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    Summary
    EudraCT Number:2011-000973-30
    Sponsor's Protocol Code Number:AMD-THAL
    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:2011-12-12
    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 number2011-000973-30
    A.3Full title of the trial
    PLERIXAFOR MOBILIZED STEM CELLS AS SOURCE FOR GENE THERAPY OF BETA-THALASSEMIA AMD-THAL .
    Cellule staminali mobilizzate mediante Plerixafor come fonte per la terapia genica della beta-talassemia.
    A.4.1Sponsor's protocol code numberAMD-THAL
    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 SponsorFONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNplerixafor
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.3Concentration number20
    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 typemobilizzatore di cellule staminali
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNplerixafor
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.3Concentration number20
    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 typemobilizzatore di cellule staminali
    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
    transfusion dependent beta thalassemia
    beta talassemia trasfusione dipendente
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level PT
    E.1.2Classification code 10043391
    E.1.2Term Thalassaemia beta
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. To explore the safety of Plerixafor administration to patients affected by transfusion dependent betathalassemia Efficacy 2. To explore the efficacy of Plerixafor in mobilizing CD34+ cells in patients affected by transfusion dependent beta-thalassemia
    Sicurezza espressa come valutazione dell’incidenza e della severita' degli eventi avversi (qualsiasi grado) insorti tra il giorno 0 e il giorno +30 dopo somministrazione del Plerixafor. Efficacia espressa come numero di cellule CD34+ per chilogrammo di peso corporeo raccolte (superiore a 5x10^6/kg in almeno 3 pazienti su 6).
    E.2.2Secondary objectives of the trial
    1. To explore the efficiency of transduction of Plerixafor mobilized CD34+ cells and Plerixafor primed BM cells from patients with beta-thalassemia using a lentiviral vector encoding for human beta globin 2. To evaluate yield of CD34+ cells harvest from BM following administration of Plerixafor (primed-BM). 3. To characterize Plerixafor mobilized CD34+ cells from thalassemic patients.
    1.esplorare l'efficienza di trasduzione di plerixafor nel mobilizzare cellule CD34 + e cellule BM inescate da plerixafor da pazienti affetti da beta-talassemia utilizzando una codifica lentivirale come vettore di beta globine umane 2.valutare la resa del raccolto di cellule CD34 + da BM dopo la somministrazione di plerixafor (primer-BM). 3. caratterizzare le cellule CD34 + cellule di pazienti talassemici mobilizzate con plerixafor
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Transfusion-dependent beta-thalassemia (any genotype) • = 18 years • Karnofsky Index > 80 % • Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by: o LVEF greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease o DLCO diffusing capacity > 50% and FEV1 and FVC > 60% predicted o Serum creatinine < 1.5 upper limit normal o Absent-mild-moderate liver iron overload on T2*MRI (less than 12 months before enrolment) o Absent-mild-moderate cardiac iron overload T2*MRI (less than 12 Sample size months before enrolment) o absence of severe fibrosis or cirrhosis on fibroscan or liver biopsy (less than 12 months before enrolment) • Negative thrombophilic screen and negative history for previous thrombotic events • For all patients, agreement to use highly effective and adequate method of contraception while receiving treatment and for at least 3 months following drug administration (including both females of child bearing potential and males with partners of child bearing potential) • Good adherence to transfusion and chelation programme • Written informed consent.
    Diagnosi di beta-thalassemia trasfusione dipendente (qualsiasi genotipo ammesso) - Eta' &gt; 18 anni -Performance status sec. Karnofsky &gt; 80 -Adeguata funzionalita' cardiaca, renale, epatica e polmonare -Screening trombofilico negativo e storia clinica negativa per eventi trombotici -Buona adesione al programma trasfusionale e ferrochelante -Consenso informato scritto
    E.4Principal exclusion criteria
    Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents) • Symptomatic viral, bacterial, or fungal infection within 6 weeks of eligibility evaluation or active infection (included fever of unknown origin) • Neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or history of “familial” cancer • Myelodysplasia or other serious hematological disorder than thalassemia • History of uncontrolled seizures • Other systemic disease judged non compatible with the procedure • Positivity for HIV and/or HCV RNA and/or HbsAg and/or HBV DNA • Active alcohol or substance abuse within 6 months of the study • Pregnancy or lactation.
    utilizzo di altri farmaci in sperimentazione nelle 4 settimane precedenti lo studio (entro 6 settimane se viene fatto uso di agenti ad azione prolungata) infezione virale sintomatica, batterica o fungina entro 6 settimane di valutazione di ammissibilita' o in fase attiva infezione (inclusa febbre di origine sconosciuta) neoplasia (eccetto il tumore della pelle localizzato o la neoplasia intraepiteliale cervicale)o storia ''familiare'' di tumori mielodisplasia o altri gravi disordini ematologici di talassemia precedenti di convulsioni non controllate altre malattie sistemiche giudicate non compatibili con la procedura •positivita' per l'HIV e / o RNA HCV e / o HBsAg e / o di HBV DNA abuso di alcool o altre sostanze entro 6 mesi dello studio gravidanza o allattamento.
    E.5 End points
    E.5.1Primary end point(s)
    Safety 1.Incidence and severity of adverse events following Plerixafor administration (any grade) occurring between day 0 and day 30. Efficacy 2.Harvest of > 5x10^6 CD34/kg body weight in at least 3 out of 6 patients.
    The proportion of patients who achieve >2x10^6 CD34+ cells/Kg and >5x10^6 CD34+ cells/Kg body weight after 1 apheresis • The proportion of patients who achieve >2x10^6 CD34+ cells/Kg and >5x10^6 CD34+ cells/Kg body weight after 2 apheresis • The increase in the number of circulating CD34+ cells 4 hours after the first dose of Plerixafor • The increase in the number of CD34+ cells in AMD-primed BM
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.3.1Comparator description
    ogni paziente risulta controllo di se stesso
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    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 months12
    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 months12
    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.1.3Elderly (>=65 years) Yes
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 6
    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 Decision2011-04-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-04-07
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2012-09-30
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