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    Summary
    EudraCT Number:2008-003801-15
    Sponsor's Protocol Code Number:12011.202
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-01-08
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2008-003801-15
    A.3Full title of the trial
    A phase II, randomised, double-blind, placebo controlled, parallel group,
    dose-finding clinical trial to investigate the efficacy and safety of 10 and 20
    mg/day aerosolised liposomal ciclosporin A (L-CsA) versus placebo in the
    treatment of bronchiolitis obliterans syndrome (BOS) in allogeneic
    haematopoietic stem cell transplant (HSCT) patients
    A.4.1Sponsor's protocol code number12011.202
    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 SponsorPARIPharma GmbH
    B.1.3.4CountryGermany
    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 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 No
    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/3/04/210
    D.3 Description of the IMP
    D.3.1Product nameAerolised Liposomal Ciclosporin A
    D.3.2Product code L-CsA
    D.3.4Pharmaceutical form Powder for nebuliser solution
    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 INNCiclosporin
    D.3.9.1CAS number 59865-13-3
    D.3.9.2Current sponsor code081400
    D.3.9.3Other descriptive nameCiclosporine, ciclosporina
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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) 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 Yes
    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.IMP: 2
    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 nameSodium Chloride Solution
    D.3.2Product code Sodium Chloride solvent
    D.3.4Pharmaceutical form Nebuliser solution
    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.3.9.2Current sponsor codeSodium Chloride
    D.3.9.3Other descriptive nameSodium Chloride
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.25
    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 placeboPowder for nebuliser 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
    Bronchiolitis Obliterans Syndrome
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10049202
    E.1.2Term Bronchiolitis obliterans
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to establish an IMP dosage with the most favourable risk-benefit ratio for the treatment of BOS in allogeneic HSCT patients.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to compare efficacy and safety data from IMP versus placebo and to evaluate IMP PK data in whole blood and sputum samples.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject’s written informed consent obtained prior to any screening procedure. For subjects below 18 years of age, the written informed consent is obtained from the subject’s legal representative
    2.Received an allogeneic haematopoietic stem cell transplantation (HSCT)
    3.Has a baseline FEV1 value available prior to HSCT according to Appendix III
    4.Has a diagnosis of bronchiolitis obliterans (BOS) of grade 1, 2 or 3 and confirmation to initiate BOS-specific treatment based on declining FEV1 values according to Appendix III within 1 week prior to first IMP administration
    5.Male or female, >/= 12 years of age
    6.Capable of self-administration medications
    7.Capable of understanding the purpose and risk of the clinical trial
    8.Received at least 1 week prior to first IMP administration the following immunosuppressive agents and dosages for the treatment of chronic graft-versus-hostdisease (GVHD) including BOS:
    a)Tacrolimus approximately 0.1 to 0.2 mg/kg/day adjusted to a target trough serum level (C0) of 5 to 15 µg/L
    b)Prednisone 1 to 3 mg/kg/day for 2 to 6 weeks, tapered down subsequently
    9.Female patients with reproductive potential must have a negative serum pregnancy test within 3 days prior to screening. Both women and men must agree to use a medically acceptable method of contraception throughout the IMP treatment period and for 3 months after IMP discontinuation. Acceptable methods of contraception are disclosed in Appendix II of the protocol
    10.Estimated life expectancy > 6 months
    E.4Principal exclusion criteria
    1. Received systemic or topical ciclosporin A within one week prior to IMP administration and during the clinical trial
    2.Any active invasive bacterial, viral or fungal infection within one week prior to first IMP admiistration
    3.Received systemic immunosuppressive therapy for chronic GVHD other than listed in the inclusion criteria within one week prior to first IMP administration
    4.Has steroid-refractory or steroid-intolerant chronic GVHD due to insulin-dependent diabetes or symptomatic avascular necrosis
    5.Current mechanical ventilation
    6.Pregnant or breast-feeding woman
    7.Has a known hypersensitivity to ciclosporin A
    8.Has a serum creatinine value of more than 265 µmol/L (3 mg/dL) or chronic dialysis (haemodialysis)
    9.Unlikely to comply with the visits, inhalation procedures or spirometric measurements scheduled in the protocol
    10.Receipt of an investigational drug as part of a clinical trial within 4 weeks prior to first administration of IMP
    11.Any co-existing medical condition that in the investigator’s judgement will substantially increase the risk associated with the subject’s participation in the clinical trial
    12.Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary procedures
    13.Subject was previously included in the present clinical trial
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Endpoint
    • Mean forced expiratory volume in one second (FEV1) at baseline, 3, 6, 9 and 12 month after first IMP administration
    • Mean FEV1 slope from baseline to 3, 6, 9 and 12 months after first IMP administration
    • Mean forced midexpiratory flow (FEF25-75), vital capacity (VC) and total lung capacity (TLC) at baseline, 3, 6, 9 and 12 months after first IMP administration
    • Mean single breath diffusing capacity (DLCO) and capillary blood gases at baseline, 3, 6, 9 and 12 months after first IMP administration
    • Cumulative mean incidence of invasive bacterial, viral or fungal infections 3, 6, 9 and 12 months after first IMP administration
    • Cumulative mean incidence of acute GVHD at baseline, 3, 6, 9 and 12 months after first IMP administration
    • Cumulative mean incidence of chronic GVHD other than BO at baseline, 3, 6, 9 and 12 months after first IMP administration
    • Mean walking distance from 6 min walk test at baseline, 3, 6, 9 and 12 months after first IMP administration
    • Mean cumulative dose of systemic immunosuppressants 3, 6, 9 and 12 months after first IMP administration
    • Mean cumulative number of days without systemic immunosuppressants 3, 6, 9 and 12 months after first IMP administration
    • Mean cumulative number of overnight hospital stays 3, 6, 9 and 12 months after first IMP administration
    • Mean levels of inflammatory markers and L-CsA from sputum from at least two visits
    during the clinical trial period
    • Cumulative overall survival during the clinical trial period

    Safety
    • Incidence of AEs including clinically relevant laboratory findings until EoS
    • Mean L-CsA blood levels at baseline and 3 months after first IMP administration
    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 Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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 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 years
    E.8.9.1In the Member State concerned months18
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months18
    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) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 Information not present in EudraCT
    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
    This refers to the adolescent group of subjects aged 12 - 17 years. The consent will be signed by a legally acceptable representative of the subject.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state19
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 70
    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 Decision2009-03-12
    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 StatusCompleted
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