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    Summary
    EudraCT Number:2018-004179-11
    Sponsor's Protocol Code Number:2018-4898
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-03-12
    Trial 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 ConcernedGermany - BfArM
    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,
    the SAIPAN-trial: a multicenter, randomized, open-label, parallel-group,
    superiority trial.
    Effektivität von Somatostatin Analoga bei Patienten mit hereditärer
    hämorrhagischer Teleangiektasie und symptomatischer
    gastrointestinaler Beteiligung, die SAIPAN Studie: eine multizentrische,
    randomisierte, open-label, Überlegenheitsstudie mit parallelen Gruppen
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The effectiveness of Octreotide in hereditary hemorrhagic telangiectasia
    (a.k.a. Rendu-Osler-Weber disease) patients who suffer from
    gastrointestinal bleeding.
    Effektivität von Octreotid bei Patienten mit hereditärer hämorrhagischer
    Teleangiektasie, die an Magen-Darm-Blutungen leiden.
    A.3.2Name or abbreviated title of the trial where available
    SAIPAN-trial
    SAIPAN Studie
    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.1.1Name of SponsorRadboudumc
    B.1.3.4CountryNetherlands
    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.4.2CountryNetherlands
    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 pointLia Goltstein
    B.5.3 Address:
    B.5.3.1Street AddressGeert Grooteplein Zuid 10
    B.5.3.2Town/ cityNijmegen
    B.5.3.3Post code6525
    B.5.3.4CountryNetherlands
    B.5.4Telephone number00312481817313
    B.5.5Fax number0031243635129
    B.5.6E-maillia.goltstein@radboudumc.nl
    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 AuthorisationGermany
    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.3.9.1CAS number 79517-01-4
    D.3.9.3Other descriptive nameOCTREOTIDE ACETATE
    D.3.9.4EV Substance CodeSUB03490MIG
    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
    Hereditary hemorrhagic telangiectasia (HHT), also known as Osler–
    Weber–Rendu disease (in specific patients with gastrointestinal
    bleedings)
    Morbus Osler (Patienten mit Morbus Osler und gastrointestinalen
    Blutungen)
    E.1.1.1Medical condition in easily understood language
    Patients suffering from HHT/Rendu-Osler-Weber disease and bleeding
    from the stomach/bowel
    Patienten mit Morbus Osler und Blutungen im Magen-Darm Bereich
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    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
    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.
    Untersuchung der Effektivität von Somatostatin Analoga hinsichtlich
    der Reduktion von Transfusionen bei Patienten mit HHT und
    gastrointenstinalen Blutungen, bei denen eine endoskopische
    Intervention nicht ausreichte
    E.2.2Secondary objectives of the trial
    Investigate the effectiveness of octreotide on: decreasing the
    endoscopic
    treatment frequency, increasing
    the quality of life, decreasing fatigue and epistaxis symptoms and
    costeffectiveness.
    Untersuchung der Effektivität von Somatostatin Analoga hinsichtlich
    der Reduktion der Häufigkeit gastronintestinaler endoskopiescher
    Interventionen, Verbesserung der Lebensqualität und Steigerung der
    Müdigkeit, Epistaxis-Symptomatik und Kosteneffektivität
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    In order to be eligible to participate in this study, a subject must meet all of the following criteria:
    • Patients older than 18 years with written informed consent.
    • Diagnosis of HHT: either confirmed by genetic testing or the Curaçao criteria (definite diagnosis) (7).
    • 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.
    • Diagnosis of iron deficiency anemia (IDA). IDA is defined as:
    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 Hemoglobin below 5.6 mmol/l (9.0 g/dl) or are in need of transfusions due to anemia related symptoms within the last 6 months and
    o The absence of other common causes of anemia (Vitamin B12 and/or folate deficiency in patients with macrocytic anemia and bone marrow disorders in patients with pancytopenia)
    • Substantial transfusion dependency: at least 4 blood units and / or intravenous iron in the 6 months prior to study inclusion.
    E.4Principal exclusion criteria
    A potential subject who meets any of the following criteria will be excluded from participation in this study:
    • Liver cirrhosis Child-Pugh C,
    • Insulinoma,
    • Uncontrolled diabetes mellitus as defined by HbA1c >64 mmol/ml, despite adequate therapy,
    • Juvenile Polyposis Syndrome
    • Symptomatic cholecystolithiasis (possible side-effect octreotide)
    Chronic or acute pancreatitis
    • Bradycardia (heart rate below 50)*
    • Hypersensitivity to the active ingredient (octreotide) or to auxiliary materials of the study medication
    • Severe disease/comorbidities with a life expectancy < 1 year
    • Use of chemotherapy and / or ciclosporin
    • Pregnancy or nursing women or women who have a pregnancy wish during the study period.
    • Women of childbearing potential who do not have a confirmed menstrual period and a negative, highly sensitive urine or serum pregnancy test < 7 days before inclusion
    • Women of childbearing potential who do not use a highly effective contraceptive measure (according to section 4.1 of the Recommendations related to contraception and pregnancy testing in clinical trials of the Heads of Medicines Agencies (HMA)) or refuse to maintain this measure during the entire treatment-and relevant systemic exposure period.(25)
    • Women of childbearing potential who refuse to undergo additional pregnancy testing during the treatment period
    • Use of other anti-angiogenic drug treatment (thalidomide and/or bevacizumab
    • Use of hormonal (estrogen) therapy and/or antifibrinolytic therapy (tranexamic acid) to treat anemia due to telangiectasia with sufficient effect. However, when patients are red blood cell transfusion or iron infusion dependent despite these treatment options and are naïve to octreotide treatment, patients are still eligible for inclusion.
    *If a patient has a heart rate below 60 and uses cardiovascular medication that affect the heart rate (e.g. beta blockers and calcium channel blockers) the prescribing specialist (or another competent specialist) will be consulted about the possibility to adjust the dose of these medicines. Patients with a heartrate below 50 (despite dose adjustments) will be excluded from participation.
    E.5 End points
    E.5.1Primary end point(s)
    The difference in the number of patients in the treatment arm and the number of patients in the observational arm with a ‘successful response’. A successful response is defined as a decrease of ≥50% in the number of red blood cell transfusions and/or number of IV iron infusions (per 500mg) given between baseline period (26 weeks prior to study inclusion) and during the treatment study period (26 weeks). Patients who do not have a successful response (patients with a decrease of <50%) will be counted as treatment failures.
    NB Patients who exclusively or primarily used IV iron infusions at baseline and required (more) red blood cell transfusions during the treatment study period will be counted as treatment failures regardless of their percentual decrease in IV iron infusions. Patients who exclusively or primarily used red blood cell transfusions at baseline and required (more) iron infusions during the treatment study period will be counted as treatment failures if their total number of red blood cell transfusions and iron infusions during the treatment study period exceeds half of the total number of red blood cell transfusions (and iron infusions) during baseline.
    NB The exact preparation and dosage of IV iron infusions should be noted for each infusion. The preparation of IV iron should be kept consistent at baseline and during study period.
    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 Wochen vor Studieneintritt und 26 Wochen währen der
    Medikamentenapplikation
    E.5.2Secondary end point(s)
    The absolute mean and median difference and the percentage mean and median difference between baseline (26 weeks prior to study inclusion) and the treatment study period (26 weeks) of patients in the treatment arm compared to patients in the observational arm in:
    - Number of red blood cell transfusions
    - Number of Intravenous iron infusions (per 500mg)
    - Number of endoscopic treatments
    The absolute mean and median difference and the percentage mean and median difference at baseline (< 7 days before inclusion) and after 4 weeks, 12 weeks, and 26 weeks of the study period between patients in the treatment arm and patients in the observational arm in the value of:
    - Hemoglobin and ferritin levels
    The absolute mean and median difference and the percentage mean and median difference between baseline (26 weeks prior to study inclusion) and the treatment study period (26 weeks) of patients in the treatment arm compared to patients in the observational arm in:
    - Patient reported outcome measures (PROMS): which include quality of life (measured by the SF-36), level of fatigue (measured by the multidimensional fatigue inventory (MFI)-20) and epistaxis severity (measured by the ESS tool).
    The absolute mean and median difference and the percentage mean and median difference in the number of (S)AE’s during the treatment study period (26 weeks) between patients in the treatment arm and patients in the observational arm.
    The absolute mean and median difference and the percentual mean and median difference between baseline (26 weeks prior to study inclusion) and the treatment study period (26 weeks) of patients in the treatment arm compared to patients in the observational arm in cost effectiveness. Cost effectiveness will be defined by measuring the healthcare costs. The following volumes of healthcare will be registering in Case Record Forms (CRF): (co) medication, visits and consultations to specialists or other health care professionals, lab and imagine techniques, operations, general practitioners and hospitalizations. The volumes of healthcare will be multiplied with the respective (Dutch) cost prices to get the healthcare costs (see economic evaluation).
    E.5.2.1Timepoint(s) of evaluation of this end point
    0 weeks, 4 weeks, 12 weeks, 26 weeks, 30 weeks (follow up)
    0 Wochen, 4 Wochen, 12 Wochen, 26 Wochen, 30 Wochen (Follow-up)
    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) 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.8.2.3.1Comparator description
    Standardtherapie
    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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    France
    Germany
    Italy
    Netherlands
    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
    LVLS
    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 years2
    E.8.9.2In all countries concerned by the trial months1
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 38
    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 have follow-up according to regular care.
    Nach Beendigung der Studie können die Patienten mit HHT auch
    weiterhin im westdeutschen Morbus Osler Zentrum entsprechend der
    vor der Studie geltenden Standards behandelt werden.
    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 Decision2020-08-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-05-08
    P. End of Trial
    P.End of Trial StatusOngoing
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