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    The EU Clinical Trials Register currently displays   39177   clinical trials with a EudraCT protocol, of which   6419   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).
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    EudraCT Number:2016-004786-80
    Sponsor's Protocol Code Number:ANAKIN
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2017-06-30
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2016-004786-80
    A.3Full title of the trial
    A phase IIa, randomized, placebo-controlled, double-blind, cross-over study to evaluate safety and efficacy of subcutanous administration of anakinra in patients with cystic fibrosis.
    Phase IIa, randomisierte, Placebo-kontrollierte, doppel-blinde, cross-over Studie zur Untersuchung von Sicherheit und Wirksamkeit der subkutanen Verabreichung von Anakinra bei Patienten mit Cystischer Fibrose (CF).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Tolerability and Efficacy of Anakinra in patients with cystic fibrosis.
    Verträglichkeit und Wirksamkeit von Anakinra bei Patienten mit Cystischer Fibrose.
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberANAKIN
    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 SponsorRuprecht-Karls-University Heidelberg, Medical Faculty
    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 supportBMBF
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Hospital Heidelberg, Department of Pediatrics III
    B.5.2Functional name of contact pointDivision of Pediatric Pulmonology
    B.5.3 Address:
    B.5.3.1Street AddressIm Neuenheimer Feld 430
    B.5.3.2Town/ cityHeidelberg
    B.5.3.3Post code69120
    B.5.4Telephone number+496221568355
    B.5.5Fax number+496221568806
    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 Kineret
    D. of the Marketing Authorisation holderSwedish Orphan Biovitrum AB (SOBI)
    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 in pre-filled syringe
    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.3.8INN - Proposed INNANAKINRA
    D.3.9.1CAS number 143090-92-0
    D.3.9.4EV Substance CodeSUB05500MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 placeboSolution for injection in pre-filled syringe
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic fibrosis
    E.1.1.1Medical condition in easily understood language
    Cystic fibrosis, mucoviscidosis
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10028141
    E.1.2Term Mucoviscidosis
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • Assessment of absolute pre-post change of the lung clearance index (LCI).
    E.2.2Secondary objectives of the trial
    • Evaluation of the safety and tolerability of the 28-days-treatment with anakinra.
    • Investigation of the effects of anakinra on lung function.
    • Evaluation of the impact of anakinra on the quality-of-life (QoL).
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Both sub-studies are described directly in the main trial protocol. Their objectives are:
    • Assessment of the influence of anakinra on lung structure and perfusion.
    • Assessment of the influence of anakinra on airway inflammation and bronchial infection
    E.3Principal inclusion criteria
    1. Age ≥ 18 years (1st cohort). If justified by interim analysis, 18 > age ≥ 12 years (2nd cohort),
    2. Informed consent,
    3. Sufficient fluency in German language,
    4. Confirmed diagnosis of cystic fibrosis,
    5. Best FEV1 ≥ 70 % pred. in the last 12 months prior to screening
    6. FEV1 ≥ 60 % pred. at screening,
    7. LCI ≥ 7.5 at screening,
    8. Ability to performed study specific procedures,
    9. Oxyhaemoglobin saturation of ≥ 90% on room air at screening,
    10. No recent changes in the medication for cystic fibrosis lung disease,
    11. Adequate bone marrow function,
    12. Adequate liver function,
    13. Adequate blood clotting,
    14. Negative serology for HIV, HBV, and HCV, negative Interferon-gamma release assay,
    15. Negative pregnancy test in women of childbearing potential,
    16. Use of adequate contraception in sexually active female subjects.
    E.4Principal exclusion criteria
    1. Expected non-compliance,
    2. Known allergy to anakinra or any ingredient of the pharmaceutical formulation of Kineret®,
    3. Planned (during the treatment) or recently completed immunization with attenuated (live) vaccine(s),
    4. Renal failure,
    5. History of tuberculosis or repeated detection of non-tuberculous mycobacteria from airway samples in the last 12 months,
    6. History of detection of Burkholderia species in the last 12 months,
    7. Current colonization with multi-resistant Staphylococcus aureus (MRSA) and/or 4-multi-resistant gram negative (MRGN) Pseudomonas aeruginosa,
    8. Acute bronchopulmonary exacerbation,
    9. Signs of other active infection within 14 days prior to the screening,
    10. Immunosuppressive treatment due to organ transplantation, rheumatic or autoimmune diseases,
    11. Recent Participation in another interventional trial,
    12. Current oral corticosteroid use,
    13. Current oxygen supplementation,
    14. Current treatment with etanercept,
    15. Medical history of lung transplantation,
    16. Pregnancy or nursing,
    17. Known hypersensitivity to hypertonic saline (used for induction of sputum).
    E.5 End points
    E.5.1Primary end point(s)
    • Absolute pre-post change of the lung clearance index (LCI).
    E.5.1.1Timepoint(s) of evaluation of this end point
    At all trial visits, i.e. at 6 timepoints: Screening visit, directly prior and directly after administration of IMP and Placebo, End of trial visit.
    E.5.2Secondary end point(s)
    • Evaluation of the safety and tolerability of the 28-days-treatment with anakinra by means of:
    o Physical examination
    o (Serious) adverse events
    o Laboratory safety parameters
    • Investigation of the effects of anakinra on lung function by means of absolute change in percentage points predicted forced expiratory volume in 1 second (FEV1 and FEV1 % pred).
    • Evaluation of the impact of anakinra on the quality-of-life (QoL) in the considered population by means of the Cystic Fibrosis Questionnaire – Revised (CFQ-R, German version).
    • Investigation of the effects of anakinra on lung function by means of absolute change in forced expiratory flow75 in liters/second and percent predicted (FEF75 and FEF75 % pred) and forced vital capacity in liter and percent predicted (FVC and FVC % pred).

    • Assessment of the influence of anakinra on lung structure and perfusion determined by chest MRI.
    • Assessment of the influence of anakinra on airway inflammation by means of the following parameters:
    o Absolute and differential cell count in sputum samples,
    o Inflammatory markers in sputum samples.
    • Assessment of the influence of anakinra on the bronchial infection status by means of sputum microbiology.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At all trial visits, i.e. at 6 timepoints: Screening visit, directly prior and directly after administration of IMP and Placebo, End of trial visit.
    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 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 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 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 No
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    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 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 concerned5
    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 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 years2
    E.8.9.1In the Member State concerned months3
    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 months3
    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 Yes
    F.1.1Number of subjects for this age range: 27
    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. of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 27
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    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 Yes
    F. of subjects incapable of giving consent
    Adolescents below age of 18 years.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state52
    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 Decision2018-11-19
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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