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    Summary
    EudraCT Number:2016-005110-22
    Sponsor's Protocol Code Number:SNT-I-018
    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:2017-06-23
    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 number2016-005110-22
    A.3Full title of the trial
    Phase-Ib/IIa study to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of orally inhaled multiple doses of POL6014 in patients with Cystic Fibrosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to investigate safety, tolerability and dose of orally inhaled multiple doses of POL6014 in patients with Cystic Fibrosis
    A.4.1Sponsor's protocol code numberSNT-I-018
    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 SponsorSanthera Pharmaceuticals (Switzerland) Ltd
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSanthera Pharmaceuticals (Switzerland) Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanthera Pharmaceuticals (Switzerland) Ltd
    B.5.2Functional name of contact pointAnna Carratú
    B.5.3 Address:
    B.5.3.1Street AddressHohenrainstrasse 24
    B.5.3.2Town/ cityPratteln
    B.5.3.3Post code4133
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41619068917
    B.5.5Fax number+41619068951
    B.5.6E-mailanna.carratu@santhera.com
    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 namePOL6014
    D.3.4Pharmaceutical form Nebuliser solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation 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.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 placeboNebuliser solution
    D.8.4Route of administration of the placeboInhalation 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
    mucoviscidosis
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    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 assess the safety and tolerability of up to three multiple ascending dose levels of inhaled POL6014 after 15 days q.d. or b.i.d.
    -To identify the highest tolerated dose level and associated dose regimen of inhaled POL6014 after 15 days q.d. or b.i.d. treatment.
    E.2.2Secondary objectives of the trial
    -To assess the pharmacokinetics (PK) in plasma, sputum and urine of up to 3 multiple ascending dose levels of inhaled POL6014 after 15 days q.d or b.i.d.; determine when steady state is reached during 15 days q.d. or b.i.d. treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Patient has given written informed consent to participation in the trial prior to their enrolment and any trial-related procedure.
    2.Male patients or female patients of non-childbearing potential, aged 18 to 55 years, inclusive. Women of non-childbearing potential are defined as those who have no uterus, or ligation of the fallopian tubes, or permanent cessation of ovarian function due to ovarian failure or surgical removal of the ovaries. Documentation of surgical procedure is required for patients who have had a hysterectomy or tubal ligation.
    3.Men must agree to practice contraception from start of study medication up to 90 days after the last dose of the study medication.
    4.Patient with a diagnosis of CF documented by a compatible clinical or radiographic presentation and laboratory criteria, more specifically, sweat or genetic testing (i.e., presence of the most common genetic defect ΔF 508 or any other mutation that can produce CF).
    5.Patient should confirm to produce frequently spontaneous sputum with a frequency of over 3 expectorates per day. Patient should be capable of producing a spontaneous sputum sample at screening (within a time range of approx. 3h during the screening visit).
    6.Except for CF and CF-related diseases, no other significant disease as assessed by a screening examination including medical history, physical examination, vital signs, ECG assessment, pulmonary function testing (PFT), and clinical laboratory results. Deviations of clinical laboratory results can be accepted if they are in accordance with the diagnosis of CF and CF-related diseases, supposed they do not indicate a clinical state that is expected to constitute a significant additional risk.
    7.Patient must have an FEV1 ≥ 40% of predicted value at screening.
    8.Body mass index (BMI) between 16.5 and 30 (both inclusive).
    9.Non-smoker or ex-smoker who has stopped smoking for at least one (1) year prior to the Screening Visit.
    10.Patient should be willing to refrain from caffeine- or theophylline-containing products within 24 h prior to a clinic visit for a full PK profile.
    11.Ability to inhale in an appropriate manner (Patients will be trained to inhale from the eFlow® nebuliser device with a commercially available saline solution at the Screening Visit once informed consent has been obtained).
    12.Patients should agree to start routine course of inhaled antibiotic cycle on the same day or not earlier than 3 days before IMP administration.
    E.4Principal exclusion criteria
    1.Patient with unstable lung disease, as defined by a change in treatment regimen during the preceding two (2) weeks, or a significant new finding on chest radiography (such as, but not limited to, pneumothorax, lobar/segmental collapse or consolidation), or in the opinion of the investigator, patient with a decline in pulmonary status within the last 12 months not considered a part of the usual, chronic progression of CF lung disease. Routine cyclic antibiotic treatment regimens including ”off/on” cycles are not considered to be changes to treatment regimens.
    2.Patient has had an exacerbation of respiratory symptoms within the past four (4) weeks before screening/randomization that required initiation of a new or altered respiratory therapy, and, in the opinion of the investigator, the patient has not returned to a stable level of health
    3.Patient with a history of lung transplantation.
    4.Patient with a history of clinically significant renal, hepatic, gastrointestinal, cardiovascular and particularly respiratory disease (excluding CF and CF-related disease).
    5.Patient with active gastrointestinal ulcer, history of intracranial bleedings, injuries and other bleedings.
    6.Patient, as per assessment of the investigator, with severe hepatic impairment (e.g. laboratory values (ALT, AST > 3 x ULN and total bilirubin > 1.5 x ULN) or Child-Pugh-Class C could be indicative of such condition).
    7.ECG abnormalities of clinical relevance (e.g., QTc according to Bazett’s formula ≥440 ms, PR >200 ms, or QRS ≥120 ms).
    8.Patient with a resting heart rate in supine position <50 bpm, systolic blood pressure <100 mmHg or >140 mmHg, diastolic blood pressure <60 mmHg or >90 mmHg.
    9.Proneness to orthostatic dysregulation, fainting, or blackouts.
    10.Diagnosis of a tricuspid insufficiency in combination with a mean pulmonary arterial pressure (mPAP) > 25 mmHg, measured by Doppler echography, or, if no tricuspid insufficiency is detectable, any echocardiographic or clinical signs of severe pulmonary heart disease (cor pulmonale) or depending congestive heart failure.
    11.History or presence of any malignancy.
    12.Positive results in any of the following virology tests: human immunodeficiency virus (HIV) antibodies and antigen, Anti-hepatitis B-core antibody, hepatitis B surface antigen (HbsAg) and anti-hepatitis C virus antibody.
    13.Known local or systemic hypersensitivity to any aerosol, medication or food that led to admission to an emergency room.
    14.Participation in another clinical study with any investigational medicinal product (IMP) or device, before randomisation, within an interval of 5 half-lives (minimum 4 weeks) from the last use of that investigational drug.
    15.Blood or plasma donation of more than 500 mL during the previous month before randomisation, as declared by the patient.
    16.Mental condition rendering the patient incapable to understand the nature, scope, and possible consequences of the study.
    17.Not willing to comply with all clinical study procedures.
    E.5 End points
    E.5.1Primary end point(s)
    -Proportion of patients with changes from baseline in physical examination findings
    -Changes from baseline in laboratory safety assessments (clinical chemistry, haematology, urinalysis)
    -Changes from baseline and pre-dose in vital signs (blood pressure, pulse, respiratory rate body temperature)
    -Changes from baseline and pre-dose in ECG parameters
    -Occurrence and severity of AEs
    -Proportion of patients with local irritation of the nose or pharynx by visual inspection
    -Proportion of patients with bronchospasm by symptoms such as dyspnoea, wheezing, chest tightness, cough
    -Changes from baseline and pre-dose in lung function parameters (FEV1, FVC) by spirometry
    -Changes from baseline in oxygen saturation in peripheral blood as measured by pulse oximetry.
    E.5.1.1Timepoint(s) of evaluation of this end point
    -physical examination findings: screening; V1; FU
    - laboratory safety assessments: screening, V2, 3, 4 and FU
    -vital signs (blood pressure, pulse, respiratory rate body temperature): screening, V2, 3, 4 and FU
    -ECG parameters:screening, V2, 3, 4 and FU
    - Occurrence and severity of AEs: throught all the study
    -Proportion of patients with local irritation of the nose or pharynx by visual inspection: V2, 3, 4
    -Proportion of patients with bronchospasm by symptoms such as dyspnoea, wheezing, chest tightness, cough: throught the study
    -lung function parameters (FEV1, FVC) by spirometry: screening, V2,3, 4 and FU
    -oxygen saturation in peripheral blood as measured by pulse oximetry: screening, V2,3, 4 and FU
    E.5.2Secondary end point(s)
    PK parameters derived from plasma, urine and sputum concentrations of POL6014 and metabolites
    E.5.2.1Timepoint(s) of evaluation of this end point
    Sputum and plasma PK samples: V2, 3, 4

    Urine sample: V2 and 4
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    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 No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase Ib/IIa
    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 No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    sequential-group dose-escalation
    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 trial3
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months14
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months14
    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: 40
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 40
    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)
    None. See section 5.4.3 of the protocol
    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 Mukoviszidose Institut - gemeinniitzige Gesellschaft
    G.4.3.4Network Country Germany
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation European Cystic Fibrosis Society - European Clinical
    G.4.3.4Network Country Belgium
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-11-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-09-19
    P. End of Trial
    P.End of Trial StatusOngoing
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