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    Summary
    EudraCT Number:2014-001670-34
    Sponsor's Protocol Code Number:AZP01-CLI-002
    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:2014-10-24
    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 number2014-001670-34
    A.3Full title of the trial
    A Phase IIa, randomized, double-blind, placebo-controlled, multi-center study to evaluate the safety, tolerability, and effects of AZP-531, an Unacylated Ghrelin analog, on food-related behavior in patients with Prader-Willi Syndrome
    Ensayo en fase IIa multicéntrico, aleatorizado, con doble enmascaramiento y controlado con placebo para evaluar la seguridad, tolerabilidad y efectos de AZP-531, un análogo de la grelina no acilada, en la conducta alimentaria en pacientes con síndrome de Prader-Willi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase IIa study to evaluate the safety, tolerability, and effects of AZP-531 on food-related behavior in patients with Prader-Willi Syndrome
    Ensayo en fase IIa para evaluar la seguridad, tolerabilidad y efectos de AZP-531 en la conducta alimentaria en pacientes con síndrome de Prader-Willi
    A.4.1Sponsor's protocol code numberAZP01-CLI-002
    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 SponsorAlize Pharma
    B.1.3.4CountryFrance
    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 supportAlizé Pharma
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlizé Pharma
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street Address15G Chemin du Saquin
    B.5.3.2Town/ cityEcully,
    B.5.3.3Post code69130
    B.5.3.4CountryFrance
    B.5.4Telephone number+33472180924
    B.5.5Fax number+33478332629
    B.5.6E-mailallas@alz-pharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAZP-531
    D.3.2Product code AZP-531
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNINN Not yet proposed
    D.3.9.2Current sponsor codeAZP-531
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.2
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboLyophilisate for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Prader-Willi Syndrome
    Síndrome de Prader-Willi
    E.1.1.1Medical condition in easily understood language
    Prader-Willi Syndrome
    Síndrome de Prader-Willi
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level HLGT
    E.1.2Classification code 10003018
    E.1.2Term Appetite and general nutritional disorders
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10036476
    E.1.2Term Prader-Willi syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level HLT
    E.1.2Classification code 10003022
    E.1.2Term Appetite disorders
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of one dose of AZP-531 adjusted for bodyweight (2, 3 or 4 mg) administered over 2 weeks as daily subcutaneous injection.
    Evaluar la seguridad y tolerabilidad de una dosis de AZP-531 ajustada al peso corporal y administrada durante 2 semanas en forma de inyección subcutánea diaria.
    E.2.2Secondary objectives of the trial
    To evaluate effects of AZP-531 after 2 weeks of administration on fullness/satiety and appetite/prospective food consumption as assessed by Numerical Rating Scales (NRS).
    Evaluar los efectos de AZP-531 después de 2 semanas de administración con plenitud/saciedad y apetito/consumo alimento prospectivo, evaluados con escalas de valoración numéricas (NRS).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Male and female patients with genetically confirmed diagnosis of Prader-Willi Syndrome using standard DNA methylation test or fluorescent in situ hybridization
    -Patients aged 18 years to 40 years old inclusive initially
    o Patients aged 12 years old to less than 18 years old after formal decision of the Data Monitoring Committee
    -Have evidence of increased appetite or hyperphagia, as judged by the investigator
    -Patients on well-balanced controlled diet and on a regular exercise program as recommended in common clinical practice for this patient population
    -Women of Child Bearing Potential (WCBP) must have a negative pregnancy test on admission to the study center
    -All WCBP, sexually active male subjects, and all partners of subjects should agree to use adequate methods of birth control (diaphragm, condoms with spermicide?) throughout the study and for 30 days after the last dose of study drug
    -Adequate renal function, defined as serum creatinine inferior or equal to 1.5 x Upper Limit of Normal (ULN) and urine protein/creatinine ratio <0.2
    -Adequate hepatic function, defined as total bilirubin inferior or equal to 1.5 x ULN and Aspartate aminotransferase (AST) and Alanine aminotransferase (ALT) levels inferior or equal to 3 x ULN
    -Growth hormone treatment will be permitted if doses have been stable for at least 1 month prior to screening
    -Psychotropic treatment will be permitted and should be stable at least 1 month prior to screening
    -Any other treatment including thyroid hormones should be stable for at least 1 month prior to screening
    -Informed consent signed by the patient, parent or legal guardian, as appropriate
    -Pacientes, hombres y mujeres, con diagnóstico genético confirmado de síndrome de Prader-Willi mediante prueba estándar de metilación de ADN o hibridación fluorescente in situ.
    -Pacientes con edades comprendidas entre 18 y 40 años, ambos incluidos, inicialmente.
    o Pacientes entre 12 y menos de 18 años después de una decisión formal del Comité de vigilancia de datos de seguridad.
    -Tener pruebas de aumento del apetito o hiperfagia, a criterio del investigador.
    -Pacientes con dietas equilibradas y controladas y que siguen un programa de ejercicio regular según recomienda la práctica clínica común para esta población de pacientes.
    -Las mujeres con capacidad reproductiva deben dar negativo en una prueba de embarazo al ser admitidas en el centro del ensayo.
    -Todas las mujeres con capacidad reproductiva, sujetos varones sexualmente activos y todas las parejas de los sujetos deberían acordad utilizar métodos adecuados de control de natalidad (diafragma, preservativos con espermicida...) durante la duración del ensayo y hasta 30 días después de la última dosis del fármaco del ensayo.
    -Función renal adecuada, definida como creatinina sérica inferior o igual a 1,5 por límite superior normal (LSN) y proporción de proteína/creatinina en orina <0,2.
    -Función hepática adecuada, definida como bilirrubina total inferior o igual a 1,5 por LSN y niveles de aspartato aminotransferasa (AST) y alanina aminotransferasa (ALT) inferior o igual a 3 por LSN.
    -Se permitirá el tratamiento con hormona del crecimiento si las dosis han sido estables durante al menos 1 mes antes de la selección.
    ?Se permitirá tratamiento psicotrópico y debería ser estable al menos 1 mes antes de la selección.
    -Cualquier otro tratamiento, incluidas las hormonas para la tiroides, debería ser estable durante al menos 1 mes antes de la selección.
    -Consentimiento informado firmado por los pacientes, padres o tutores legales, según sea el caso.
    E.4Principal exclusion criteria
    -History of chronic liver disease, such as cirrhosis or chronic hepatitis due to any cause, or suspected alcohol abuse
    - History of acute or chronic pancreatitis
    -Type 1 diabetes
    -Insulin treatment
    -Use of weight loss agents or drugs known to affect appetite (including GLP-1 analogs) within 2 months prior to screening
    -Co-morbid condition or disease (such as respiratory disease or psychiatric disorder) diagnosed less than 1 month prior to screening
    -Co-morbid condition or disease or abnormal laboratory finding that would in the investigator judgment increase the subject risk to participating in this study and that will not allow the patient to complete the study
    -History or presence of gastrointestinal, hepatic or renal disease or any other condition known to interfere with the absorption, distribution, metabolism or excretion of drugs
    -Participation in a clinical trial with an investigational agent within 2 months prior to screening
    -Clinically significant abnormalities on ECG at screening
    -Pregnant or lactating woman
    -History of hypersensitivity to drugs with a similar chemical structure or class as AZP-531 (Acylated Ghrelin and Unacylated Ghrelin)
    -Unwillingness or inability to follow the procedures outlined in the protocol
    -Historias de enfermedades hepáticas crónicas, como cirrosis o hepatitis crónica provocadas por cualquier causa, o posible abuso del alcohol.
    -Historia de pancreatitis aguda o crónica
    -Diabetes tipo 1.
    -Tratamiento con insulina.
    -Uso de agentes o fármacos adelgazantes que afectan al apetito (incluidos análogos del GLP-1) en los 2 meses previos a la selección.
    -Afección o enfermedad comórbida (como afección respiratoria o trastorno psiquiátrico) diagnosticada menos de 1 mes antes de la selección.
    -Afección o enfermedad comórbida o resultado de laboratorio anormal que, a juicio del investigador, aumentaría el riesgo del sujeto al participar en este ensayo y que no permitirá al paciente completar el ensayo.
    -Historial o presencia de enfermedad gastrointestinal, hepática o renal o cualquier otra afección conocida por interferir en la absorción, distribución, metabolización o excreción de medicamentos.
    ?Participación en un ensayo clínico con un agente en fase de investigación 2 meses antes de la selección.
    -Anomalías clínicamente significativas en ECG en el momento de la selección.
    -Mujer embarazada o en periodo de lactancia.
    ?Historial de hipersensibilidad a los fármacos con una clase o estructura química similar a AZP-531 (grelina acilada y grelina no acilada).
    -Reticencia a seguir los procedimientos marcados en el protocolo o incapacidad para seguirlos.
    E.5 End points
    E.5.1Primary end point(s)
    Evaluation of safety will be based on patients who have received at least one dose of study medication. Individual and summary statistics including mean, median, standard-deviation (SD) and range will be presented in tabular form by treatment group for vital signs and clinical laboratory data.
    Adverse events will be tabulated and summarised according to the current version of the Medical Dictionary for Regulatory Activities (MedDRA) and presented using the total number of AEs and the total number and percent of patients who experienced an AE per treatment group.
    La evaluación de la seguridad se basará en pacientes que han recibido al menos una dosis de la medicación del estudio. Las estadísticas individuales y sumarias, incluyendo, mediana, desviación estándar-media (DE) y rango se presentarán en forma de tabla por grupo de tratamiento de los signos vitales y datos de laboratorio clínico.
    Los eventos adversos serán tabulados y resumidos de acuerdo con la versión actual del Diccionario Médico para Actividades Regulatorias (MedDRA) y presentado utilizando el número total de eventos adversos y el número total y el porcentaje de pacientes que experimentaron un AE por grupo de tratamiento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    AEs will be collected from the signature of informed consent.
    Safety laboratory evaluation will be performed at screening (D-2) and at the end of treatment period (D14)
    Vital signs will be obtained at screening (D-2), Baseline (D-1) and at the end of treatment period (D14)
    Waist Circumference, Weight, Height and BMI will be measured (derived from weight and height for BMI) at Baseline (D-1) and at the end of treatment period (D14) except for Height (only D-1)
    EAs se recogerán a partir de la firma del consentimiento informado.
    Evaluación de laboratorio de seguridad se llevará a cabo en el screening (D-2) y al final del período de tratamiento (D14)
    Los signos vitales se obtendrán en el screening (D-2), visita basal (D-1) y al final del período de tratamiento (D14)
    Circunferencia de la cintura, peso, talla e índice de masa corporal se mide (derivada del peso y la altura para el IMC) en visita basal (D-1) y al final del período de tratamiento (D14), excepto para la altura (sólo D-1)
    E.5.2Secondary end point(s)
    The Numeric Rating Scales: fullness/satiety and appetite/prospective food consumption will be presented descriptively by time (before meal, just after meal, 120 minutes after meal), meal (breakfast, lunch), visit (D-1, D1, D14) and treatment.

    Analysis of covariance (ANCOVA) or analysis of variance (ANOVA) will be performed at Day 14 for the afforded mentioned parameters. The model will include the parameter at baseline (when appropriate), the stratification variables and the treatment group. Key interactions will also be included to explore subgroup effect (e.g. Age Group-by-Treatment, Genetic Subtype-by-Treatment).
    Las escalas de valoración numéricas: saciedad o apetito/ganas de comer se presentarán de manera descriptiva por momento (antes de una comida, justo después de una comida, 120 minutos después de una comida), comida (desayuno, comida), visita (día -1, día 1 y día 14) y tratamiento.
    El análisis de covarianza (ANCOVA) o análisis de la varianza (ANOVA) se llevará a cabo en el día 14 para los parámetros mencionados. El modelo incluirá el parámetro al inicio del estudio (cuando sea aplicable), las variables de estratificación y el grupo de tratamiento. Interacciones clave también se incluirán para explorar el efecto de subgrupos (por ejemplo, grupo de edad-por-Tratamiento, Subtipo por tratamiento genético).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Numerical Rating Scales will be administered to patients at baseline (D-1), first day of treatment (D1) and at the end of treatment period (D14)
    las escalas de valoración numéricas serán administradas a los pacientes en la visita basal (D-1), primer día de tratamiento (D1) y al final del período de tratamiento (D14)
    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 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) No
    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 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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 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
    Última visita último paciente
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    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
    Some of the Prader-Willy patients are not able to give consent personnaly
    Algunos de los pacientes de Prader-Willy no son capaces de dar su consentimiento personalmente
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 40
    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)
    Following analysis of unblinded study data and if clinical benefits are observed, AZP-531 will be made available for 14 days for placebo patients. Informed consent should be obtained from patients, parents and/or legal guardians, as appropriate. Safety data will be collected during this time period.
    Tras el análisis de datos de estudios no ciegos, y si se observan beneficios clínicos, AZP-531 estará disponible durante 14 días para los pacientes tratados con placebo. El consentimiento informado debe ser obtenido de pacientes, padres y / o tutores legales, según corresponda. Los datos de seguridad se recogerán durante este período de tiempo.
    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 Decision2015-01-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-11-04
    P. End of Trial
    P.End of Trial StatusCompleted
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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
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