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    Summary
    EudraCT Number:2011-004096-36
    Sponsor's Protocol Code Number:THALI-HHT
    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:2012-03-19
    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 number2011-004096-36
    A.3Full title of the trial
    Efficacy of thalidomide in the treatment of severe recurrent epistaxis in hereditary hemorrhagic telangiectasia (HHT)
    Efficacia di talidomide nel trattamento dell'epistassi severa ricorrente nella teleangectasia emorragica ereditaria (HHT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy of thalidomide in the treatment of heavy and frequent nose bleeding in patients affected by hereditary hemorrhagic telangiectasia
    Efficacia della terapia medica a base di talidomide nel trattamento del sanguinamento nasale abbondante e frequente in pazienti affetti da teleangectasia emorragica ereditaria
    A.4.1Sponsor's protocol code numberTHALI-HHT
    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 SponsorOSPEDALE POLICLINICO S. MATTEO
    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 supportCelgene S.r.L.
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione IRCCS Policlinico San Matteo
    B.5.2Functional name of contact pointStruttura di Medicina Generale III
    B.5.3 Address:
    B.5.3.1Street AddressV.le Golgi 19
    B.5.3.2Town/ cityPavia
    B.5.3.3Post code27100
    B.5.3.4CountryItaly
    B.5.4Telephone number0382 502169
    B.5.5Fax number0382 502508
    B.5.6E-mailr.invernizzi@smatteo.pv.it
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name THALIDOMIDE CELGENE*28CPS 50MG
    D.2.1.1.2Name of the Marketing Authorisation holderCELGENE Srl
    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 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 INNTHALIDOMIDE
    D.3.9.1CAS number 50-35-1
    D.3.9.4EV Substance CodeSUB10958MIG
    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.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
    Severe recurrent epistaxis in hereditary hemorrhagic telangiectasia
    Epistassi severa ricorrente nella teleangectasia emorragica ereditaria
    E.1.1.1Medical condition in easily understood language
    Heavy and frequent nose bleeding in hereditary hemorrhagic telangiectasia
    Sanguinamento nasale abbondante e frequente nella teleangectasia emorragica ereditaria
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10038554
    E.1.2Term Rendu-Osler-Weber syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the clinical effects of thalidomide therapy on the severity of epistaxis in subjects with HHT who are refractory to standard therapies.
    Valutare gli effetti della terapia con talidomide sulla severità dell’epistassi in soggetti con HHT refrattari ai trattamenti standard.
    E.2.2Secondary objectives of the trial
    - Safety and Tolerability - To ascertain whether thalidomide reduces telangiectasias in nasal mucosa - To assess specific biological and clinical parameters predictable for patients response and side effects profile.
    - Sicurezza e tollerabilità del farmaco - Valutare se la talidomide riduce le teleangiectasie a livello della mucosa nasale - Valutare specifici parametri biologici e clinici predittivi di risposta alla terapia e comparsa di effetti collaterali
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    OTHER SUBSTUDIES:
    Molecular biology studies - Appendix 4 of Study Protocol (ver 1 - 11/08/2011)

    ALTRI SOTTOSTUDI:
    Studi di biolgia molecolare - Appendice 4 del protocollo di studio (ver 1 - 11/08/2011)

    E.3Principal inclusion criteria
    - Diagnosis of HHT, according to the diagnostic criteria world-wide recognized (Curacao criteria, with severe recurrent epistaxis (grade 2-3) and refractory to mini-invasive surgical procedures. - Age > 18 years - Ability of signing written informed consent - Women of childbearing potential: declared intention not to start a pregnancy throughout the study and for four weeks following the date of the last dose of thalidomide, negative serum pregnancy test obtained within 48 hours prior to the first dose of Thalidomide, declared intention to undergo pregnancy tests periodically while on the study medication - Males with female partner of childbearing potential: declared intention not to father throughout the study and for one week following the date of the last dose of thalidomide - Estimated life expectancy must be greater than 10 months
    - Diagnosi di HHT, secondo i criteri Curacao, in presenza di epistassi severe ricorrenti (grado 2-3), refrattarie alle procedure standard di chirurgia mini-invasiva - Età &gt; 18 anni - Capacità di fornire consenso informato scritto - Per le donne in età fertile: dichiarata intenzione di non intraprendere una gravidanza durante il periodo di trattamento e nel mese successivo alla sospensione del farmaco; negatività del test di gravidanza effettuato nelle 48 ore precedenti l’inizio della terapia; disponibilità ad effettuare controlli del test di gravidanza durante lo studio - Per gli uomini: dichiarata intenzione di adottare misure contraccettive ogniqualvolta abbiano un rapporto sessuale con una donna potenzialmente fertile, durante il periodo di trattamento e nella settimana successiva alla sospensione del farmaco - Aspettativa di vita superiore a 10 mesi.
    E.4Principal exclusion criteria
    - Pregnant or lactating women, or potentially fertile (both males and females) who have not agreed to avoid pregnancy during the trial period and for four weeks (females) or one week (males) following the date of the last dose of thalidomide - Neurological diseases - Psychiatric illness that would prevent granting of informed consent - Active cardiovascular disease - High risk for thromboembolic events - Patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption since thalidomide capsules contain lactose
    - Donne in gravidanza o in allattamento o donne (e uomini) potenzialmente fertili che non accettano di evitare il concepimento durante il trattamento o nelle quattro settimane (donne) o nella settimana (uomini) successiva alla sospensione della terapia - Patologie neurologiche - Patologie psichiatriche che compromettono la capacità di fornire il consenso informato. - Patologie cardiovascolari attive - Alto rischio di eventi tromboembolici - Intolleranza al galattosio, deficit di Lapp lattasi o malassorbimento glucosio-galattosio
    E.5 End points
    E.5.1Primary end point(s)
    Percentage of patients showing a decrease in the frequency, intensity and duration of epistaxis and in the blood transfusion requirement.
    Percentuale di pazienti che mostrano una riduzione nella frequenza, intensità e durata dell'epistassi e nella richiesta di trasfusioni.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Not applicable
    Non applicabile
    E.5.2Secondary end point(s)
    - Size and number of telangiectasias evaluated by endoscopy of nasal mucosa at baseline and at least every 8 weeks thereafter - Requirement of blood transfusions - Minimum dose of the drug that reduces bleeding - Time to Response - Time to relapse after the end of treatment - Safety and tolerability of thalidomide in HHT - Correlations between the mutations that are responsible for HHT and response to treatment - Correlations between polymorphisms of CYP2C19 and response to treatment - Correlations between polymorphisms of CYP2C19 and side effects profile
    - Numero e dimensioni delle telengectasie, valutate mediante endoscopia della mucosa nasale al basale e dopo 8 settimane di trattamento - Richiesta di trasfusione - Minima dose che riduce il sanguinamento - Tempo di risposta - Tempo di ricaduta dopo la fine del trattamento - Sicurezza e tollerabilità della talidomide - Correlazione tra mutazioni responsabili dell’HHT e risposta al trattamento - Correlazione tra polimorfismo del CYP2C19 e risposta al trattamento - Correlazione tra polimorfismo del CYP2C19 ed effetti collaterali
    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 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 Information not present in EudraCT
    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 Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned 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: 11
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state31
    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)
    Not applicable
    Non applicabile
    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 Decision2011-10-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-08-29
    P. End of Trial
    P.End of Trial StatusCompleted
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