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   42891   clinical trials with a EudraCT protocol, of which   7066   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

    EudraCT Number:2018-004179-11
    Sponsor's Protocol Code Number:2018-4898
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-08-17
    Trial results
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-004179-11
    A.3Full title of the trial
    Effectiveness of Somatostatin Analogues in Patients with hereditary hemorrhagic
    telangiectasia and symptomatic gastrointestinal bleeding, SAIPAN-trial: a multicentre,
    randomized, open-label, parallel-group, superiority trial.
    Efficacia degli analoghi della somatostatina in pazienti con teleangiectasia emorragica
    ereditaria e sanguinamento digestivo sintomatico, SAIPAN-trial: studio multicentrico,
    randomizzato, open-label, a gruppi paralleli, trial di superiorità
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effectiveness of somatostatin analogues for GI bleeding in patients with hereditary
    hemorrhagic telangiectasia
    Efficacia degli analoghi della somatostatina nei sanguinamenti digestivi in pazienti
    con teleangiectasia emorragica ereditaria
    A.3.2Name or abbreviated title of the trial where available
    SAIPAN study
    Studio SAIPAN
    A.4.1Sponsor's protocol code number2018-4898
    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.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 supportZonMw
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRadboudumc
    B.5.2Functional name of contact pointServizio Informazione sperimentazio
    B.5.3 Address:
    B.5.3.1Street AddressPostbus 9101
    B.5.3.2Town/ cityNijmegen
    B.5.3.3Post code6500HB
    B.5.4Telephone number+310248187313
    B.5.5Fax number+310243635129
    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.3.1Product nameoctreotide a azione pronta 0,1 mg
    D.3.2Product code [OCTREOTIDE ACETATO]
    D.3.4Pharmaceutical form Concentrate for solution for injection/infusion
    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.3.9.1CAS number 83150-76-9
    D.3.9.2Current sponsor codeOPR00000697
    D.3.9.4EV Substance CodeSUB09417MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product 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
    Hereditary hemorrhagic telangiectasia (HHT), also known as Osler–Weber–Rendu disease
    (in specific patients with gastrointestinal bleeding and transfusion dependency)
    Teleangiectasia emorragica ereditaria HHT anche conosciuta come s. di rendu Osler
    Weber , e che causa sanguinamento cronico digestivo in una parte degli affetti con
    elevato fabbisogno trasfusionale
    E.1.1.1Medical condition in easily understood language
    Patients suffering from HHT/Rendu-Osler-Weber disease and bleeding from the
    Pazienti con HHT e sanguinamento cronico dal tubo digerente
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10019883
    E.1.2Term Hereditary haemorrhagic telangiectasia
    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 investigate the effectiveness of somatostatin analogues in decreasing the
    transfusion requirements in patients with HHT and GI bleeding who are refractory to
    endoscopic therapy.
    Studiare l'efficacia degli analoghi della somatostatina nel ridurre il fabbisogno
    trasfusionale in pazienti HHT con sanguinamento GI refrattari alla terapia
    E.2.2Secondary objectives of the trial
    To investigate the effectiveness of octreotide on: decreasing the endoscopic treatment
    frequency, increasing the health-related quality of life, decreasing fatigue and
    epistaxis symptoms, and to assess the effects of long-term octreotide on adverse
    events and cost- effectiveness.
    Studiare l'efficacia dell'octreotide su: diminuire la frequenza del trattamento
    endoscopico, aumentare la qualità della vita , ridurre l'affaticamento e i sintomi
    dell'epistassi e valutare gli effetti dell'octreotide a lungo termine sugli eventi
    avversi e sull'efficacia economica.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    · Patients older than 18 years with written informed consent.
    · Diagnosis of HHT: either confirmed by genetic testing or the Curaçao criteria
    (definite diagnosis)
    · Presence of endoscopic proven GI AVM manifestations / telangiectasias confirmed
    within the last 12 months (upper and/or lower endoscopy and/or capsule endoscopy).
    · Endoscopic refractory: at least 1 endoscopic APC, laser, or other endoscopic
    treatment modality performed in the past 5 years.
    · Substantial transfusion dependency: at least 4 blood units and / or intravenous
    iron in the 6 months prior to study inclusion with:
    o At least one serum ferritin below < 30 ug/l within the last 6 months requiring iron
    infusion above or equal to 1 g and/or
    o Haemoglobin below 5.6 mmol/l (9.0 g/dl) or are in need of transfusions due to anaemia
    related symptoms within the last 6 months requiring blood transfusion above.
    · Pazienti di età superiore ai 18 anni con consenso informato scritto.
    · Diagnosi di HHT: confermata da test genetici o dai criteri curaçao (diagnosi
    · Presenza di manifestazioni endoscopiche comprovate gi AVM / telangiectasie
    confermate negli ultimi 12 mesi (endoscopia superiore e/o inferiore e/o endoscopia
    · Refrattarietà all' endoscopia: almeno 1 APC endoscopico, laser, o altre modalità di
    trattamento endoscopico eseguite negli ultimi 5 anni.
    · Dipendenza trasfusionale sostanziale: almeno 4 unità ematiche e/o ferro endovenoso
    nei 6 mesi precedenti l'inclusione dello studio con:
    o Almeno una ferritina sierica inferiore < 30 ug/l negli ultimi 6 mesi che richiedono
    infusione di ferro superiore o uguale a 1 g e/o
    o Emoglobina inferiore a 5,6 mmol/l (9,0 g/dl) o richiesta di trasfusioni a causa di
    sintomi correlati all'anemia negli ultimi 6 mesi che richiedono trasfusioni di sangue
    E.4Principal exclusion criteria
    · Liver cirrhosis Child-Pugh C,
    · Insuloma,
    · Uncontrolled diabetes mellitus as defined by HbA1c >64 mmol/ml, despite adequate
    · Juvenile Polyposis Syndrome
    · Symptomatic cholecystolithiasis (possible side-effect octreotide)
    · Cirrosi epatica CHILD C
    · Insulinoma,
    diabete scompensato
    poliposi giovanile
    colecistolitiasi sintomatica
    E.5 End points
    E.5.1Primary end point(s)
    ‘Successful response’, defined as a decrease of =50% in the amount of units
    intravenous iron and/or blood transfusions given between the number of transfusions at
    baseline (26 weeks prior to study inclusion) and during the treatment study period (26
    NB Patients included based on intravenous iron requirements and needing bloodtransfusion
    during the study period are treatment failures.
    diminuzione del =50% nella quantità di unità di ferro e/o trasfusioni di sangue
    endovenose somministrate tra il numero di trasfusioni al basale (26 settimane prima
    dell'inclusione dello studio) e durante il periodo di studio del trattamento (26
    I pazienti inclusi in base alla necessità di infusione di ferro per via endovenosa e
    che necessitano di trasfusioni di sangue durante il periodo di studio sono considerati
    fallimenti del trattamento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    26 weeks prior to study inclusion and during the treatment study period (26 weeks)
    26 settimane prima dell'inclusione e durante il trattamento di 26 settimane
    E.5.2Secondary end point(s)
    The percentual or mean/median difference between the half year prior to inclusion and
    the treatment period of a half year between the treatment and observational arm in:
    - blood and intravenous iron requirements
    - number of endoscopic treatments
    - cost-effectiveness (see economic evaluation)
    The percentual or mean/median difference between the value at baseline and after a
    half year between the treatment and observational arm in:
    - PROMs: quality of life (SF-36), level of fatigue (multidimensional fatigue inventory
    (MFI)-20), epistaxis severity (ESS tool), and patient satisfaction
    The percentual or mean/median difference after a half year between the treatment and
    observational arm in:
    - (S)AE.
    The percentual or mean/median difference between the value at baseline, after 4 weeks,
    12 weeks, and after a half year between the treatment and observational arm in:
    - hemoglobin and ferritin levels
    La differenza percentuale o media/mediana tra il semestre precedente l'inclusione e il
    periodo di trattamento di un semestre tra il trattamento e il braccio osservazionale
    - fabbisogno di sangue e ferro per via endovenosa
    - numero di trattamenti endoscopici
    - rapporto costo/efficacia (cfr. valutazione economica)
    La differenza percentuale o media/mediana tra il valore al basale e dopo un semestre
    tra il trattamento e il braccio osservazionale in:
    - PROM: qualità della vita (SF-36), livello di affaticamento (inventario della fatica
    multidimensionale (IFM)-20), gravità dell'epistassi (strumento ESS) e soddisfazione
    del paziente
    La differenza percentuale o media/mediana dopo un semestre tra il trattamento e il
    braccio osservazionale in:
    - (S)AE.
    La differenza percentuale o media/mediana tra il valore al basale, dopo 4 settimane,
    12 settimane e dopo un semestre tra il trattamento e il braccio osservazionale in:
    - livelli di emoglobina e ferritina
    E.5.2.1Timepoint(s) of evaluation of this end point
    0 weeks, 4 weeks, 12 weeks, 26 weeks, 30 weeks (follow up)
    0-4-12-26-30 settimane
    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 No
    E.6.5Efficacy No
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled 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. description
    standard di cura
    standard of care
    E.8.2.4Number of treatment arms in the trial2
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA5
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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.1.1.1In 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: 20
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 33
    F.4.2.2In the whole clinical trial 38
    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)
    patients will be followed up according to specific surveillance and treatment plans of
    i pazienti proseguiranno la sorveglianza con trattamenti necessari secondo la presa in
    carico specifica per HHT
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation NESSUNO
    G.4.3.4Network Country Italy
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-09-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-21
    P. End of Trial
    P.End of Trial StatusOngoing
    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