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    Summary
    EudraCT Number:2020-003913-36
    Sponsor's Protocol Code Number:REFSAL
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2020-12-07
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-003913-36
    A.3Full title of the trial
    Evaluation of frequency occurrence persistent pulmonary hypertension of newborns in group of newborns born between 32 and 42 weeks of pregnancy treated by salbutamol
    Ocena częstości występowania przetrwałego nadciśnienia płucnego noworodków w grupie noworodków urodzonych pomiędzy 32 a 42 tygodniem ciąży leczonych salbutamolem

    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of frequency occurrence persistent pulmonary hypertension of newborns treated by salbutamol
    Ocena częstości występowania przetrwałego nadciśnienia płucnego noworodków leczonych salbutamolem
    A.3.2Name or abbreviated title of the trial where available
    REFSAL
    REFSAL
    A.4.1Sponsor's protocol code numberREFSAL
    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 SponsorWarszawski Uniwersytet Medyczny
    B.1.3.4CountryPoland
    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 supportthe Medical Research Agency based on the Medical University of Warsaw proposal no: 2019/ABM/01/00058/P/03
    B.4.2CountryPoland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDr. hab. n. med. Renata Bokiniec / Warszawski Uniwersytet Medyczny
    B.5.2Functional name of contact pointStudy Country Coordinator
    B.5.3 Address:
    B.5.3.1Street AddressKarowa 2
    B.5.3.2Town/ cityWarszawa
    B.5.3.3Post code00-315
    B.5.3.4CountryPoland
    B.5.4Telephone number00482259 66 155
    B.5.5Fax number0482259 66 484
    B.5.6E-mailrenata.bokiniec@wum.edu.pl
    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 Ventolin, 1 mg/ml (0,1%), nebulisation solution
    D.2.1.1.2Name of the Marketing Authorisation holderGlaxoSmithKline (Ireland) Limited 12 Riverwalk 7, Citywest Business Campus, Dublin 24 Irlandia
    D.2.1.2Country which granted the Marketing AuthorisationPoland
    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 Nebulisation 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 placeboNebulisation 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
    Persistent pulmonary hypertension of newborns in group of newborns born between 32 and 42 weeks of pregnancy
    Przetrwałe nadciśnienie płucne noworodków (PPHN) w grupie noworodków urodzonych pomiędzy 32 a 42 tygodniem ciąży
    E.1.1.1Medical condition in easily understood language
    Breathing disorders as a cause of neonatal respiratory failure
    Zaburzenia oddychania jako przyczyna niewydolności oddechowej noworodków
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    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 frequency occurrence PPHN defined as the need to use ventilation with an additional oxygen fraction in the respiratory mixture (FiO2)> 0.30 and echocardiographic features of increased pulmonary pressure (right - left or bi-directional leakage through an unobstructed arterial duct or oval opening) or the difference in pre- and post-ductal saturation > 10%
    Ocena częstości występowania PPHN definiowanego jako konieczność stosowania wentylacji z dodatkową frakcją tlenu w mieszaninie oddechowej (FiO2) > 0,30 oraz echokardiograficzne cechy podwyższonego ciśnienia płucnego (przeciek prawo – lewy lub dwukierunkowy przez drożny przewód tętniczy lub otwór owalny) albo różnica saturacji przed i zaprzewodowej > 10%
    E.2.2Secondary objectives of the trial
    Evaluation on the TTN scale (modified Silverman score) in comparison to the concentration of oxygen used during ventilation and acid-base equilibrium parameters: pH, carbon dioxide pressure (pCO2) and alkaline deficiency (BE) in compared groups
    Ocena w skali TTN (zmodyfikowana skala Silvermana) w porównaniu do stężenia tlenu stosowanego podczas wentylacji oraz parametrów badania równowagi kwasowo-zasadowej: pH, prężności dwutlenku węgla (pCO2) i niedoboru zasad (BE) w porównywanych grupach
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Screening / Eligibility criteria:

    Newborns born from a pregnancy lasting min. 32 0/7 and no longer than 41 6/7 weeks.
    Eligibility period from birth until the inclusion criteria are met or 24 hours are reached, whichever comes first.

    Inclusion criteria:
    Each Patient in the Study must meet all of the following inclusion criteria:

    1. Disturbances of tachypnoe breathing and expiratory bulking persisting for more than 15 minutes after birth or the above mentioned respiratory disturbances occurring up to the age of 6 hours for at least 15 minutes or the need for breathing support at birth before the age of 6 hours
    2. Newborns born from a pregnancy of at least 32 0/7 weeks to 41 6/7 weeks.
    3. Chest X-ray image taken up to 6 hours of age.
    4. Performed acid-base balance (ABS) test from umbilical cord blood or capillary blood drawn from the heel of a newborn in the first hour of life.
    Kryteria kwalifikacji:

    Noworodki urodzone z ciąży trwającej min. 32 0/7 i nie dłużej niż 41 6/7 tygodni.
    Okres kwalifikacji od urodzenia do momentu spełnienia kryteriów włączenia lub do osiągnięcia 24 godzin, w zależności od tego, co nastąpi wcześniej.

    Kryteria włączenia:

    Każdy pacjent biorący udział w badaniu musi spełniać wszystkie poniższe kryteria włączenia:
    1. Zaburzenia oddychania pod postacią tachypnoe oraz postękiwania wydechowego utrzymujące się powyżej 15 min po urodzeniu lub wyżej wymienione zaburzenia oddychania występujące do 6 godziny życia przez co najmniej 15 minut lub konieczność stosowania wsparcia oddechu po urodzeniu przed ukończeniem 6 godziny życia
    2. Noworodki urodzone z ciąży trwającej co najmniej 32 0/7 tygodni do 41 6/7 tygodni.
    3. Wykonane zdjęcie RTG klatki piersiowej do 6 godziny życia.
    4. Wykonane badanie równowagi kwasowo-zasadowej (RKZ) z krwi pępowinowej lub z krwi włośniczkowej pobranej z pięty u noworodka w pierwszej godzinie życia.
    E.4Principal exclusion criteria
    If the Patient meets one of the following conditions cannot be included in the Study:
    1. Need for intubation immediately after birth
    2. Perinatal hypoxia defined on the basis of an abnormal acid-base balance (ABS) result from umbilical cord blood or capillary blood taken from the heel of a newborn in the first hour of life, with a pH < 7.0 or BE below -14 mmol/L.
    3. Age over 24 hours from birth
    4. Meconium aspiration syndrome
    5. Neonatal air leak syndrome
    6. Congenital heart defects
    7. Congenital diaphragmatic hernia
    8. Other severe congenital malformations and genetically determined syndromes associated with a higher risk of respiratory failure diagnosed before and after birth.
    9. Necessity for surfactant administrating directly after birth regardless of the method of administration (respiratory distress syndrome - RDS).
    Jeżeli pacjent spełni jeden z poniższych warunków nie może zostać włączony do badania:
    1. Konieczność intubacji bezpośrednio po urodzeniu
    2. Niedotlenienie okołoporodowe definiowane na podstawie nieprawidłowego wyniku RKZ z krwi pępowinowej lub krwi włośniczkowej pobranej z pięty u noworodka w pierwszej godzinie życia, z pH < 7,0 lub BE poniżej -14 mmol/L.3. Wiek powyżej 24 godziny życia od urodzenia
    4. Zespół aspiracji smółki
    5. Zespoły ucieczki powietrza
    6. Wrodzone wady serca
    7. Wrodzona przepuklina przeponowa
    8. Inne ciężkie wady wrodzone i zespoły uwarunkowane genetycznie związane z wyższym ryzykiem niewydolności oddechowej rozpoznane przed i po urodzeniu.
    9. Konieczność podaży surfaktantu bezpośrednio po urodzeniu niezależnie od sposobu jego podania (zespół zaburzeń oddychania - RDS)
    E.5 End points
    E.5.1Primary end point(s)
    Assessment of the incidence of PPHN defined as the need for ventilation with additional oxygen fraction in the respiratory mixture (FiO2) > 0. 30 and echocardiographic features of elevated pulmonary pressure or the difference in pre and postprandial saturation > 10%
    Ocena częstości występowania PPHN definiowanego jako konieczność stosowania wentylacji z dodatkową frakcją tlenu w mieszaninie oddechowej (FiO2) > 0,30 oraz echokardiograficzne cechy podwyższonego ciśnienia płucnego albo różnica saturacji przed i zaprzewodowej > 10%
    E.5.1.1Timepoint(s) of evaluation of this end point
    Echocardiographic examination during the observation period, i. e. Extended 48 hours from randomisation to 7 days of age or discharge.
    Badanie echokardiograficzne w okresie obserwacji, tj. okres rozszerzony 48h od randomizacji do 7 dnia życia lub wypisu.
    E.5.2Secondary end point(s)
    1. Scoring on the TTN score.
    2. Prevalence of failures defined as the need for intubation
    3. Duration of respiratory support
    4. Hospitalisation time
    5. Results of the acid-base balance (ABS) and ionogram
    6. Occurrence of Adverse Events
    1. Punktacja w skali TTN
    2. Częstość występowania niepowodzeń definiowanych jako konieczność intubacji
    3. Czas stosowania wsparcia oddechowego
    4. Czas hospitalizacji
    5. Wyniki RKZ i jonogramu
    6. Występowanie zdarzeń niepożądanych
    E.5.2.1Timepoint(s) of evaluation of this end point
    Treatment/observation period - II Period (24h to 48h from randomization), during the observation period to the Investigator decision.
    Okres Leczenie/Obserwacja – II Okres (24h do 48h od randomizacji), w okresie obserwacji do decyzji badacza.
    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 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.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 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 concerned8
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 Yes
    F.1.1Number of subjects for this age range: 608
    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: 547
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 61
    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 No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects's age - newborn infants and newborns
    wiek Uczestnika – noworodki
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state608
    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
    Nie dotyczy
    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 Decision2021-06-23
    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 StatusTrial now transitioned
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