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    Summary
    EudraCT Number:2014-001670-34
    Sponsor's Protocol Code Number:AZP01-CLI-002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-07-08
    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 ConcernedItaly - Italian Medicines Agency
    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
    Studio multicentrico di Fase IIa randomizzato, in doppio cieco, controllato con placebo per valutare la sicurezza, la tollerabilità e gli effetti di AZP-531, un analogo della grelina deacilata, sul comportamento alimentare dei pazienti con Sindrome di 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
    Uno studio di fase IIa per valutare la sicurezza, la tollerabilità e gli effetti di AZP-531 sul comportamento alimentare nei pazienti con sindrome di 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
    Sindrome di Prader Willi.
    E.1.1.1Medical condition in easily understood language
    Prader-Willi Syndrome
    Sindrome di 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 18.0
    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 18.0
    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 18.0
    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.
    L'obiettivo primario è di valutare la sicurezza e la tollerabilità di una dose di AZP-531 in funzione del peso corporeo, somministrata nel corso di 2 settimane sotto forma di iniezioni sottocutanee giornaliere.
    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).
    Gli obiettivi secondari sono di valutare gli effetti di AZP-531 dopo 2 settimane di somministrazione su pienezza/sazietà e appetito/consumo previsto di cibo, valutati tramite Scale Numeriche di Valutazione (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 50 years old inclusive
    • 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
    • In France, patients must be affiliated to the sécurité sociale and must be under guardianship measures
    • Informed consent signed by the legal guardian or by the subject assisted by his/her legal representative, as appropriate
    • Pazienti di sesso maschile e femminile con Sindrome di Prader-Willi diagnosticata e confermata geneticamente da test standard di metilazione del DNA o ibridazione fluorescente in situ
    • Inizialmente solo pazienti di età compresa tra 18 e 50 anni
    o Dopo la decisione formale del DMC, anche pazienti di età compresa tra 12 e 18 anni
    • Evidente aumento dell'appetito o iperfagia, a giudizio dello sperimentatore,
    • Pazienti a dieta controllata ben bilanciata e che seguono un programma di esercizio fisico regolare, come raccomandato nella normale pratica clinica per questa popolazione di pazienti
    • Donne in età fertile (WCBP) devono avere il test di gravidanza negativo all'ammissione in studio
    • Tutte le donne in età fertile, i soggetti di sesso maschile sessualmente attivi, e tutti i compagni/e dei soggetti partecipanti allo studio devono accettare l'uso di adeguati metodi anticoncezionali (diaframma, preservativo con spermicida…) per tutta la durata dello studio e per 30 giorni dopo l'ultima dose di farmaco in studio
    • Adeguata funzionalità renale, definita come creatinina sierica 1,5  ULN (limite superiore di normalità) e rapporto proteine/creatinina urinario <0,2
    • Adeguata funzionalità epatica, definita come bilirubina totale 1,5  ULN e livelli di aspartato aminotransferasi (AST) e alanina aminotransferasi (ALT) 3  ULN
    • E’ ammesso il trattamento con ormone della crescita se le dosi sono stabili da almeno 1 mese prima della visita di screening
    • Sono consentiti i trattamenti con farmaci psicotropi se stabili da almeno 1 mese prima della visita di screening
    • E’ ammesso qualsiasi altro trattamento, compresi gli ormoni tiroidei, se stabili da almeno 1 mese prima della visita di screening
    • Modulo di consenso informato firmato dal/la paziente, genitore/i o tutore/i legale/i, come richiesto
    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
    • Anamnesi di epatopatia cronica, come cirrosi o epatite cronica dovute a qualsiasi causa, o sospetto abuso di alcool
    • Anamnesi di pancreatite acuta o cronica
    • Diabete di tipo 1
    • Trattamento con insulina
    • Uso di farmaci per perdere peso o farmaci noti per la loro azione sull'appetito (compresi gli analoghi del GLP-1) entro 2 mesi prima della visita di screening
    • Comorbilità (come patologia respiratoria o disturbo psichiatrico) diagnosticata entro di 1 mese della visita di screening
    • Comorbilità o anomalie nelle analisi di laboratorio che, a giudizio dello sperimentatore, potrebbero aumentare il rischio per il soggetto, alla partecipazione a questo studio, e che non permetteranno al paziente di completare lo studio
    • Anamnesi o presenza di patologia gastrointestinale, epatica o renale o di qualsiasi altra condizione nota per interferire con l'assorbimento, distribuzione, metabolismo o escrezione dei farmaci
    • Partecipazione a una sperimentazione clinica con un prodotto sperimentale entro 2 mesi dalla visita di screening
    • Anomalie clinicamente significative dell'ECG alla visita di screening
    • Gravidanza o allattamento
    • Anamnesi di ipersensibilità a farmaci dalla struttura chimica simile o di classe simile ad AZP-531 (grelina acilata e grelina deacilata)
    • Riluttanza o incapacità di seguire le procedure delineate nel protocollo
    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 valutazione della sicurezza si baserà sui pazienti ai quali è stata somministrata almeno una dose di farmaco in studio. I dati statistici individuali e riepilogativi comprendenti media, mediana, deviazione standard (SD) e intervallo verranno presentati in tabella, per gruppo di trattamento, per segni vitali e dati clinici di laboratorio.
    Gli eventi avversi verranno presentati in tabella, riassunti conformemente alla versione attuale del MedDRA (Medical Dictionary for Regulatory Activities) e presentati come numero totale di EA e numero totale e percentuale di pazienti con EA, per gruppo di trattamento.
    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)
    Gli AEs saranno raccolti dalla firma del ICF. I dati clinici di laboratorio saranno valutati alla visita di screening (giorno-2) e alla visita di fine trattamento (giorno 14). Segni vitali alla visita -2, al basale (giorno -1) e alla fine del trattamento (giorno 14). La circonferenza vita, il peso, l’altezza, la massa grassa e la misurazione Indice di massa corporea saranno misurati al basale (giorno-) alla fine del trattamento (giorno 14), ad eccezione dell’altezza che verrà misurata solo al giono -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).
    Le scale numeriche di valutazione riguardano: la pienezza/sazietà e appetito/cibo consumato teorico raccolti a tempi stabiliti (prima del pasto, appena dopo il pasto, 120 min dopo il pasto), a pasto (colazione, pranzo), a visita (giorno-1, 1, 14) e al trattamento.
    L'analisi della covarianza (ANCOVA) o l'analisi della varianza (ANOVA) verranno effettuate al Giorno 14 per i parametri summenzionati. Il modello comprenderà il parametro al basale (ove appropriato), le variabili di stratificazione e il gruppo di trattamento. Verranno incluse anche le interazioni chiave per analizzare l'effetto dei sottogruppi (per es. Gruppo di età per trattamento, sottotipo genetico per trattamento).
    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)
    Le scale numeriche di valutazione saranno fornite ai pazienti al basale al primo giorno di trattamento e alla fine del period di trattamento.
    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 concerned2
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months8
    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 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: 40
    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
    Prader-Willy patients may not give their consent personnaly because under guardianship measures
    Pazienti affetti dalla sindrome di Prader-Willy non possono dare il loro consenso e quindi sono sotto tutela legale
    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 the legal guardian or the subject assisted by his/her legal representative, as appropriate. Safety data will be collected during this time period.
    Dopo l'analisi dei dati dello studio in cieco se saranno evidenziati dei benefici clinici, AZP-531 sarà messo a disposizione dei soggetti appartenenti al braccio del placebo per 14 giorni. Il consenso informato deve essere ottenuto dal tutore legale o dal soggetto assistito dal suo / sua rappresentante legale, a seconda dei casi. I dati di sicurezza saranno raccolti durante questo periodo di tempo.
    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 Decision2015-08-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-07-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-01-14
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