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    Summary
    EudraCT Number:2017-002713-58
    Sponsor's Protocol Code Number:HZNP-TEP-302
    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:2019-01-15
    Trial results View 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 number2017-002713-58
    A.3Full title of the trial
    Multicenter, Safety and Efficacy, Open-Label Extension Study Evaluating Teprotumumab (HZN-001) Treatment in Subjects with Thyroid Eye Disease
    Studio multicentrico di estensione in aperto, sulla sicurezza ed efficacia, per valutare il trattamento con Teprotumumab (HZN-001) in soggetti con oftalmopatia tiroidea
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of Graves` Orbitopathy to Reduce Proptosis with Teprotumumab Infusions in an Open-Label Clinical Extension Study
    Trattamento dell’orbitopatia di Graves per ridurre l’esoftalmo con infusioni di Teprotumumab in uno studio clinico di estensione in aperto
    A.3.2Name or abbreviated title of the trial where available
    OPTIC-X
    OPTIC-X
    A.4.1Sponsor's protocol code numberHZNP-TEP-302
    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 SponsorHORIZON PHARMA USA, 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 supportHorizon Pharma USA, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHorizon Pharma USA, Inc.
    B.5.2Functional name of contact pointSenior Medical Director
    B.5.3 Address:
    B.5.3.1Street Address150 S. Saunders Road
    B.5.3.2Town/ cityLake Forest
    B.5.3.3Post codeIL 60045
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 224 383 3123
    B.5.5Fax number0
    B.5.6E-mailtvescio@horizonpharma.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 nameTeprotumumab
    D.3.2Product code HZN-001
    D.3.4Pharmaceutical form Powder and solvent for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTeprotumumab
    D.3.9.1CAS number 89957-37-9
    D.3.9.2Current sponsor codeHZN-001
    D.3.9.3Other descriptive nameTEPROTUMUMAB
    D.3.9.4EV Substance CodeSUB169240
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number48 to 50
    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 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 Yes
    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
    Active Thyroid Eye Disease (TED)
    Oftalmopatia tiroidea (thyroid eye disease, TED) attiva
    E.1.1.1Medical condition in easily understood language
    Graves' ophthalmopathy
    Oftalmopatia di Graves
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10057889
    E.1.2Term Graves' ophthalmopathy
    E.1.2System Organ Class 100000004853
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the effect of teprotumumab on the proptosis responder rate (i.e., the percentage of subjects with a ≥ 2 mm reduction from Baseline in the study eye without deterioration [≥ 2 mm increase] of proptosis in the fellow eye) at the EOT Visit.
    L’obiettivo primario è valutare l’effetto di teprotumumab sul tasso di soggetti responsivi alla terapia per l’esoftalmo (ovvero, la percentuale di soggetti con riduzione ≥2 mm rispetto al Basale nell’occhio oggetto di studio senza peggioramento [aumento ≥2 mm] dell’esoftalmo nell’altro occhio) alla Visita di fine trattamento (EOT).
    E.2.2Secondary objectives of the trial
    Secondary objectives will evaluate the effect of teprotumumab on the following:
    1. Percentage of subjects with a Clinical Activity Score (CAS) value of 0 or 1 at the EOT Visit in the study eye.
    2. Mean change from Baseline to the EOT Visit in proptosis measurement in the study eye.
    3. Mean change from Baseline to the EOT Visit in the Graves'Ophthalmopathy Quality of Life (GO-QoL) questionnaire overall score.
    Gli obiettivi secondari consisteranno nella valutazione dell’effetto di teprotumumab sui seguenti esiti:
    1. Percentuale di soggetti con valore di Clinical Activity Score (CAS) di 0 o 1 alla Visita EOT nell’occhio oggetto di studio.
    2. Variazione media dal Basale alla Visita EOT nella misurazione dell’esoftalmo a livello dell’occhio oggetto di studio.
    3. Variazione media dal Basale alla Visita EOT nel punteggio complessivo del Questionario per misurare la qualità della vita nei pazienti con oftalmopatia di Graves (GO-QoL).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent.
    2. Completed the 24-week double-maskedTreatment Period in Study HZNP-TEP-301.
    3. Proptosis non-responder (< 2 mm reduction in proptosis in the study eye) at Week 24 ofStudy HZNP-TEP-301 OR proptosis responder at Week 24 who relapses (see Section 9.3.4)during the Follow-Up Period of Study HZNP-TEP-301.
    4. Subject must be euthyroid with the baseline disease under control, or have mild hypo- or hyperthyroidism (defined as free thyroxine [FT4] and free triiodothyronine [FT3] levels < 50% above or below the normal limits) at the most recent clinic visit.Every effort should be made to correct the mild hypo-or hyperthyroidism promptly and to maintain the euthyroid state for the full duration of the clinical trial.
    5. Alanine aminotransferase (ALT) or aspartate aminotransferase(AST) ≤3 times the upper limit of normal (ULN) or serum creatinine <1.5 times the ULN (according to age) at the most recent clinic visit.
    6. Diabetic subjects must have well-controlled disease (defined as HgbA1c < 9.0%at most recent clinic visit).
    7. Does not require immediate surgical ophthalmological interventionand is not planning corrective surgery/irradiation during the course of the study.
    8. Women of childbearing potentialmust have a negative urine pregnancy testat Baseline/Day 1and agree to use 2 reliable forms of contraception during the trialand continue for 180 days after the last dose of study drug.One of the 2 forms of contraceptionis recommended to be hormonal, such as an oral contraceptive.Hormonal contraception must be in use for at least one full cycle prior to Baseline. Highly effective contraceptive methods (with a failure rate less than 1% per year), when used consistently and correctly, includes implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomised partner.
    9. Male subjects must be surgically sterile or must agree to use a barrier contraceptive method from Baseline through 180days after the last dose of study drug.
    10. Subject is willing and able to comply with the prescribed treatment protocol and evaluations for the duration of the study.
    11. Has not received any treatment for TED since Week 24 of the HZNP-TEP-301 study.
    1. Consenso informato scritto.
    2. Completamento del Periodo di trattamento in doppio cieco di 24 settimane nell’ambito dello studio HZNP-TEP-301.
    3. Soggetto non responsivo alla terapia per l’esoftalmo (riduzione <2 mm dell’esoftalmo nell’occhio in studio) alla Settimana 24 dello studio HZNP-TEP-301 OPPURE soggetto responsivo alla terapia per l’esoftalmo alla Settimana 24 che manifesta recidiva (vedere Sezione 9.3.4) durante il Periodo di follow-up dello studio HZNP-TEP-301.
    4. Il soggetto deve essere eutiroideo con malattia sotto controllo al Basale, oppure presentare ipo o ipertiroidismo lieve (definito come livelli di tiroxina libera [FT4] e triiodiotironina libera [FT3] <50% al di sopra o al di sotto dei limiti normali) alla visita in clinica più recente. Deve essere compiuto ogni sforzo per correggere tempestivamente l’ipo o l’ipertiroidismo lieve e mantenere lo stato eutiroideo per tutta la durata della sperimentazione clinica.
    5. Alanina aminotransferasi (ALT) o aspartato aminotransferasi (AST) ≤3 volte il limite superiore della normalità (ULN) o creatinina sierica <1,5 volte l’ULN (in base all’età) alla visita in clinica più recente.
    6. I soggetti diabetici devono presentare malattia ben controllata (definita come emoglobina [Hb] A1C <9,0% alla visita in clinica più recente).
    7. Il soggetto non richiede un intervento chirurgico oculistico immediato né prevede di sottoporsi a chirurgia correttiva/radioterapia nel corso dello studio.
    8. Le donne in età fertile devono disporre di un test di gravidanza sulle urine negativo al Basale/Giorno 1 e acconsentire all’uso di 2 metodi contraccettivi affidabili durante lo sperimentazione, da proseguire per 180 giorni dopo l’ultima dose di farmaco dello studio. Si raccomanda che uno dei 2 metodi contraccettivi sia ormonale, per es. un contraccettivo orale. La contraccezione ormonale deve essere in uso da almeno un ciclo completo prima del Basale. I metodi contraccettivi altamente efficaci (con tasso di insuccesso annuale inferiore all’1%), se utilizzati regolarmente e correttamente includono impianti, contraccettivi iniettabili, contraccettivi orali combinati, alcuni dispositivi intrauterini [IUD], l’astinenza sessuale o la vasectomia del partner.
    9. I soggetti di sesso maschile devono essere chirurgicamente sterili o acconsentire all’uso di un metodo contraccettivo di barriera dal Basale fino a 180 giorni dopo l’ultima dose di farmaco dello studio.
    10. Il soggetto deve essere disposto e in grado di rispettare il protocollo di trattamento e le valutazioni prescritti per la durata dello studio.
    11. Nessun trattamento per TED dalla Settimana 24 dello studio HZNP-TEP-301.
    E.4Principal exclusion criteria
    Subjects will be ineligible if, in the opinion of the Investigator, they are unlikely to comply with the study protocol or have a concomitant disease or condition that could interfere with the conduct of the study or potentially put the subject at unacceptable risk.
    I soggetti saranno considerati non idonei se, a giudizio dello sperimentatore, è improbabile che possano rispettare il protocollo dello studio oppure se presentano una malattia o una condizione concomitante che potrebbe interferire con la conduzione dello studio o comportare un rischio inaccettabile per il soggetto.
    E.5 End points
    E.5.1Primary end point(s)
    The primary outcome measure is the proptosis responder rate (percentage of subjects with a ≥ 2 mm reduction from Baseline in proptosis in the study eye, without deterioration [≥ 2 mm increase] of proptosis in the fellow eye) at the EOT Visit.
    La misura dell’outcome primario è il tasso di soggetti responsivi alla terapia per l’esoftalmo (percentuale di soggetti con riduzione ≥2 mm dell’esoftalmo rispetto al Basale nell’occhio oggetto di studio senza peggioramento [aumento ≥2 mm] dell’esoftalmo nell’altro occhio) alla Visita EOT.
    E.5.1.1Timepoint(s) of evaluation of this end point
    at End of Trial visit
    alla Visita di fine studio
    E.5.2Secondary end point(s)
    1. Percentage of subjects with a CAS value of 0 or 1 in the study eye at the EOT Visit.
    2. Mean change from Baseline to EOT in proptosis measurement in the study eye.
    3. Mean change from Baseline to EOT in the GO-QoL questionnaire overall score.
    1. Percentuale di soggetti con valore del CAS pari a 0 o 1 nell’occhio oggetto di studio alla Visita EOT.
    2. Variazione media dal Basale alla Visita EOT nella misurazione dell’esoftalmo a livello dell’occhio oggetto di studio.
    3. Variazione media dal Basale alla Visita EOT nel punteggio complessivo del questionario GO-QoL.
    E.5.2.1Timepoint(s) of evaluation of this end point
    at End of Trial visit
    alla Visita di fine studio
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Germany
    Italy
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    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.1.1Number of subjects for this age range: 1
    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: 44
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 50
    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)
    post trial treatment is based on standard of care.
    Il trattamento post studio è basato sullo standard di cura.
    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 Decision2018-07-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-03
    P. End of Trial
    P.End of Trial StatusCompleted
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