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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   42556   clinical trials with a EudraCT protocol, of which   7007   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 in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

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    Summary
    EudraCT Number:2008-004949-28
    Sponsor's Protocol Code Number:P071003
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2009-06-22
    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 ConcernedFrance - ANSM
    A.2EudraCT number2008-004949-28
    A.3Full title of the trial
    Traitement par le monoxyde d’azote inhalé (NO) du syndrome thoracique aigu (STA) chez l'enfant drépanocytaire : Essai randomisé, double aveugle, contre placebo, preuve de concept
    A.3.2Name or abbreviated title of the trial where available
    INNOSTA PED
    A.4.1Sponsor's protocol code numberP071003
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.1.3.4CountryFrance
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Inomax 800ppm
    D.2.1.1.2Name of the Marketing Authorisation holderINO Therapeutics
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameInomax 800ppm
    D.3.4Pharmaceutical form Inhalation gas
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMonoxyde d'azote
    D.3.9.1CAS number 10102-43-9
    D.3.10 Strength
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number800 ppm Mole/Mole
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 placeboInhalation gas
    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
    Traitement du syndrome thoracique aigu (STA)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 8.1
    E.1.2Level PT
    E.1.2Classification code 10051835
    E.1.2Term Drepanocytose
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Comparer l'efficacité de l'apport exogène de 40ppm de NO inhalé par rapport à un gaz placebo (azote, N2) sur le besoin en transfusion sanguine chez les enfants drépanocytaires avec syndrome thoracique aigu.
    E.2.2Secondary objectives of the trial
    Comparer entre le bras NO inhalé et bras placebo (N2) l'évolution de l'oxygénation, le
    besoin en antalgiques, le délai de transfusion et la quantité totale de sang transfusé, la
    durée d'hospitalisation, enfin la tolérance du traitement par le NO inhalé.

    Comparer entre le bras NO inhalé et bras placebo (N2) l'évolution de l'oxygénation, le
    besoin en antalgiques, le délai de transfusion et la quantité totale de sang transfusé, la
    durée d'hospitalisation, enfin la tolérance du traitement par le NO inhalé.

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Examen médical préalable
    - Enfant de 1-18 ans non révolu drépanocytaire homozygote SS ou équivalent (double hétérozygote Sß0thal) dont le poids est 65 kg.
    - Présentant un syndrome thoracique aigu sévère défini par la survenue d'une tachypnée (fréquence respiratoire normale pour l'âge >2DS), ou d'une gêne respiratoire, et/ou d'une toux, et/ou de douleurs thoraciques, et d'une fièvre supérieure à 38°C, avec la présence d'opacités alvéolaires radiologiques à type de condensation.
    - Hypoxémie définie par une SpO2 < 92% sous air
    - Consentement signé par les titulaires de l'autorité parentale, après information préalable par un des médecins du centre de drépanocytose.
    - Accord de participation de l'enfant, après information préalable s'il est apte à exprimer sa volonté.
    - Titulaire de l'autorité parentale affilié à un régime de sécurité sociale ou bénéficiaire de
    la CMU.
    E.4Principal exclusion criteria
    - Détresse respiratoire aiguë définie par une polypnée, une SpO2 <92% malgré un débit d'oxygène >5L/min par lunettes nasales ou par masque à haute concentration (FiO2 >40%), et/ou des signes de fatigue respiratoire (sueurs, altération de la conscience, PaCO2 > 60mmHg) qui nécessite le transfert de l'enfant dans l'unité de surveillance continue ou de réanimation pédiatrique pour échange transfusionnel ou intubation trachéale et ventilation mécanique.
    - Crise d'asthme aiguë isolée qui nécessite l'inhalation d'agents beta-adrénergiques et une corticothérapie p.os ou i.v.
    - Accident vasculaire aigu associé (thrombose cérébrale, priapisme) qui nécessitent un échange transfusionnel immédiat
    - Anémie aiguë définie par une diminution de l'hémoglobine >20% par rapport à l'hémoglobine de base (soit une Hb <6,4g/L pour une Hb de base à 8g/dL). [Dans les deux études de la Collaborative Study of Sickle Cell Disease,39, 40 la diminution moyenne de l'hémoglobine est de 10% par rapport à une Hb de base entre 7,5 et 8,5g/dL]
    - Programme d'échanges transfusionnels
    - Hypersensibilité au monoxyde d'azote
    - les patients présentant un défaut cardio-vasculaire rare dans lequel l'oxygénation systémique dépend entièrement d'un shunt droit-gauche extra-pulmonaire, l'administration de INOmax peut diminuer le flux sanguin de droite à gauche, ce qui peut être fatal dans cette situation.
    - Patient inclus précédemment dans le protocole
    - Patient participant à un autre essai clinique concomitant sur d'autres médicaments ou conduites thérapeutiques.
    - Titulaire de l'autorité parentale non affilié à un régime de sécurité sociale ou ne bénéficiant pas de la CMU.

    E.5 End points
    E.5.1Primary end point(s)
    Critère principal d'évaluation:
    Sera défini par la nécessité ou non de transfusion. Les critères de transfusion seront donc très stricts : persistance ou majoration des signes respiratoires (tachypnée, signes de lutte, douleurs thoraciques, toux) et d'une oxygéno-dépendance définie par une SpO2<92% sous air .
    ¦ Critères d'évaluation secondaire :
    seront :
    1) le nombre de transfusions et la quantité totale de sang transfusé ;
    2) les besoins en antalgiques notamment la dose cumulée de morphine nécessaire ;
    3) la durée du traitement par NO inhalé ;
    4) la durée de l'oxygénothérapie.

    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 No
    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) 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 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.8.3 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 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 Information not present in EudraCT
    E.8.7Trial has a data monitoring committee No
    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 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.1.1.1In 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) Yes
    F.1.1.5Children (2-11years) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    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 No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    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 Decision2009-09-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-08-14
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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