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   43851   clinical trials with a EudraCT protocol, of which   7283   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:2011-000203-41
    Sponsor's Protocol Code Number:AC-052-391
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2014-07-11
    Trial results View 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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2011-000203-41
    A.3Full title of the trial
    Multicenter, double-blind, placebo-controlled, randomized, prospective
    study of bosentan as adjunctive therapy to inhaled nitric oxide in the
    management of persistent pulmonary hypertension of the newborn (PPHN)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research project to find out whether adding bosentan to inhaled nitric
    oxide in newborns with persistent pulmonary hypertension of newborns
    (PPHN) is a supporting and safe therapy.
    A.3.2Name or abbreviated title of the trial where available
    FUTURE 4
    A.4.1Sponsor's protocol code numberAC-052-391
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/283/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorActelion Pharmaceuticals 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 supportActelion Pharmaceuticals Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationActelion Pharmaceuticals Ltd.
    B.5.2Functional name of contact pointGLOBAL MEDICAL INFORMATION
    B.5.3 Address:
    B.5.3.1Street AddressGewerbestrasse 16
    B.5.3.2Town/ cityAllschwil
    B.5.3.3Post code4123
    B.5.3.4CountrySwitzerland
    B.5.6E-mailmedinfo@actelion.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 Yes
    D.2.1.1.1Trade name Tracleer
    D.2.1.1.2Name of the Marketing Authorisation holderActelion Registration Ltd
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/01/019
    D.3 Description of the IMP
    D.3.1Product namebosentan
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPNasogastric use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBOSENTAN
    D.3.9.1CAS number 147536-97-8
    D.3.9.2Current sponsor codeACT-050088
    D.3.9.4EV Substance CodeSUB05877MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number32
    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 placeboDispersible tablet
    D.8.4Route of administration of the placeboNasogastric use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Persistent Pulmonary Hypertension of the Newborn (PPHN)
    E.1.1.1Medical condition in easily understood language
    PPHN is a rare and life threatening disease causing severe breathing
    difficulties in babies which can lead to suffocation and death.
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10053592
    E.1.2Term Newborn persistent pulmonary hypertension
    E.1.2System Organ Class 100000004850
    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 efficacy of bosentan in neonates with persistent
    pulmonary hypertension of the newborn (PPHN) who are in need of
    continued inhaled nitric oxide (iNO) after at least 4 hours of continuous
    iNO treatment
    E.2.2Secondary objectives of the trial
    To evaluate the pharmacokinetics (PK), tolerability, and safety of
    bosentan in this patient population
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed informed consent by the parent(s) or the legal
    representative(s).
    2. Term and near term newborns (gestational age > 34 weeks).
    3. Post natal age ≥ 12 hours and < 7 days at randomization.
    4. Weight at birth ≥ 2,000 g.
    5. Idiopathic PPHN or PPHN due to parenchymal lung disease.
    6. Documented diagnosis of pulmonary hypertension (PH) confirmed by echocardiography.
    7. Need for continued iNO at a dose > 10ppm after at least 4 hours of
    iNO treatment, and at a dose ≥ 10 ppm at randomization.
    8. Two oxygenation index (OI) values ≥ 12, taken at least 30 minutes
    apart, in the 12 hours prior to randomization and while the patient is
    receiving iNO treatment.
    9. Mechanical ventilation with fraction of inspired oxygen (FiO2) ≥ 50%
    at randomization.
    E.4Principal exclusion criteria
    1. PH associated with conditions other than PPHN.
    2. Immediate need for cardiac resuscitation or extracorporeal membrane oxygenation (ECMO).
    3. Lethal congenital anomalies.
    4. Congenital Diaphragmatic Hernia.
    5. Significant structural cardiac anomalies.
    6. Medically significant pneumothorax.
    7. Active seizures.
    8. Expected duration of mechanical ventilation of less than 48 hours.
    9. Mean systemic blood pressure < 35 mmHg despite therapy with
    volume infusions and cardiotonic support.
    10. Hepatic failure or all conditions with ALT value > 2 x ULN.
    11. Renal function impairment such as serum creatinine > 3 x ULN or
    anuria.
    12. Known intracranial hemorrhage grade III or IV.
    13. Either hemoglobin or hematocrit level < 75% of the LLN.
    14. Thrombocytopenia (platelet count < 50,000 cells /μL).
    15. Leukopenia (WBC < 2,500 cells/ μL).
    16. Any condition precluding the use of a nasogastric/orogastric tube.
    17. Administration of prohibited medication prior to randomization.
    E.5 End points
    E.5.1Primary end point(s)
    - Proportion of patients with treatment failure defined as:
    . Need for ECMO
    . Initiation of alternative pulmonary vasodilator
    - Time to complete weaning from iNO.
    - Time to weaning from mechanical ventilation.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of treatment
    E.5.2Secondary end point(s)
    - Proportion of patients requiring re-initiation of iNO therapy.
    - Change from baseline to 3, 5, 12, and 24 hours following the first drug
    administration; then, daily until EoT for:
    Oxygenation Index
    Arterial blood gas values (pH, SaO2, PaO2, PaCO2)
    Pulse oximetry (SpO2)
    FiO2
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of treatment
    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 Yes
    E.6.6Pharmacokinetic Yes
    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 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) 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 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
    exploratory
    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 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Australia
    Korea, Republic of
    Russian Federation
    Singapore
    Switzerland
    United States
    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 is defined as the end of treatment (EoT=date of last
    study drug administration) + 7 days.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 30
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
    F.1.1.2.1Number of subjects for this age range: 15
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 15
    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) No
    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 No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    pediatric population
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 30
    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)
    Normal treatment
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Australia
    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 Apr 20 11:56:52 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA