currently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study.
    19. Females who are nursing, pregnant, intending to become pregnant or intending to nurse during the time of the study, or who have a positive pregnancy test at baseline. All sexually-active subjects of reproductive potential are required to use a reliable method of birth control. Females and males are required to use or start using a reliable method of birth control at least 2 weeks prior to randomization, throughout the study, and for at least 3 months following completion of study therapy. A reliable method of birth control is defined as one of the following: oral or injectable contraceptives, intrauterine device, contraceptive implants, tubal ligation, hysterectomy, or a double-barrier method (diaphragm with spermicidal foam or jelly, or a condom), abstinence or vasectomy.
    1. Uso actualmente de Pancreaze como preparado enzimático para el tratamiento de la IPE. No es necesario que los pacientes no hayan usado nunca Pancreaze, pero no deben haberlo recibido en los 30 días previos a la selección.
    2. Mujeres en periodo de lactancia durante el estudio.
    3. Antecedentes o diagnóstico de colonopatía fibrosante (CF).
    4. Antecedentes de síndrome de obstrucción intestinal distal (SOID) en los 6 meses previos a la visita de selección.
    5. Cualquier enfermedad diarreica crónica no relacionada con la insuficiencia pancreática (p. ej., gastroenteritis infecciosa, intolerancia a la lactosa, esprúe o enfermedad inflamatoria intestinal).
    6. Antecedentes de trasplante de hígado, trasplante de pulmón o resección quirúrgica importante del intestino. Se define resección quirúrgica importante del intestino como cualquier resección del íleon terminal o la válvula ileocecal. También se debe excluir a los pacientes que hayan presentado cambios cualitativos a largo plazo en el estado nutricional después de cualquier otra resección intestinal (p. ej., nueva necesidad o aumento de la necesidad de suplemento de enzimas pancreáticas en comparación con el estado preoperatorio para mantener el mismo estado nutricional).
    7. Niveles de alanina aminotransferasa (ALT) o aspartato aminotransferasa (AST) ?5 veces el límite superior normal (LSN) o un nivel de bilirrubina total ?1,5 veces el LSN en la visita de selección.
    8. Signos y/o síntomas de cirrosis hepática o hipertensión portal (p. ej., esplenomegalia, ascitis, varices esofágicas) o enfermedad hepática documentada no relacionada con la FQ.
    9. Volumen espiratorio forzado en 1 segundo (VEF1) <30 % del valor previsto.
    10. Hipersensibilidad conocida a aditivos alimenticios o a Pancreaze.
    11. Uso de un preparado enzimático en exceso de 10 000 unidades de lipasa/kg/día en la selección o 2500 unidades de lipasa/kg por comida.
    12. Hiperglucemia no controlada o diabetes no controlada relacionada con la FQ.
    13. Hiperuricemia o hiperuricosuria no controlada según lo determinado por el investigador principal (IP).
    14. Alimentación mediante sonda enteral.
    15. Uso habitual de antidiarreicos, antiespasmódicos o laxantes catárticos, o un cambio en el pauta del laxante osmótico crónico (p. ej., polietilenglicol) en los 3 meses previos.
    16. Cualquier afección que el investigador considere que podría interferir con la intención de este estudio o hacer que la participación no sea lo mejor para el paciente.
    17. Escasas probabilidades de completar el estudio, según lo determinado por el investigador.
    18. Estar incluido actualmente en, o haber abandonado en los últimos 30 días (antes de la visita de selección), un ensayo clínico que implique el uso de un fármaco, producto biológico o dispositivo en investigación, o estar actualmente incluido en cualquier otro tipo de investigación médica considerada no compatible con este estudio desde el punto de vista científico o médico.
    19. Mujeres en periodo de lactancia, embarazadas, con intención de quedarse embarazadas o con intención de dar el pecho durante el estudio, o que tengan una prueba de embarazo positiva al inicio del estudio. Todos los sujetos sexualmente activos potencialmente fértiles deben usar un método fiable de control de la natalidad. Tanto mujeres como varones deben usar o empezar a usar un método fiable de control de la natalidad al menos 2 semanas antes de la aleatorización, durante todo el estudio, y durante al menos 3 meses después de completar el tratamiento del estudio. Un método fiable de control de la natalidad se define como uno de los siguientes: anticonceptivos orales o inyectables, dispositivo intrauterino, implantes anticonceptivos, ligadura de trompas, histerectomía o método de doble barrera (diafragma con espuma o gel espermicida, o preservativo), abstinencia o vasectomía.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy criterion is the CFA (Coefficient of Fat Absorption).
    CFA will be calculated from fat intake and fat excretion, according to the
    formula: CFA (%) = 100 [fat intake ? fat excretion] / fat intake
    El principal criterio de eficacia es el CAG (Coeficiente de Absorción de Grasas).
    Se calculará el CAG desde la ingesta de grasa y a la excreción de grasa, en base a la fórmula: CAG (%) = 100 [ingesta de grasa ? excreción de grasa] / ingesta de grasa
    E.5.1.1Timepoint(s) of evaluation of this end point
    The values at the end of the treatment period of liprotamase, and of the active control Pancreaze®, will be compared .
    Se compararán los valores al final del periodo de tratamiento con liprotamasa, y con el control activo Pancreaze®.
    E.5.2Secondary end point(s)
    The secondary endpoints for this trial are as follows:
    ?the change in coefficient of nitrogen absorption (CNA) from screening to the end of the treatment period.
    ?the change in stool weight from screening to the end of the treatment period (measured during the marker-to-marker stool collection).
    ?the signs and symptoms of malabsorption (abdominal pain, bloating, steatorrhea, flatulence, etc) at baseline, during the treatment period, and the extension period.
    ?the change from baseline in height, weight, and body mass index (BMI) to the end of the treatment period and the extension period.
    ?the change in total cholesterol, vitamin (A, D, E, and K) levels, albumin, and prealbumin from baseline to the end of the treatment period and the extension period.
    The exploratory objectives of the study are:
    ?the impact on health related quality of life at the end of the treatment period and the extension period as measured by the Cystic Fibrosis Questionnaire ? Revised (2002; CFQ-R)

    The safety data collected during the study are adverse events (AEs) including serious AEs (SAEs), blood chemistry, hematology, urinalysis, physical examinations, vital signs, and measurement of total cholesterol, albumin, prealbumin and fat-soluble vitamin levels.
    Los criterios secundarios de valoración para este ensayo clínico son los siguientes:
    ? cambio en el coeficiente de absorción de nitrógeno (CAN) desde la selección hasta el final del periodo de tratamiento.
    ? el cambio en el peso de las heces desde la selección hasta el final del periodo de tratamiento (medido durante la recogida de heces de marcador a marcador).
    ? los signos y síntomas de malabsorción (dolor abdominal, distensión abdominal, esteatorrea, flatulencia, etc.) al inicio del estudio, durante el periodo de tratamiento y durante el periodo de extensión.
    ? el cambio con respecto al inicio del estudio en la estatura, el peso y el índice de masa corporal (IMC) hasta el final del periodo de tratamiento y el periodo de extensión.
    ? el cambio en el colesterol total, niveles de vitaminas (A, D, E y K), albúmina y prealbúmina desde el inicio del estudio al final del periodo de tratamiento y del periodo de extensión.
    Los objetivos exploratorios del estudio son:
    ? el impacto sobre la calidad de vida relacionada con la salud al final del periodo de tratamiento y del periodo de extensión según se determina mediante el cuestionario de fibrosis quística revisado (2002, CFQ-R por sus siglas en inglés).

    Los datos de seguridad que se recogerán a lo largo de estudio son acontecimientos adversos (AA) incluyendo los AA graves (AAG), análisis bioquímico de la sangre, hematología, análisis de orina, exploraciones físicas, constantes vitales y medidas de colesterol total, albúmina, prealbúmina y niveles de vitaminas liposolubles.
    E.5.2.1Timepoint(s) of evaluation of this end point
    For the secondary endpoints, the values at the end of the treatment period of liprotamase, and of the active control Pancreaze®, will be compared .

    The exploratory objective is evaluated at the end of treatment period and at the end of the extension period.

    Safety parameters are assessed throughout the study. Height, weight, BMI, and signs and symptoms of malabsorption will be also evaluated during the extension period.
    Para los criterios de valoración secundarios, se compararán los valores al final del periodo de tratamiento con liprotamasa, y con el control activo Pancreaze®.

    El objetivo exploratorio es evaluar al final del periodo de tratamiento y al final del periodo de extensión.

    Se evaluarán a lo largo del estudio los parámetros de seguridad. La estatura, el peso, el IMC, y los signos y síntomas de malabsorción serán también evaluados durante el periodo de extensión.
    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 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 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) 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Canada
    Israel
    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
    LVLS
    LVLS (Ultima Visita Ultimo Sujeto)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    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 years1
    E.8.9.2In all countries concerned by the trial months3
    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: 75
    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: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 45
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 75
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    For minors, the parents will have to give their consent. The minors will also have to give their assent.
    Para los menores, los padres tendrán que otorgar su consentimiento. Los menores tendrán que otorgar también su asentimiento.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 150
    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)
    The patient will return to original PERT therapy.
    El paciente volverá a su terapia TSEP original
    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 Decision2015-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-12-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-01-17
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