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    Summary
    EudraCT Number:2018-002911-80
    Sponsor's Protocol Code Number:LOCHNES
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-05-04
    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 number2018-002911-80
    A.3Full title of the trial
    Open-label study to evaluate the safety, tolerability, and efficacy of LOmitapide for the treatment of patients with Familial CHylomicroNEmia Syndrome
    Studio in aperto per valutare sicurezza, tollerabilità ed efficacia di Lomitapide per il trattamento di pazienti affetti da Sindrome Chilomicronemica Familiare
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate the safety, tolerability, and efficacy of LOmitapide for the treatment of patients with Familial CHylomicroNEmia Syndrome
    Studio per valutare sicurezza, tollerabilità ed efficacia di Lomitapide per il trattamento di pazienti affetti da Sindrome Chilomicronemica Familiare
    A.3.2Name or abbreviated title of the trial where available
    LOCHNES
    LOCHNES
    A.4.1Sponsor's protocol code numberLOCHNES
    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 SponsorAZIENDA OSPEDALIERA UNIVERSITARIA POLICLINICO "PAOLO GIACCONE" DI PALERMO
    B.1.3.4Country
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAmryt Pharma
    B.4.2Country
    B.4.1Name of organisation providing supportAmryt Pharmaceuticals DAC
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Trial Consulting s.s. di Aquilani e Scala
    B.5.2Functional name of contact pointRegulatory Approval Department
    B.5.3 Address:
    B.5.3.1Street AddressVia Innocenzo XI, 7
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00165
    B.5.4Telephone number0699704831
    B.5.5Fax number0699704962
    B.5.6E-maile.scala@clinicaltrialconsulting.it
    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 Lojuxta
    D.2.1.1.2Name of the Marketing Authorisation holderAmryt Pharmaceuticals DAC
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameLojuxta
    D.3.2Product code [C10AX12 Codice ATC]
    D.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Lojuxta
    D.2.1.1.2Name of the Marketing Authorisation holderAmryt Pharmaceuticals DAC
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameLojuxta
    D.3.2Product code [C10AX12 - Codice ATC]
    D.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 Lojuxta
    D.2.1.1.2Name of the Marketing Authorisation holderAmryt Pharmaceuticals DAC
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameLojuxta
    D.3.2Product code [C10AX12 Codice ATC]
    D.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Familial Chylomicronemia Syndrome
    Sindrome Chilomicronemica Familiare
    E.1.1.1Medical condition in easily understood language
    Familial hypercholesterolemia is a rare inherited disease that causes high levels of cholesterol in the blood and serious cardiovascular consequences.
    L'ipercolesterolemia familiare è una rara malattia ereditaria che causa elevati livelli di colesterolo nel sangue e gravi conseguenze cardiovascolari.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10058108
    E.1.2Term Dyslipidaemia
    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
    The primary objective of the study is to evaluate the efficacy of Lomitapide in combination with other lipid lowering therapy in patients with Familial Chylomicronemia Syndrome (FCS).
    L'obiettivo primario dello studio è valutare l'efficacia di Lomitapide in combinazione con altre terapie ipolipemizzanti in pazienti affetti da Sindrome Chilomicronemica Familiare (SCF).
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are to evaluate:
    1. other lipid parameters
    2. long-term safety of Lomitapide
    3. hepatic fat and liver stiffness and Chylomicron kinetics in combination with other lipid lowering agents in patients with FCS during the full length of the clinical study.
    Gli obiettivi secondari dello studio sono di valutare:
    1. altri parametri lipidici
    2. sicurezza a lungo termine di Lomitapide
    3. grasso epatico e rigidità epatica e cinetica dei chilomicroni in combinazione con altri agenti ipolipemizzanti in pazienti con FCS durante l'intera durata dello studio clinico.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent
    2. Age = 18 years at time of informed consent
    3. History of chylomicronemia as evidenced by documentation of lactescent serum or documentation of fasting TG measurement = 885 mg/dl (10 mmol/L)
    4. A diagnosis of Familial Chylomicronemia Syndrome by documentation of at least one of the following: confirmed homozygote, compound heterozygote or double heterozygote for known loss-of-function mutations in genes causing FCS
    5. Fasting TG = 750 mg/dl (8.4 mmol/L) at screening
    6. History of pancreatitis
    7. Willing to follow a diet comprising = 20 g fat per day during the study con quanto riportato a pag. 21 “Screening procedures include also dietary counseling with regard to low-fat diet (=20% energy from fat) and to avoid alcohol completely while receiving Lomitapide
    8. Satisfy one of the following:
    • Females: non-pregnant and non-lactating; surgically sterile, post-menopausal, abstinent, or if engaged in sexual relations of child-bearing potential, patient is using an acceptable contraceptive method
    • Males: Surgically sterile, abstinent or if engaged in sexual relations with a female of child-bearing potential, patient is utilizing an acceptable contraceptive method
    1. Consenso informato scritto
    2. Età = 18 anni al momento del consenso informato
    3. Storia di chilomicronemia documentata dall’evidenza di siero lattescente o TG a digiuno = 885 mg/dl (10 mmol/L)
    4. Diagnosi di Sindrome Chilomicronemica Familiare documentata con le seguenti modalità: pazienti omozigoti, eterozigoti composti o doppi eterozigoti per mutazioni con perdita di funzione nei geni candidati noti responsabili di FCS
    5. TG a digiuno = 750 mg/dl (8.4 mmol/L) al momento dello screening
    6. Storia di pancreatiti
    7. Disponibili a seguire una dieta con contenuto giornaliero di energia da grassi = 20% durante lo studio
    8. Soddisfare uno dei seguenti requisiti:
    • Donne: non in stato di gravidanza o allattamento; chirurgicamente sterile, post-menopausa, in età fertile utilizzare una forma accettabile di contraccezione
    • Uomini: chirurgicamente sterili, utilizzare una forma accettabile di contraccezione
    E.4Principal exclusion criteria
    1. Diabetes mellitus with any of the following: newly diagnosed within 12 weeks of screening; HbA1c = 9.0% at screening; recent change in anti-diabetic pharmacotherapy; anticipated need to change dose or type of medication during the treatment period of the study; current use of GLP-1 agonists.
    2. Severe hypertriglyceridemia other than due to FCS.
    3. Active pancreatitis within 4 weeks prior to screening.
    4. History within 6 months of screening of acute or unstable cardiac ischemia, stroke, transient ischemic attack or unstable congestive cardiac failure requiring a change in medication or major surgery within 3 months of screening.
    5. Any of the following laboratory values at screening: total bilirubin > upper limit of normal (ULN); AST > 2.0 x ULN; ALT > 2.0 x ULN; persistently positive for protein on urine dipstick; persistently positive for blood or urine dipstick; estimated creatinine clearance calculated according to the formula of Cockcroft and Gault < 50 mL/min.
    6. Uncontrolled hypertension (BP > 160/100 mmHg).
    7. History of bleeding diathesis or coagulopathy or clinically significant abnormality in coagulation parameters at screening.
    8. History of heart failure with NYHA greater than Class II.
    9. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to begin treatment with Lomitapide (Baseline Visit).
    10. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B.
    11. Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
    12. Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer.
    13. Unwilling to comply with lifestyle requirements.
    14. Known lactose intolerance.
    15. Use of the following:
    • Statins, omega-3 fatty acids or fibrates unless on a stable dose for at least 3 months prior to screening and dose and regimen expected to remain constant during the treatment period. Patients taking omega-3 fatty acids should make every effort to remain on the same brand throughout the study
    • Nicotinic acid or derivatives of nicotinic acid within 4 weeks prior to screening
    • Systemic corticosteroids or anabolic steroids within 6 weeks prior to screening unless approved by the Investigator
    • Atypical antipsychotic medications unless on a stable dose for at least 4 weeks prior to screening and dose and regimen expected to remain constant during the treatment period
    • Glybera gene therapy within 2 years prior to screening
    • Oral anticoagulants (e.g., warfarin, dabigatran, rivaroxaban, and apixaban) unless on a stable dose for at least 4 weeks prior to screening and regular clinical monitoring is performed
    • Tamoxifen, estrogens or progestins unless on a stable dose for at least 4 months prior to screening and dose and regimen expected to remain constant during the treatment period
    • Plasma apheresis within 4 weeks prior to screening or planned during the study
    • Any of the medication unless stable at least 4 weeks prior to screening
    16. Blood donation of 50 to 499 mL within 30 days of screening or of > 499 mL within 60 days of screening
    17. Have any other conditions, which, in the opinion of the Investigators or the Sponsor would make the patient unsuitable for inclusion, or could interfere with the patient participating in or completing the study
    1. Diabete mellito se: recentemente diagnosticato (entro 12 settimane dalla visita di screening), HbA1c = 9,0% (alla visita di screening); variazioni recenti della terapia ipoglicemizzante; previsione di variazione della dose o tipo di farmaco durante il periodo di trattamento ; trattamento con agonisti del GLP-1.
    2. Ipertrigliceridemia severa diversa da FCS.
    3. Episodio di pancreatite acuta nelle 4 settimane che precedono la visita di screening.
    4. Nei 6 mesi che precedono lo screening: ischemia cardiaca acuta o instabile (infarto miocardico, sindrome coronarica acuta, angina di nuova insorgenza), ictus, attacco ischemico transitorio o insufficienza cardiaca congestizia instabile che richiede un cambiamento nel trattamento farmacologico o chirurgia maggiore entro 3 mesi dallo screening.
    5. Riscontro di uno dei seguenti valori di laboratorio durante lo screening
    • Fegato: bilirubina totale > limite superiore della norma (ULN), a meno di una diagnosi e/o documentazione precedente di sindrome di Gilbert; AST> 2,0 x ULN; ALT> 2,0 x ULN;
    • Renale: proteinuria persistente positiva al dipstick; ematuria persistente positiva al dipstick positiva; determinazione della clearance della creatinina calcolata secondo la formula di Cockcroft e Gault <50 ml/min.
    6. Ipertensione non controllata (BP> 160/100 mmHg)
    7. Storia di diatesi emorragica o coagulopatia o anomalie clinicamente significative nei parametri della coagulazione allo screening.
    8. Storia di scompenso cardiaco con NYHA superiore alla Classe II.
    9. Infezione attiva che richiede una terapia antivirale o antimicrobica sistemica che non sarà completata prima dell’inizio del trattamento con lomitapide (Baseline Visit).
    10. Anti-HCV e HBsAg positività, infezione da HIV
    11. Neoplasie diagnosticate nei 5 anni che precedono la vista di screening, ad eccezione del carcinoma basocellulare o squamoso della pelle o del carcinoma in situ della cervice trattato con successo.
    12. Trattamento con un altro farmaco sperimentale, agente biologico o dispositivo entro un mese dallo screening o 5 emivite dell'agente utilizzato per la sperimentazione.
    13. Riluttante per soddisfare i requisiti di stile di vita.
    14. Intolleranza nota al lattosio.
    15. Una delle seguenti condizioni:
    • Statine, acidi grassi omega-3 o fibrati a meno che non a dose stabile da almeno 3 mesi prima dello screening e si prevede rimangano costanti durante il periodo di trattamento.
    • Per i pazienti in trattamento con acidi grassi omega-3 è consigliabile l’utilizzo dello stesso marchio o formulazione durante lo studio
    • Acido nicotinico o derivati dell'acido nicotinico entro 4 settimane prima dello screening
    • Corticosteroidi sistemici o steroidi anabolizzanti entro 6 settimane prima dello screening, a meno che non siano approvati dallo sperimentatore
    • Farmaci antipsicotici atipici a meno che non a dose stabile per almeno 4 settimane prima dello screening e che si prevede rimangano costanti durante il periodo di trattamento
    • Terapia genica con Glybera nei 2 anni che precedono la visita di screening
    • Anticoagulanti orali (ad es. Warfarin, dabigatran, rivaroxaban e apixaban) a meno che non siano somministrati a dose stabile da almeno 4 settimane prima dello screening e venga eseguito un monitoraggio clinico regolare
    • Tamoxifene, estrogeni o progestinici a meno che non assumano una dose stabile da almeno 4 mesi prima dello screening e si prevede che rimangano costanti durante il periodo di trattamento
    • Plasmaferesi o plasma exchange entro 4 settimane prima dello screening o programmata durante lo studio
    • Qualsiasi farmaco a meno che non sia a dose stabile da almeno 4 settimane prima dello screening
    16. Donazione di sangue di 50-499 mL entro 30 giorni dallo screening o > 499 mL entro 60 giorni dallo screening
    17. Qualsiasi altra condizione che, secondo il parere degli Investigatori, renderebbero il paziente inadatto all'inclusione o potrebbero interferire con la partecipazione o completamento dello studio
    E.5 End points
    E.5.1Primary end point(s)
    Percent change in tryglicerides (TG) at the maximum tolerated dose compared to baseline after 26 weeks of treatment in combination with other lipid lowering therapy in patients with Familial Chylomicronemia Syndrome (FCS).
    Variazione percentuale dei trigliceridi tra il valore al basale e dopo 26 settimane di trattamento in combinazione con altre terapie ipolipemizzanti in pazienti affetti da Sindrome Chilomicronemica Familiare (SCF).
    E.5.1.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 settimane
    E.5.2Secondary end point(s)
    Other lipid parameters, hepatic fat and liver stiffness and Chylomicron kinetics of Lomitapide in combination with other lipid lowering agents in term of changes at 26 weeks from baseline.
    In particular we will assess:
    a) Percent change in TC, non-HDL-C, LDL-C, VLDL, Lp(a), as well as apolipoproteins B and A1.
    b) Safety of Lomitapide in patients with FCS assessed by changes in laboratory parameters, electrocardiogram, physical examinations and weight.
    c) Record of episodes of pancreatitis
    d) Change in hepatic fat liver stiffness measured by MRI and/or Transient Elastography (Fibroscan).
    e) Chylomicron kinetics.
    Altri parametri lipidici, rigidità del fegato dovuta alla componente grassa e la cinetica dei Chilomicroni di Lomitapide in combinazione con altri agenti ipolipemizzanti in termini di cambiamenti a 26 settimane dal basale.
    In particolare sarà valutato:
    a) Variazione percentuale di colesterolo totale, non-HDL, LDL, VLDL, Lp(a), apolipoproteine B e A1
    b) Sicurezza di Lomitapide in pazienti affetti da SCF determinata in base alle modifiche dei parametri di laboratorio, ECG, esame obiettivo e peso
    c) Registrazione degli episodi di pancreatite
    d) Variazione della rigidità del fegato dovuta alla componente grassa, valutata mediante risonanza magnetica (RMI) e/o elastografia epatica (Fibroscan)
    e) Cinetica dei Chilomicroni
    E.5.2.1Timepoint(s) of evaluation of this end point
    26 weeks
    26 settimane
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Information not present in EudraCT
    E.6.2Prophylaxis Information not present in EudraCT
    E.6.3Therapy Information not present in EudraCT
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Information not present in EudraCT
    E.6.8Bioequivalence Information not present in EudraCT
    E.6.9Dose response Information not present in EudraCT
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    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 No
    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
    Studio in aperto
    Open Label
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States No
    E.8.5.1Number of sites anticipated in the EEA0
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Information not present in EudraCT
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months6
    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 months6
    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 Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 17
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Information not present in EudraCT
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception Yes
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    At the end of the study, patients will be treated according to normal clinical practice: controlled diet, Omega3 fatty acids, filtered and MCT oil integration.
    Al termine dello studio i pazienti verranno trattati secondo la normale pratica clinica: regime alimentare controllato, acidi grassi Omega3, filtrati e integrazione olio MCT.
    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 Decision2018-12-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-15
    P. End of Trial
    P.End of Trial StatusOngoing
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