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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   42330   clinical trials with a EudraCT protocol, of which   6971   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.
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    EudraCT Number:2015-005527-12
    Sponsor's Protocol Code Number:55868
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2015-12-16
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2015-005527-12
    A.3Full title of the trial
    Sirolimus for the treatment of severe intestinal polyposis in patients with familial adenomatous polyposis (FAP); a pilot study
    Sirolimus voor de behandeling van ernstige intestinale polyposis bij patiënten met familiaire adenomateuze polyposis (FAP); een pilot studie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Sirolimus for the treatment of severe intestinal polyposis in patients with familial adenomatous polyposis (FAP); a pilot study
    Sirolimus voor de behandeling van ernstige intestinale polyposis bij patiënten met familiaire adenomateuze polyposis (FAP); een pilot studie
    A.3.2Name or abbreviated title of the trial where available
    Sirolimus and FAP
    Sirolimus en FAP
    A.4.1Sponsor's protocol code number55868
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    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. name Rapamune
    D. of the Marketing Authorisation holderPfizer
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Familial adenomatous polyposis
    Familiaire adenomateuze polyposis
    E.1.1.1Medical condition in easily understood language
    Familial adenomatous polyposis
    Familiaire adenomateuze polyposis
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    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
    The aim of our study is to investigate the effect of sirolimus on the progression of intestinal adenomas in patients with FAP and to assess the safety of this treatment
    Het doel van deze pilotstudie is het onderzoeken van het effect van sirolimus op intestinale adenomen bij patienten met FAP en het beoordelen van de veiligheid van deze behandeling
    E.2.2Secondary objectives of the trial
    Not applicable
    Niet van toepassing
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - ≥ 18 years
    -A genetically confirmed APC mutation
    -Classical FAP phenotype (100-1000 colorectal adenomatous polyps)
    -Subtotal colectomy with ileorectal anastomosis (IRA) or ileo-anal pouch anastomosis
    -Severe rectal or pouch polyposis, defined as having >25 polyps amenable to complete removal (InSiGHT 2011 Staging System score of 3)
    -Fertile patients must use effective contraception during study treatment and until 12 weeks after treatment
    - ≥ 18 jaar
    - Een genetisch bewezen APC mutatie
    - Klassiek FAP fenotype (100-1000 colorectale adenomen)
    - Subtotale colectomie met ileorectale anastomose (IRA) of een ileo-anale pouch anastomose
    - Ernstige rectale of pouch polyposis, gedefinieerd als >25 poliepen die compleet verwijderd zouden kunnen worden (InSiGHT 2011 Staging System score van 3)
    - Vruchtbare patiënten moeten een effectieve anticonceptie methode gebruiken gedurende de studieperiode en tot 12 weken na studiedeelname
    E.4Principal exclusion criteria
    -Inability to give informed consent
    -Participation in another interventional clinical trial
    -Subjects who are pregnant or breast-feeding
    -Prior pelvic irradiation
    -Invasive malignancy in the past 5 years
    -Subjects who are HIV positive
    -Subjects with severe systemic infections, current or within 2 weeks prior to study start
    -Subjects with known severe restrictive or obstructive pulmonary disorders
    -History of pulmonary embolism or deep venous thrombosis
    -Major surgery less than or equal to 2 weeks prior to enrollment or any planned surgery within treatment period
    -Active post-operative complication, e.g. infection, delayed wound healing
    -History of hypersensitivity to sirolimus or to drugs of similar chemical classes
    -Regular NSAID use
    -Use of other FAP directed drug therapies
    -Subjects requiring systemic anticoagulation
    -Co-medication that could interact with sirolimus
    -Significant abnormalities in hepatic function
    -Significant hematologic abnormalities
    -Increased fasting serum cholesterol or triglyceride (whether or not on lipid-lowering therapy)
    -Increased glucose (venous, fasting): >6.4 mmol/L
    -Electrolyte abnormalities
    -Calculated glomerular filtration rate (GFR) less than 40 mL/min/1.73m2
    using the simplified Modification of Diet in Renal Disease (MDRD) formula
    -Spot urine protein to creatinine ratio (UPr/Cr) greater than or equal to 0.5
    -Niet in staat informed consent te geven
    -Deelname in een andere interventionele klinische trial
    -Personen die zwanger zijn of borstvoeding geven
    -In verleden bestraald in bekkengebied
    -Een invasieve maligniteit in de afgelopen 5 jaar
    -Personen die HIV positief zijn
    -Personen met ernstige systemische infecties, nu of binnen 2 weken voor deelname
    -Personen met ernstige restrictieve of obstructieve longaandoeningen
    -Longembolie of diepe veneuze trombose in voorgeschiedenis
    -Grote chirurgische ingreep binnen 2 weken van start deelname of een geplande operatie tijdens de studieperiode
    -Actieve post-operatieve complicatie
    -Hypersensitiviteit voor sirolimus or vergelijkbare geneesmiddelen
    -Regelmatig gebruik van NSAID
    -Gebruik van andere op FAP gerichte medicatie
    -Gebruik van systemische anticoagulantia
    -Comedicatie die interactie kan hebben met sirolimus
    -Significante hepatologische labafwijkingen
    -Significante hematologische labafwijkingen
    -Verhoogd serum cholesterol of triglyceriden
    -Verhoogd glucose
    -GFR < 40 mL/min/1.73m2
    -Urine proteine creatinine ratio (UPr/Cr) > of gelijk aan 0.5
    E.5 End points
    E.5.1Primary end point(s)
    • Size of 5 marked polyps 5 patients with FAP and a large intestinal polyp burden
    • Safety outcomes reported by summary analysis of adverse events , clinical laboratory abnormalities and regular physical examination
    • Grootte van 5 gemarkeerde poliepen bij 5 patiënten met FAP met een ernstige intestinale polyposis
    • Veiligheid van de behandeling (adverse events, labafwijkingen, lichamelijk onderzoek)
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 Months
    6 Maanden
    E.5.2Secondary end point(s)
    • Number of polyps
    • Global polyp burden
    • Histology (tubular, tubulovillous or villous histology and degree of dysplasia)
    • Patient reported quality of life using HRQoL questionnaires
    • Rate of cell proliferation in healthy intestinal mucosa and adenomatous tissue
    • Immunohistochemistry of mTOR targets (such as eEF2 kinase, phospho-S6) in healthy intestinal mucosa and adenomatous tissue
    • Aantal poliepen
    • 'Global polyp burden'
    • Histologie (tubulaire, tubulovilleuze of villeuze histologie en mate van dysplasie)
    • Patient gerapporteerde kwaliteit van leven mbv vragenlijsten
    • Celproliferatie in gezond en adenomateus intestinaal weefsel
    • Immunohistochemie van mTOR targets (zoals as eEF2 kinase, phospho-S6) in gezond en adenomateus intestinaal weefsel
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 Months
    6 Maanden
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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. 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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    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.8.5The 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
    Laatste bezoek van de laatste deelnemer
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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.1.1.1In 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: 5
    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 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.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)
    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 Decision2015-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-12-02
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-12-10
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