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 were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   44334   clinical trials with a EudraCT protocol, of which   7366   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 on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    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

    Summary
    EudraCT Number:2019-003324-20
    Sponsor's Protocol Code Number:STOPFOP1
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2019-10-16
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2019-003324-20
    A.3Full title of the trial
    Saracatinib trial TO Prevent FOP
    Een onderzoek ter beoordeling van de veiligheid, verdraagbaarheid en effecten op abnormale botvorming van het onderzoeksgeneesmiddel AZD0530 (saracatinib) bij patiënten met FOP
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to judge the safety, tolerability and effects on abnormal bone formation of reseach medication AZD 0530 (sarcatinib) in patients with FOP
    Een onderzoek ter beoordeling van de veiligheid, verdraagbaarheid en effecten op abnormale botvorming van het onderzoeksgeneesmiddel AZD0530 (saracatinib) bij patiënten met FOP
    A.3.2Name or abbreviated title of the trial where available
    STOPFOP
    A.4.1Sponsor's protocol code numberSTOPFOP1
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04307953
    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 SponsorVU University Medical Center
    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 supportInnovative Medicines Initiative EU
    B.4.2CountryBelgium
    B.4.1Name of organisation providing supportAstraZeneca
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVU University Medical Center
    B.5.2Functional name of contact pointFOP Amsterdam
    B.5.3 Address:
    B.5.3.1Street AddressDe Boelelaan 1117
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1081HV
    B.5.3.4CountryNetherlands
    B.5.6E-mailFOP.Amsterdam@vumc.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 No
    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 nameSaracatinib
    D.3.2Product code AZD0530
    D.3.4Pharmaceutical form Tablet
    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 INNSaracatinib
    D.3.9.1CAS number 893428-72-3
    D.3.9.3Other descriptive nameSARACATINIB DIFUMARATE
    D.3.9.4EV Substance CodeSUB91845
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number100
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fibrodyplasia Ossificans Progressiva (FOP)
    E.1.1.1Medical condition in easily understood language
    Genetic condition which causes abnormal formation of bone at abnormal locations such as in the muscles, tendons and ligaments.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10068715
    E.1.2Term Fibrodysplasia ossificans progressiva
    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 effectivity of treatment with AZD0530 on HO formation
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of AZD0530 and to further assess effectivity by functional and other radiological/nuclear imaging endpoints
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female aged 18-65 with a clinical diagnosis of FOP at screening, including congenital malformation of the great toes and a history of spontaneous or injury-induced heterotopic ossification (HO), and have a confirmed classic-like FOP phenotype by the documentation of an ACVR1R206H/+or variant genomic sequence
    a. Female participants who are women of child-bearing potential will be required to use a highly effective method of contraception as defined in section 5.4, in combination with a condom or diaphragm or cervical/vault caps with spermicidal foam/gel/film/suppository), from the time of enrolment until 4 weeks after final dose of study drug, unless practicing true sexual abstinence as defined in section 5.4.
    b. Male participants will be required to avoid procreative sexual intercourse with women of child-bearing potential from time of enrollment until 4 weeks after final dose of study drug through use of highly effective contraceptive methods. Male participants with a pregnant female partner will be required to use a condom for the duration of the study and for 4 weeks final dose of study drug. Male study participants will not be permitted to donate sperm for from the time of enrolment and until 4 weeks after final dose of study drug.

    2. Participants will have to be able to understand and complete study and willing to sign informed consent (IC). They have to be able to attend and comply with the study visits and related activities, adhere to all study-related restrictions, and able to undergo pro-cedures such as PET and CT imaging.
    E.4Principal exclusion criteria
    A potential subject who meets any of the following criteria will be excluded from participation in this study:
    1. Not willing to strictly adhere to the reproductive restrictions as defined in section 5.4
    2. Women who are pregnant or breast-feeding (from the time 3 months prior to 4 weeksafter completion of participation in the study)
    3. The presence of significant concomitant illness or history of significant illness such as cardiac, respiratory, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic, lymphatic disease, or infectious disease, that might confound the results of the study or pose additional risk to the patient;
    4. Evidence of active bleeding (including hematuria or hematochezia,) acute or chronic gastrointestinal illness, inflammatory bowel disease, or mucositis
    5. Malignant disease / cancer requiring treatment in the past 3 years (except some primary non melanoma skin cancer);
    6. Severely impaired renal function defined as estimated glomerular filtration rate <30 mL/min/1.73 m2 calculated by the Modification of Diet in Renal Disease equation;
    7. Showing uncontrolled diabetes mellitus with an HbA1C > 9%;
    8. Significant viral illness or active infections at screening or randomisation; Subjects should not have subacute or acute fevers of >101F at time of screening or randomisation
    9. Evidence of prolonged QT interval at screening or randomization (defined as QTc of >450 ms) .or known congenital long-QT syndrome.
    10. Neutropenia defined as an absolute neutrophil count of <1,500/µl,
    11. Thrombocytopenia defined as platelet count <100 × 103/µl,
    12. Current blood clotting or bleeding disorder, or significantly abnormal INR-prothrombin time or partial thromboplastin time at screening, or clinically significant abnormalities in other screening laboratories, including significant abnormalities in vitamin B12 or thyroid function tests would be cause for exclusion.-
    13. Abnormal liver function test results defined as aspartate aminotransferase (AST) >2.0 x upper limit of normal (ULN); alanine aminotransferase (ALT) >2.0 x ULN; and / or total bilirubin >1.5 x ULN;
    14. Known allergy or intolerance to AZD0530 or any excipients used in the investigational medicinal products.
    15. Simultaneous participation in another interventional clinical study or a non-interventional study with imaging measures or invasive procedures (eg. collection of blood or tissue samples); Participation in the FOP Connection Registry (www.fopconnection.org) or other studies in which patients completed study questionnaires are possible.
    16. Treatment with another investigational or drug that might interfere with HO formation and the interpretation of the study drug in the last 90 days
    17. Current use or history of regular alcohol consumption exceeding 14 units/week (6 glasses of 13.0% wine (175ml), 6 pints of 4.0% lager or ale (568ml), 5 pints of 4.5% cider (568 ml) or 14 glasses of 10.0% spirits (25ml)) within 6 months of screening.
    18. Currently active metabolic bone disease, other than FOP.
    E.5 End points
    E.5.1Primary end point(s)
    1. The primary endpoint:
    Primary Safety: Incidence and severity of treatment- emergent adverse events through the end of the Treatment Period 1 at week 26 (RCT)

    Primary Efficacy: Number of new lesions in the RCT defined as:
     Number of individual new active HO lesions assessed by [18F]-NaF PET at week 26 in placebo vs. drug arms of the RCT*
     Number of individual new HO lesions detected by CT at week 26 in placebo vs. drug arms of the RCT
    New lesions will be defined by their appearance on sequential [18F]-NaF PET, sequential CT scans, or both.
    * not related to trauma such as fractures

    E.5.1.1Timepoint(s) of evaluation of this end point
    The first evaluation will take place after one year of the study. Unblinding of the study will take place after all participants have completed one year of study.All other analyzes will be conducted at the end of the study (after all participants have completed the 18 months.
    E.5.2Secondary end point(s)
    The key secondary endpoints are:
    1. The incidence and severity of adverse events (AE)
    a. During the 6-month RCT
    b. During 6-month open label extension of AZD0530
    2. Number of new lesions after one year of cross-over therapy, defined as:
     Number of individual new active HO lesions assessed by [18F]-NaF PET between the placebo arm at week 26 and the subsequent open-label phase at week 52 among pa-tients receiving placebo during the RCT*
     Number of individual new HO lesions detected by CT be-tween the placebo arm at week 26 and the subsequent open-label arm at week 52 among patients receiving pla-cebo during the RCT
    New lesions will be defined by their appearance on sequential [18F]-NaF PET, sequential CT scans, or both.
    These cross-over treatment data may be considered separately, or combined with RCT data.
    * not related to trauma such as fractures
    3. Difference in [18F] NaF PET activity of individual active lesion(s) by PET at week 26 between the placebo and drug arms of the RCT, or between the placebo arm at week 26 and its subsequent open label arm at week 52 among cross-over patients.
    4. Difference in volume of individual active lesions as assessed by [18F] NaF PET at week 26 between the placebo and drug arm of the RCT, or between the placebo arm at week 26 and its subsequent open label arm at week 52 among cross-over patients.
    5. Change in volume of individual HO lesions as assessed by by CT at week 26 between the placebo and drug arm of the RCT, or between the placebo arm at week 26 and its subsequent open label arm at week 52 among cross-over patients.
    6. Change in functional mobility outcomes from baseline to week 26 between the place-bo versus drug arm of the RCT, or change between baseline and week 26 in the pla-cebo arm versus the change between week 26 and week 52 among cross-over pa-tients initially treated with placebo in the RCT, as assessed by:
    a. Change in the cumulative analogue joint involvement scale for FOP (CAJIS),
    b. Change in the quantitative detailed multi-joint assessment by goniometry,
    c. Change in interincisal distance (mouth opening, mm.)
    d. Change in forced vital capacity (FVC) as percent of predicted based on age, sex, and body mass index.
    7. Change in functional mobility outcomes at week 52, 104, and week 156 of drug treat-ment compared to baseline measurements, in reference to Ipsen natural history data (NCT02322255) and/or other history data on rate of progression of these measure-ments in an untreated FOP population where available.
    a. Change in the cumulative analogue joint involvement scale for FOP (CAJIS),
    b. Change in the quantitative detailed multi-joint assessment by goniometry,
    c. Change in interincisal distance (mouth opening, mm.)
    d. Change in forced vital capacity (FVC) as percent of predicted based on age, sex, and body mass index.

    E.5.2.1Timepoint(s) of evaluation of this end point
    The first evaluation will take place after one year of the study. Unblinding of the study will take place after all participants have completed one year of study.All other analyzes will be conducted at the end of the study (after all participants have completed the 18 months.
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Double blind RCT followed by open-label extension phase
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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 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
    The end of study for this study is defined as the last visit of the last patient.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months6
    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: 17
    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 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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    Standard treatment
    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 Decision2019-10-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-08
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat May 03 08:54:32 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA