Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   42559   clinical trials with a EudraCT protocol, of which   7007   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2011-005675-16
    Sponsor's Protocol Code Number:FBS0701-CTP-16
    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:2012-08-02
    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 number2011-005675-16
    A.3Full title of the trial
    A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation Therapy
    Studio clinico multicentrico di fase 2, della durata di 24 settimana randomizzato, in aperto, volto a valutare la sicurezza, la tollerabilita', la farmacocinetica e la farmacodinamica di due dosi di FBS0701 nel trattamento del sovraccarico di ferro cronico che richiede una terapia chelante.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of chronic iron overload with a chelation therapy (FBS0701)
    Trattamento del sovraccarico di ferro con una terapia chelante (FBS0701)
    A.3.2Name or abbreviated title of the trial where available
    FBS0701 in the treatment of chronic iron overload
    FBS0701 nel trattamento del sovraccarico di ferro
    A.4.1Sponsor's protocol code numberFBS0701-CTP-16
    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 SponsorFERROKIN BIOSCIENCES 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 supportFerrokin Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFerrokin Bioscience Ltd
    B.5.2Functional name of contact pointClinical Research Dpt
    B.5.3 Address:
    B.5.3.1Street Address311,Shoreham Street
    B.5.3.2Town/ citySheffield
    B.5.3.3Post codeS2 4FA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44 (0) 7780 113652
    B.5.5Fax numberN/A
    B.5.6E-mailamber.jones@ferrokin.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 nameFBS0701 CAPSULE
    D.3.2Product code FBS0701
    D.3.4Pharmaceutical form Capsule, hard
    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 INNiron chelating agents
    D.3.9.1CAS number 1173092-59-5
    D.3.9.2Current sponsor codeFBS0701
    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 Yes
    D.3.11.13.1Other medicinal product typeA novel Iron Chelator, FBS0701 is a magnesium salt of a desferrithiocin analogue,(S)-3-(OH)-DADFT-PE
    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 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 nameFBS0701 CAPSULE
    D.3.2Product code FBS0701
    D.3.4Pharmaceutical form Capsule, hard
    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 INNIron chelating agents
    D.3.9.1CAS number 1173092-59-5
    D.3.9.2Current sponsor codeFBS0701
    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 Yes
    D.3.11.13.1Other medicinal product typea novel Iron Chelator,FBS0701 IS A MAGNESIUM SALT OF A DESFERRITHIOCIN ANALOGUE,(S)-3-(OH)-DADFT-PE
    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 nameFBS0701 CAPSULE
    D.3.2Product code FBS0701
    D.3.4Pharmaceutical form Capsule, hard
    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 INNIron chelating agents
    D.3.9.1CAS number 1173092-59-5
    D.3.9.2Current sponsor codeFBS0701
    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 Yes
    D.3.11.13.1Other medicinal product typeanovel Iron Chelator,FBS0701 is a magnesium salt of a desferrithiocin analogue,(S)-3-(OH)-DADFT-PE
    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 nameFBS0701 CAPSULE
    D.3.2Product code FBS0701
    D.3.4Pharmaceutical form Capsule, hard
    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 INNiron chelating agents
    D.3.9.1CAS number 1173092-59-5
    D.3.9.2Current sponsor codeFBS0701
    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 Yes
    D.3.11.13.1Other medicinal product typea novel Iron Chelator, FBS0701 is a magnesium salt of a desferrithiocin analogue,(S)-3(OH)-DADFT-PE
    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 nameFBS0701 CAPSULE
    D.3.2Product code FBS0701
    D.3.4Pharmaceutical form Capsule, hard
    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 INNiron chelating agents
    D.3.9.1CAS number 1173092-59-5
    D.3.9.2Current sponsor codeFBS0701
    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 Yes
    D.3.11.13.1Other medicinal product typea novel Iron Chelator,FBS0701 is a magnesium salt of desferrithiocin analogue,(S)-3-(OH)-DADFT-PE
    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 with transfusional iron overload, with the following primary diagnosis:hereditary anemia (such as sickle cell disease),β-thalassemia and Diamond Blackfan anemia
    pazienti adulti affetti da sovraccarico di ferro trasfusione-dipendente documentato,che presentano le seguenti diagnosi primarie: anemia ereditaria (ad esempio anemia falciforme), talassemia β e anemia di Diamond-Blackfan
    E.1.1.1Medical condition in easily understood language
    patients with transfusional iron overload
    pazienti adulti affetti da sovracarico di ferro trasfusione -dipendente
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluate, in patients with transfusion-dependent iron overload, whose primary diagnosis is hereditary or congenital anemia: - Pharmacokinetic parameters provided a constant (steady state) of two doses of FBS0701; - Changes in the concentrations of iron in the liver, after 12 and 24 weeks following administration of two doses of FBS0701; - The safety and tolerability of two doses of FBS0701, administered daily for 24 weeks.
    Valutare, in pazienti affetti da sovraccarico di ferro trasfusione-dipendente, la cui diagnosi primaria è anemia ereditaria o congenita: - i parametri farmacocinetici a condizione costante (steady state) di due dosaggi di FBS0701; - le modifiche delle concentrazioni di ferro nel fegato, dopo 12 e 24 settimane, a seguito della somministrazione di due dosaggi di FBS0701; - la sicurezza e la tollerabilità di due dosaggi di FBS0701, somministrate quotidianamente per 24 settimane.
    E.2.2Secondary objectives of the trial
    NA
    NA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The patient must be willing to sign the informed consent form approved and must be able to do so. 2. Age: 18-60 years at screening 3. Patients who, until then, had more than 20 transfusions and iron overload are affected by transfusion-dependent, requiring chronic treatment with a chelating agent of iron. Please note that Patients with sickle cell disease who carry out periodic exchange transfusions, and patients with iron overload with thalassemia intermedia are eligible to participate in the study. 4. Agree to discontinue any iron chelation therapy in progress, for a minimum period of one to five days before the first dose of FBS0701, for 24 weeks of study days and 1 week after the last dose, for a total of about 26 weeks. 5. Agree to stay on an empty stomach two hours before and one hour after each dose. 6. Present a serum ferritin> 500 ng / ml at screening. 7. The concentration of iron in the liver, the extent of reference (from Day -14 to Day -7) must be ≥ 5 mg of iron per g (dry weight equivalent) determined by MRI FerriScan. 8. The average concentrations of the three previous pre-transfusion hemoglobin must be ≥ 7.5 g / dl. 9. The patient agrees to use a contraceptive method approved by the time of screening up to 28 days after the last dose of study medication. Among the accepted methods of contraception are cited as follows: condoms, birth control pills approved, patches, implants or injections, vaginal diaphragm with spermicide, intrauterine device (IUD) and / or surgical sterilization (vasectomy or tubal ligation at least six months prior to administration of doses). Patients who practice abstinence must agree to use an approved method of contraception, if they become sexually active during the course of the study. All women will be fertile again subjected to pregnancy tests every month during the screening and for the duration of the study.
    1.Il paziente deve essere disposto a firmare il modulo di consenso informato approvato e deve essere in grado di farlo. 2.Età: 18-60 anni al momento dello Screening 3.Pazienti che, fino a quel momento, hanno effettuato più di 20 trasfusioni e sono affetti da sovraccarico di ferro trasfusione-dipendente, che richiede trattamento cronico con un agente chelante del ferro. N.B. I pazienti affetti da anemia falciforme che effettuano periodiche trasfusioni di scambio e pazienti affetti da sovraccarico di ferro con talassemia intermedia sono idonei a partecipare allo studio. 4.Accettare di interrompere ogni terapia chelante del ferro in corso, per un periodo minimo di uno-cinque giorni prima della prima dose di FBS0701, per le 24 settimane della durata dello studio e per 1 settimana dopo l’ultima dose, per un totale di circa 26 settimane. 5.Accettare di restare a digiuno due ore prima ed un’ora dopo ciascuna dose. 6.Presentare una ferritina serica &gt; 500 ng/ml al momento dello Screening. 7.La concentrazione del ferro nel fegato, nella misura di riferimento (dal Giorno -14 al Giorno -7) deve essere ≥ 5mg di ferro per g (peso a secco equivalente) determinata da RMI FerriScan. 8.La media delle tre concentrazioni precedenti di emoglobina pre-trasfusionale deve essere ≥ 7,5 g/dl. 9.Il paziente acconsente ad utilizzare un metodo contraccettivo approvato dal momento dello Screening fino a 28 giorni dopo l'ultima somministrazione del farmaco allo studio. Fra i metodi contraccettivi accettati si citano i seguenti: profilattico; pillole anticoncezionali approvate, cerotti, impianti o iniezioni; diaframma con spermicida vaginale; dispositivo intrauterino (spirale) e/o sterilizzazione chirurgica (vasectomia o legatura delle tube almeno sei mesi prima della somministrazione delle dosi). I pazienti che praticano l’astinenza devono acconsentire ad utilizzare un metodo contraccettivo approvato, nel caso in cui diventino sessualmente attivi durante lo svolgimento dello studio. Tutte le donne ancora fertili saranno sottoposte ogni mese a test di gravidanza durante lo Screening e per la durata dello studio.
    E.4Principal exclusion criteria
    1. Following clinical evaluation, physical examinations or investigations carried out during the screening, the principal investigator considered the patient unsuitable for participation in the study. 2. Non-elective hospitalization in the last 30 days before the exams reference. 3. Evidence of the presence of oral diseases, cardiovascular, gastrointestinal, hepatic, renal, endocrine, pulmonary, neurological, psychiatric, immunological, bone marrow or skin, clinically relevant, by the principal investigator confirmed that the administration of FBS0701 inadvisable. 4. Iron overload due to causes other than siderosis transfusion-dependent. 5. Found significant renal insufficiency, eg. serum creatinine greater than 1.5 the upper limit of normal, proteinuria greater than 1 g per day or calculated creatinine clearance less than 40 ml / minute. 6. Severe iron overload, including: a. Of MRI T2 *> 10 ms; b. liver iron concentration determined by R2 MRI of> 30 mg / g liver (dry weight). 7. Known sensitivity to magnesium stearate, sodium croscarmellose, or a FBS0701. 8. Platelet count less than 100,000 and / or absolute neutrophil count less than 1500/mm3 at the screening. 9. Poor venous access that precludes the possibility of blood draws for laboratory tests required for safety assessment 10. ALT at screening ≥ 200 IU / L. 11. Use of any investigational drug within 30 days preceding the baseline procedures. 12. Pregnant or breastfeeding
    1. A seguito della valutazione clinica, dell’esame obiettivo o delle indagini svolte nella fase di Screening, lo Sperimentatore principale ritiene il paziente non idoneo per partecipare allo studio. 2.Ricovero ospedaliero non elettivo negli ultimi 30 giorni prima di effettuare gli esami di riferimento. 3.Evidenza della presenza di disturbi orali, cardiovascolari, gastrointestinali, epatici, renali, endocrini, polmonari, neurologici, psichiatrici, immunologici, del midollo osseo o dermatologici, clinicamente rilevanti, accertati dallo Sperimentatore principale che sconsiglino la somministrazione di FBS0701. 4.Sovraccarico di ferro per cause diverse dalla siderosi trasfusione-dipendente. 5.Insufficienza renale significativa accertata, ad es. siero-creatinina superiore di 1,5 al limite massimo della norma, proteinuria superiore a 1 g al giorno o clearance della creatinina calcolata minore di 40 ml/minuto. 6.Grave sovraccarico di ferro, ivi compresi: a. RMI della T2* &gt; 10 ms; b.concentrazione di ferro nel fegato determinata con RMI della R2 &gt; 30 mg/g di fegato (peso a secco). 7.Sensibilità nota allo stearato di magnesio, al sodio di croscarmellose o a FBS0701. 8.Conta piastrinica inferiore a 100.000/µl e/o conta assoluta dei neutrofili inferiore a 1500/mm3 allo Screening. 9.Accesso venoso insufficiente che preclude la possibilità di effettuare i prelievi di sangue richiesti per gli esami di laboratorio per la valutazione della sicurezza 10.ALT allo Screening ≥ 200 IU/L. 11.Impiego di un qualsiasi farmaco sperimentale nei 30 giorni precedenti le procedure basali. 12.Donne in gravidanza o allattamento
    E.5 End points
    E.5.1Primary end point(s)
    - Pharmacokinetic parameters of FBS0701 after a period of 8-16 weeks of exposure to FBS0701 capsules of 50 mg and 75 mg / kg per day. -Change in liver iron concentration determined by magnetic resonance imaging (MRI) of two doses of FSB0701 after 12 and 24 weeks of administration -Safety and tolerability of two doses of FBS0701 based on clinical assessments (such as incidence and severity of adverse events, physical examinations including vital signs, clinical laboratory values ​​and ECG).
    -Parametri farmacocinetici di FBS0701 a seguito di un periodo di 8-16 settimane di esposizione a capsule di FBS0701 da 50 e 75 mg/kg al giorno. -Modifica della concentrazione di ferro nel fegato determinata con risonanza magnetica per immagini (RMI) di due dosaggi di FSB0701 dopo 12 e 24 settimane di somministrazione -Sicurezza e tollerabilità di due dosaggi di FBS0701 sulla base di valutazioni cliniche (quali incidenza e gravità di eventi avversi, esami obiettivi comprendenti parametri vitali, valori clinici di laboratorio ed ECG).
    E.5.1.1Timepoint(s) of evaluation of this end point
    NA
    NA
    E.5.2Secondary end point(s)
    NA
    NA
    E.5.2.1Timepoint(s) of evaluation of this end point
    NA
    NA
    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 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    - Stesso farmaco ad altro dosaggio
    - same IMP used at different dosage
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Canada
    Lebanon
    Turkey
    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
    The trial end date is determined as the closing date of the database
    La data di fine sperimentazione è stabilita come la data di chiusura del database
    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 months13
    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 months13
    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: 0
    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: 50
    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 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 21
    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)
    NA
    NA
    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 Decision2012-05-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-04-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2014-02-28
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2022 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA