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    Summary
    EudraCT Number:2013-000743-33
    Sponsor's Protocol Code Number:SPD602-204
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-11-25
    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 number2013-000743-33
    A.3Full title of the trial
    A 48-week, Open-label, 2-arm, Parallel-group, Randomized Exploratory Study to Assess Liver Iron Concentration Measured by FerriScan® (R2) Magnetic Resonance Imaging in β-thalassemia Subjects Administered SPD602 (SSP-004184AQ) or Exjade® (deferasirox) for Treatment of Chronic Transfusional Iron Overload
    Studio esplorativo, randomizzato, in aperto, a gruppi paralleli, a 2 bracci, della durata di 48 settimane per valutare la concentrazione epatica di ferro utilizzando la risonanza magnetica per immagini FerriScan® (R2) in soggetti affetti da β-talassemia ai quali è stato somministrato SPD602 (SSP-004184AQ) o Exjade®(deferasirox) per il trattamento del sovraccarico di ferro trasfusionale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of Iron Overload Requiring Chelation Therapy
    Trattamento del sovraccarico di ferro per il quale è richiesta una terapia chelante
    A.4.1Sponsor's protocol code numberSPD602-204
    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 SponsorShire Development LLC and International Affiliates
    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 supportShire Development LLC and International Affiliates
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Development LLC and International Affiliates
    B.5.2Functional name of contact pointLaura Rosen
    B.5.3 Address:
    B.5.3.1Street Address725 Chesterbrook Boulevard
    B.5.3.2Town/ cityWayne, PA
    B.5.3.3Post code19087
    B.5.3.4CountryUnited States
    B.5.6E-maillrosen@shire.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/647
    D.3 Description of the IMP
    D.3.1Product nameSSP-004184AQ 50 mg Capsule
    D.3.2Product code SSP-004184AQ 50 mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferitazole
    D.3.9.1CAS number 1173095-59-5
    D.3.9.2Current sponsor codeSSP-004184AQ
    D.3.9.3Other descriptive name(S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Exjade
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/02/092
    D.3 Description of the IMP
    D.3.1Product nameExjade
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferasirox
    D.3.9.1CAS number 201530-41-8
    D.3.9.3Other descriptive name[4-[(3Z,5E)-3,5-bis(6-oxo-1-cyclohexa-2,4-dienylidene)-1,2,4-triazolidin-1-yl]benzoic acid
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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.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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/647
    D.3 Description of the IMP
    D.3.1Product nameSSP-004184AQ 100 mg Capsule
    D.3.2Product code SSP-004184AQ 100 mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferitazole
    D.3.9.1CAS number 1173095-59-5
    D.3.9.2Current sponsor codeSSP-004184AQ
    D.3.9.3Other descriptive name(S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.IMP: 4
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/647
    D.3 Description of the IMP
    D.3.1Product nameSSP-004184AQ 200 mg Capsule
    D.3.2Product code SSP-004184AQ 200 mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferitazole
    D.3.9.1CAS number 1173095-59-5
    D.3.9.2Current sponsor codeSSP-004184AQ
    D.3.9.3Other descriptive name(S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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.IMP: 5
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/647
    D.3 Description of the IMP
    D.3.1Product nameSSP-004184AQ 375 mg Capsule
    D.3.2Product code SSP-004184AQ 375 mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferitazole
    D.3.9.1CAS number 1173095-59-5
    D.3.9.2Current sponsor codeSSP-004184AQ
    D.3.9.3Other descriptive name(S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number375
    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.IMP: 6
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/647
    D.3 Description of the IMP
    D.3.1Product nameSSP-004184AQ 500 mg Capsule
    D.3.2Product code SSP-004184AQ 500 mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferitazole
    D.3.9.1CAS number 1173095-59-5
    D.3.9.2Current sponsor codeSSP-004184AQ
    D.3.9.3Other descriptive name(S)-4,5-dihydro-2[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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.IMP: 7
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Exjade
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/02/092
    D.3 Description of the IMP
    D.3.1Product nameExjade
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferasirox
    D.3.9.1CAS number 201530-41-8
    D.3.9.3Other descriptive name[4-[(3Z,5E)-3,5-bis(6-oxo-1-cyclohexa-2,4-dienylidene)-1,2,4-triazolidin-1-yl]benzoic acid
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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.IMP: 8
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Exjade
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/02/092
    D.3 Description of the IMP
    D.3.1Product nameExjade
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNdeferasirox
    D.3.9.1CAS number 201530-41-8
    D.3.9.3Other descriptive name[4-[(3Z,5E)-3,5-bis(6-oxo-1-cyclohexa-2,4-dienylidene)-1,2,4-triazolidin-1-yl]benzoic acid
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients 18 years of age and older with transfusional iron overload due to β-thalassemia
    Pazienti di almeno 18 anni di età e oltre che presentano un sovraccarico di ferro trasfusionale dovuto a β-talassemia
    E.1.1.1Medical condition in easily understood language
    Patients with β-thalassemia that have received long-term blood transfusion therapy leading to an overload of iron.
    Pazienti affetti da β-talassemia che hanno ricevuto una terapia trasfusionale di sangue a lungo termine che ha portato ad un sovraccarico di ferro
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10065974
    E.1.2Term Chronic iron overload
    E.1.2System Organ Class 100000004861
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. Provide a descriptive assessment of change in LIC from baseline
    utilizing FerriScan® (R2) MRI in subjects with transfusional iron
    overload receiving SSP-004184AQ or deferasirox whose primary
    diagnosis is β-thalassemia.
    2. To evaluate the response rate over time in subjects with transfusional
    iron overload receiving SSP-004184AQ or deferasirox whose primary
    diagnosis is β-thalassemia.
    3. To assess the change in CIC measured by T2* MRI from baseline in
    subjects receiving SSP-004184AQ or deferasirox.
    4. To assess the change in serum ferritin levels from baseline in subjects
    receiving SSP-004184AQ or deferasirox.
    5. To provide a descriptive assessment of change in LIC from baseline
    utilizing R2* MRI in subjects with transfusional iron overload receiving
    SSP-004184AQ or deferasirox whose primary diagnosis is β-
    Thalassemia.
    6. To provide a descriptive assessment of change in PIC as measured by
    R2* MRI from baseline in subjects receiving SSP-004184AQ or
    deferasirox
    1.Fornire valutazione descrittiva della variazione dal basale della LIC, con la RMI FerriScan®(R2), in soggetti con sovraccarico di ferro trasfusionale riceventi SSP-004184AQ o deferasirox, con diagnosi primaria di -talassemia.
    2.Valutare tasso di risposta nel tempo in soggetti con sovraccarico di ferro trasfusionale riceventi SSP-004184AQ o deferasirox, con diagnosi primaria di -talassemia.
    3.Valutare variazione dal basale della CIC misurata con RMI T2* in soggetti riceventi SSP-004184AQ o deferasirox.
    4.Valutare variazione dal basale dei livelli di ferritina sierica in soggetti riceventi SSP-004184AQ o deferasirox.
    5.Fornire valutazione descrittiva della variazione dal basale della LIC con la RMI R2* in soggetti con sovraccarico di ferro trasfusionale riceventi SSP-004184AQ o deferasirox, con diagnosi primaria di -talassemia.
    6.Fornire valutazione descrittiva della variazione dal basale della PIC misurata con la RMI R2* in soggetti riceventiSSP-004184AQ o deferasirox.
    E.2.2Secondary objectives of the trial
    Safety

    1. To assess the safety and tolerability of SSP-004184AQ and deferasirox when administered for 48 weeks based upon assessment of LVEF via MRI, TEAEs, clinical laboratory assessments, vital signs, ECGs, Gastrointestinal Symptom Rating Scale, renal function assessments, and the TNSn©.

    2. To describe the exposure/response relationship between various safety parameters and SSP-004184AQ concentrations following administration of SSP-004184AQ.

    3. To explore the potential for early safety markers of kidney function.


    Health Economics Outcomes Research Objectives

    1. To assess Health-Related Quality of Life for subjects treated with SSP-004184AQ and those treated with deferasirox based on a subject self-report of the EQ-5D-5L.
    Sicurezza:
    1.Valutare la sicurezza e la tollerabilità di SSP-004184AQ e deferasirox dopo 48 settimane di somministrazione tramite la misurazione della LVEF tramite RMI, TEAEs, le analisi cliniche di laboratorio, i segni vitali, ECGs, la scala di valutazione dei sintomi gastrointestinali, gli esami relativi alla funzionalità renale e il TNSn©.
    2.Descrivere la relazione esposizione/risposta tra i diversi parametri di sicurezza e le concentrazioni di SSP-004184AQ dopo la somministrazione di quest’ultimo.
    3.Analizzare la presenza di potenziali marcatori precoci di sicurezza relativa alla funzionalità renale.
    Obiettivo di ricerca sugli esiti di economia sanitaria:
    1.Valutare la qualità della vita correlata alla salute (Health-Related Quality of Life, HRQoL) nei soggetti sottoposti al trattamento con SSP-004184AQ e in quelli trattati con deferasirox con un’autovalutazione tramite EQ-5D-5L del soggetto.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. An understanding, ability, and willingness to fully comply with study procedures and restrictions.

    2. Ability to voluntarily provide written, signed, and dated (personally or via a legally-authorized representative) informed consent as applicable to participate in the study before completing any study-related procedures.

    3. Subjects 18 years of age or older at the time of signing consent.

    4. Female subjects should be one of the following:
    a. Post-menopausal (12 consecutive months of spontaneous amenorrhea)
    b. Surgically sterile, or
    c. Females of child-bearing potential must have a negative serum -HCG pregnancy test at the Screening Visit (Visit 1) and a negative urine pregnancy test at the Baseline Visit (Visit 3). Females of child-bearing potential must agree to abstain from sexual activity that could result in pregnancy or agree to use acceptable methods of contraception.

    5. Subjects with β-thalassemia who have received at least 100mL/kg of packed red blood cells (or >20 transfusion units) and who have iron overload (transfusional hemosiderosis) requiring chronic treatment with an iron chelator.

    6. Serum ferritin >500μg/L at the Screening Visit (Visit 1).

    7. Baseline LIC (last MRI assessment prior to Day 1) ≥2.0mg and <30.0mg iron per g (equivalent dry weight, liver) determined by FerriScan® R2 MRI.

    8. Mean of the previous 3 pre-transfusion hemoglobin concentrations ≥7.5g/dL assessed at the Screening Visit (Visit 1) (1 value from clinical laboratory tests taken at the screening Visit [Visit 1] and the previous 2 historical values available).
    Il soggetto presenta comprensione, capacità e volontà di attenersi totalmente alle procedure e alle limitazioni previste dallo studio.
    2. Il soggetto è in grado di fornire volontariamente, per iscritto, un consenso informato firmato e datato (personalmente o tramite un rappresentante legalmente autorizzato) alla partecipazione allo studio prima di portare a termine qualsiasi procedura prevista dallo studio stesso.
    3. Il soggetto ha almeno 18 anni di età al momento della firma del consenso.
    4. I soggetti di sesso femminile devono rientrare in una delle seguenti categorie:
    a. Essere in post-menopausa (12 mesi consecutivi di amenorrea spontanea)
    b. Essere chirurgicamente sterili o
    c. I soggetti di sesso femminile potenzialmente fertili devono presentare un test di gravidanza su siero negativo β-HCG alla Visita di Screening (Visita 1) e un test di gravidanza sulle urine negativo alla Visita Basale (Visita 3). I soggetti di sesso femminile potenzialmente fertili devono acconsentire ad astenersi dall’attività sessuale che potrebbe risultare in una gravidanza o accettare di utilizzare metodi contraccettivi accettabili.
    5. Soggetti affetti da β-talassemia che hanno ricevuto almeno 100 ml/kg di globuli rossi concentrati (o >20 unità di trasfusione) e che presentano sovraccarico di ferro (emosiderosi trasfusionale) che necessitano di un trattamento cronico con un chelante del ferro.
    6. Ferritina sierica >500 µg/l alla Visita di Screening (Visita 1).
    7. LIC basale (ultima misurazione tramite RMI prima del Giorno 1) 2,0 mg e <30,0 mg di ferro per ogni g (peso secco equivalente, fegato) determinata tramite RMI FerriScan® R2.
    8. Media delle 3 concentrazioni di emoglobina pre-trasfusione 7,5 g/dl misurata alla Visita di Screening (Visita 1) (1 valore ricavato dalle analisi cliniche di laboratorio della Visita di Screening [Visita 1] e i 2 valori anamnestici precedenti disponibili).
    E.4Principal exclusion criteria
    1. Severe iron overload including: (a) cardiac T2* MRI <10.0ms; or (b) LIC by FerriScan® R2 MRI ≥30.0mg/g liver (dw) as assessed at the Screening Visit (Visit 1).

    2. Iron overload from causes other than transfusional siderosis.

    3. Subjects with thalassemia intermedia

    4. Unable to undergo MRI assessment.

    5. Cardiac LVEF <50% at baseline testing by MRI

    6. Subjects with documented liver failure (presence of portal hypertension, hepatic edema, ascites, cirrhosis), Child-Pugh C hepatic impairment, or biliary disorder.

    7. Platelet count <100 x 109/L at the Screening Visit (Visit 1).

    8. Absolute neutrophils counts of <1500mm3 at the Screening Visit (Visit 1).

    9. Evidence of renal insufficiency eg, creatinine clearance <60mL/min or serum creatinine >1.5x ULN at the Screening Visit (Visit 1).

    10. Clinically significant laboratory assessments at the Screening Visit (Visit 1).

    11. Significant proteinuria: urinary protein/creatinine ratio >1.0 in a non-first void urine sample at the Screening Visit (Visit 1).

    12. Current or recurrent disease that could affect the action, absorption, or disposition of the investigational product, or clinical or laboratory assessments.

    13. Current or relevant history of physical or psychiatric illness, any medical disorder that may require treatment or make the subject unlikely to fully complete the study, or any condition, including pregnancy, that presents undue risk from the investigational product or procedures.

    14. Current use of any medication contraindicated in the deferasirox prescribing information/SmPC. See prescribing information/SmPC for a list of prohibited and restricted medications.

    15. Known or suspected intolerance or hypersensitivity to SSP-004184AQ, deferasirox,closely-related compounds, or any of the stated ingredients in either medication.

    16. Known history of alcohol or other substance abuse within the last year.

    17. Within 30 days prior to the Baseline Visit (Visit 3):
    - Have used an investigational product
    - Have been enrolled in a clinical study (including vaccine studies) that, in the investigator’s opinion, may impact this Shire-sponsored study

    18. History of malignancy within the past 5 years, with the exception of basal cell or squamous cell skin carcinoma or cervical carcinoma in situ or completely resected colon carcinoma in situ.

    19. Insufficient venous access that precludes prescribed blood draws for safety laboratory assessments.

    20. Pregnant or lactating females.
    1. Grave sovraccarico di ferro che comprende: (a) RMI T2* cardiaca <10,0 ms; o (b) LIC utilizzando la RMI FerriScan® R2 30,0 mg/g del fegato (dw) come è stato misurato durante la Visita di Screening (Visita 1).
    2. Sovraccarico di ferro per cause differenti dalla siderosi trasfusionale.
    3. I soggetti affetti da talassemia intermedia.
    4. I soggetti che non possono essere sottoposti a valutazione con RMI.
    5. Frazione di eiezione del ventricolo sinistro di <50% al test al basale tramite RMI.
    6. I soggetti con insufficienza epatica documentata (presenza di ipertensione portale, edema epatico, ascite, cirrosi), insufficienza epatica di classe Child-Pugh C o disturbo biliare.
    7. Conta piastrinica <100 x 109/l alla Visita di Screening (Visita 1).
    8. Conta assoluta dei neutrofili <1500 mm3 alla Visita di Screening (Visita 1).
    9. Evidenza di insufficienza renale come ad es., clearance della creatinina <60 ml/min oppure creatinina sierica >1,5x il limite superiore dell’intervallo normale (upper limit of normal, ULN) alla Visita di Screening (Visita 1).
    10. Valutazioni di laboratorio clinicamente significative alla Visita di Screening (Visita 1).
    11. Proteinuria significativa: rapporto proteine/creatinina nelle urine >1,0 in un campione di urina che non era il primo del mattino alla Visita di Screening (Visita 1).
    12. Patologia attuale o ricorrente che potrebbe pregiudicare l’azione, l’assorbimento o la disponibilità del prodotto sperimentale o le valutazioni cliniche o di laboratorio.
    13. Anamnesi attuale o rilevante di patologie fisiche o psichiatriche, eventuali disturbi medici che rendono necessario un trattamento o che molto probabilmente impediscono al soggetto di completare lo studio, oppure qualsiasi condizione, compresa la gravidanza che risulti eccessivamente a rischio per il prodotto sperimentale o per le procedure.
    14. Utilizzo attuale di qualsiasi farmaco controindicato nel foglietto informativo/RCP di deferasirox. Vedasi il foglietto illustrativo/RCP per la lista dei farmaci proibiti e soggetti a limitazioni.
    15. Intolleranza nota o sospetta oppure ipersensibilità a SSP-004184AQ, a deferasirox, a composti strettamente correlati o a uno degli ingredienti dichiarati per ciascun farmaco.
    16. Anamnesi di abuso di alcol o altre sostanze stupefacenti nel corso dell’ultimo anno.
    17. Nei 30 giorni precedenti alla Visita Basale (Visita 3):
    - I soggetti che hanno usato un prodotto sperimentale
    - I soggetti che sono stati arruolati in uno studio clinico (compresi gli studi sui vaccini) che, a giudizio dello sperimentatore, potrebbero influire sul presente studio sponsorizzato da Shire.
    18. Anamnesi di neoplasia nel corso degli ultimi 5 anni, ad eccezione del carcinoma cutaneo a cellule squamose o a cellule basali o del carcinoma cervicale in situ oppure del carcinoma al colon in situ completamente resecato.
    19. Accesso venoso insufficiente che preclude i prelievi di sangue prescritti per le valutazioni di laboratorio di sicurezza.
    20. Donne in stato di gravidanza o allattamento.
    E.5 End points
    E.5.1Primary end point(s)
    • Change from baseline in Liver Iron Concentration (LIC) as assessed by FerriScan® R2 at Weeks 12, 24, and 48.
    • Rate of response defined according to the criteria listed in the protocol assessed by FerriScan® R2 at Weeks 12, 24, and 48.
    • Change from baseline in Cardiac Iron Concentration (CIC) as assessed by T2* MRI at Weeks 12, 24, and 48
    • Change from baseline in serum ferritin at Weeks 12, 24, and 48.
    • Change from baseline in LIC as assessed by R2* MRI at Weeks 12, 24, and 48.
    • Change from baseline in Pancreatic Iron Concentration (PIC) as assessed by R2* MRI at Weeks 12, 24, and 48.
    • Treatment-emergent AEs graded and recorded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v4.0
    • Vital signs measurements at Week 48
    • Clinical laboratory tests (chemistry, hematology, and urinalysis) at Weeks, 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48.
    • 12-lead electrocardiogram at Weeks 12, 24, and 48
    • Left ventricular ejection fraction via MRI at Weeks 12, 24, and 48
    • Renal function assessments (serum creatinine and estimated glomerular filtration rate) at Weeks 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48
    • Gastrointestinal Symptom Rating Scale at Weeks 2, 4, 8, 12, 24, 36, and 48.
    • Total Neuropathy Score nurse© at weeks 4, 12, 24, 36, and 48.
    • Change from baseline in Health-Related Quality of Life assessment (based on subject self report of the EQ-5D-5L) at Weeks 12, 24, and 48 or at discontinuation.
    • Variazione dal basale della LIC determinata tramite FerriScan® R2 alle Settimane 12, 24 e 48.
    • Tasso di risposta (definito in base ai criteri elencati di seguito) determinato tramite FerriScan® R2 alle Settimane 12, 24, e 48.
    • Variazione dal basale della CIC determinata tramite RMI T2* alle Settimane 12, 24 e 48.
    • Variazione dal basale della ferritina sierica alle Settimane 12, 24 e 48.
    • Variazione dal basale della LIC determinata tramite RMI R2* alle Settimane 12, 24 e 48.
    • Variazione dal basale della concentrazione pancreatica del ferro determinata tramite RMI T2* alle Settimane 12, 24 e 48.
    • Eventi avversi emergenti dal trattamento, valutati e registrati in base alla versione 4.0 dei Criteri terminologici comuni per gli eventi avversi dell’Istituto nazionale statunitense per il cancro [National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE)]
    • Misurazioni dei segni vitali alla Settimana 48
    • Analisi cliniche di laboratorio (esami ematochimici e analisi delle urine) alle Settimane 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, e 48
    • Elettrocardiogramma a 12 derivazioni alle Settimane 12, 24 e 48
    • Frazione di eiezione ventricolare sinistra misurata tramite RMI alle Settimane 12, 24 e 48
    • Valutazioni della funzionalità renale (creatinina sierica e tasso stimato di filtrazione glomerulare) alle Settimane 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, e 48
    • Scala di valutazione dei sintomi gastrointestinali alle Settimane 2, 4, 8, 12, 24, 36, e 48.
    • TNSn© allo screening e alle Settimane 4, 12, 24, 36, e 48.
    • Variazione dal basale nella valutazione della qualità della vita correlata alla salute (in base all’auto-segnalazione tramite EQ-5D-5L) alle Settimane 12, 24 e 48 o al momento del ritiro.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Timepoints included above
    tempi di rilevazioni inclusi sopra
    E.5.2Secondary end point(s)
    Not Applicable
    non applicabile
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not Applicable
    non applicabile
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    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 No
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Canada
    Egypt
    France
    Greece
    Israel
    Italy
    Lebanon
    Thailand
    United Kingdom
    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 years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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: 49
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 27
    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)
    Subjects participating in this study may be offered the opportunity to participate in a safety extension study where subjects will receive treatment with SSP-004184AQ. Subjects who are not eligible or choose not to participate will be treated according to standard of care as recommended by the subject's physician.
    ai soggetti partecipanti a questo studio può essere offerta la possibilità di partecipare ad uno studio di estensione sulla sicurezza in cui i soggetti riceveranno il trattamento con SSP-004184AQ. I soggetti che non sono arruolabili o che decidono di non partecipare saranno trattati secondo la normale pratica come raccomandato dal medico del soggetto.
    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 Decision2014-01-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-28
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2014-07-15
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