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    Summary
    EudraCT Number:2008-004562-10
    Sponsor's Protocol Code Number:BUM-5/GVH
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2009-01-13
    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
    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 ConcernedAustria - BASG
    A.2EudraCT number2008-004562-10
    A.3Full title of the trial
    Double-blind, randomised, placebo-controlled multicentre phase III clinical study followed by open-label phase on the efficacy and tolerability of budesonide 3 mg effervescent tablet in patients with resistant oral chronic GvHD
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    In this clinical study, a new drug, Budesonid 3 mg effervescent tablet for
    the preparation of a mouth rinse solution to treat oral chronic GvHD
    (Graft versus Host Disease) resulting from allogeneic (taken from
    different individuals of the same species) haematopoietic (blood cell
    forming) stem cell transplantation (HSCT), is evaluated.
    A.4.1Sponsor's protocol code numberBUM-5/GVH
    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 SponsorDr. Falk Pharma 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 supportDr. Falk Pharma GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDr. Falk Pharma GmbH
    B.5.2Functional name of contact pointProjectmanagment
    B.5.3 Address:
    B.5.3.1Street AddressLeinenweberstr. 5
    B.5.3.2Town/ cityFreiburg
    B.5.3.3Post code70108
    B.5.3.4CountryGermany
    B.5.4Telephone number+497611514156
    B.5.5Fax number+497611514377
    B.5.6E-mailmohrbacher@drfalkpharma.de
    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/3/06/413
    D.3 Description of the IMP
    D.3.1Product nameBudesonide
    D.3.2Product code Budesonid effervescent tablet
    D.3.4Pharmaceutical form Effervescent tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOromucosal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNbudesonide
    D.3.9.1CAS number 51333-22-3
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3
    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 placeboEffervescent tablet
    D.8.4Route of administration of the placeboOromucosal use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Oral chronic graft versus host disease as a complication of haematopoietic stem cell transplantation.
    E.1.1.1Medical condition in easily understood language
    Oral chronic GvHD is a disease that developed as a consequence of allogeneic (taken from different individuals of the same species) haematopoietic (blood cell forming) stem cell transplantation
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10066261
    E.1.2Term Chronic graft versus host disease
    E.1.2System Organ Class 10021428 - Immune system disorders
    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 the budesonide 3 mg effervescent tablet rinse versus placebo rinse in decreasing the severity of oral cGvHD and oral cGvHD-related mouth pain.
    E.2.2Secondary objectives of the trial
    To assess the rinse´s safety and tolerability in the form of adverse events and laboratory parameters.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed informed consent
    2. Man or woman between 18 and 75 years of age
    3. Karnofsky≥ 70
    4. Oral chronic GvHD after allogeneic haematopoietic stem cell transplantation (cGvHD defined as GvHD present beyond day 100 post-transplantation)
    5. Oral cGvHD confirmed by presence of at least one diagnostic clinical sign of cGvHD or presence of at least one distinctive manifestation confirmed by pertinent biopsy or other relevant tests
    6. Oral cGvHD of erosive and/or ulcerative type
    7. NIH scale (15-point Schubert scale) ≥ 3 but at least score 3 (moderate) for ulcers or at least score 2 (moderate) for erythema (lichenoid has to be present in case of absence of ulcers)
    8. Resistant oral cGvHD with no oral response defined as the lack of partial response to conventional primary treatment (i.e. systemic prednisone and/or cyclosporine) after 4 weeks or no need for systemic immunosuppression due to oral cGvHD only (mild involvement of 2nd organ allowed, excluding lung involvement) for at least 12 weeks
    9. In case of conventional primary treatment (i.e. systemic prednisone and/or cyclosporine) dosage unchanged within the last 4 weeks before inclusion (dose reduction and dose adjustment by therapeutic drug monitoring [TDM] allowed); 2 weeks acceptable in case of oral cGvHD for more than 6 months without any response to high-dose systemic prednisone and/or cyclosporine
    10. In case of treatment with tacrolimus (FK506), sirolimus (rapamycin), or mycophenolate mofetil (MMF) dosage unchanged within the last 4 weeks before inclusion (dose reduction and dose adjustment by TDM allowed)
    11. Negative pregnancy test at baseline visit week 0 in females of childbearing potential
    12. Male or female patients with reproductive potential must use an approved contraceptive method during study treatment evaluation
    E.4Principal exclusion criteria
    1. Uncertain diagnosis of resistant oral cGvHD
    2. Symptomatic oral cGvHD of hyperkeratotic type solely
    3. Current active oral bacterial, viral, or fungal infection
    4. Unwilling to forego concurrent treatment for mucosal lesions and/or related oral pain except prophylactic treatment to prevent infection (see 5.7 Standard of Care)
    5. Requiring addition of new systemic therapy including steroids, or radiation therapy
    6. Local intestinal infection
    7. Abnormal hepatic function or liver cirrhosis(ALT or AST > 3 x ULN); in case of hepatic cGvHD: Bilirubin > 3 x ULN and/or ALT or AST > 5 x ULN
    8. If careful medical monitoring is not ensured: tuberculosis, cardiovascular disease, diabetes mellitus, osteoporosis, active peptic ulcer disease, glaucoma, cataract, infection
    9. Second line treatment of oral cGvHD with topical steroids (e.g. dexamethasone, beclomethasone) within the last 4 weeks
    10. Use of inhaled steroids for the treatment of e.g. COPD or bronchiolitis
    11. Treatment with low dose total lymphoid irradiation, intraoral PUVA, extracorporeal
    photochemotherapy (allowed after 6 months of unsuccessful therapy with unchanged
    dosage within the last 4 weeks before inclusion; increase of dosage after study start not allowed), thalidomide, pentostatin within the last 4 weeks
    12. Treatment with ketoconazole, itraconazole or other CYP3A inhibitors except fluconazole, posaconazole, voriconazole, cyclosporine, and tacrolimus/FK506 (increase of dose not allowed, see inclusion criteria 9 and 10)
    13. Unable to demonstrate appropriate use of study medication
    14. Existing or intended pregnancy or lactation
    15. Known intolerance/hypersensitivity to study drugs or drugs of similar chemical structure or pharmacological profile
    16. Participation in another clinical study for the treatment of cGvHD within the last 30 days or simultaneous, or previous participation in this study
    17. Well-founded doubt about the patient’s cooperation, e.g. because of addiction to alcohol or drugs
    E.5 End points
    E.5.1Primary end point(s)
    Rate of objective response at the final/withdrawal visit using NIH scale (15-point Schubert scale) and WHO response criteria.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Study Visit 2-12
    E.5.2Secondary end point(s)
    Rate of response as defined by NIH
    • Rate of complete/partial response,
    stable disease, progressive disease as
    defined by WHO,
    • Time to initial objective response,
    • Rate of improvement in any of the
    subscores of NIH scale
    • Rate of subjective improvement
    (pain, swallowing, swallow foods or
    liquids),
    • Reduction in pain,
    • Duration of mouth pain and use of
    opiate analgesics following
    treatment,
    • Assessment of salivary secretion,
    • Karnofsky Performance Status,
    • Duration of use of opiate analgesics,
    • Weight loss/gain,
    • Assessment of efficacy by
    investigator
    E.5.2.1Timepoint(s) of evaluation of this end point
    Study Visit 1-12
    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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA37
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Austria
    Czech Republic
    France
    Germany
    Israel
    Italy
    Poland
    Sweden
    Switzerland
    Turkey
    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
    last patient out (LPO)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    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 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 170
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 54
    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 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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 148
    F.4.2.2In the whole clinical trial 224
    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)
    In the case of need of futher treatment after total treatment duration of 52 weeks patients can enter a compassionate use program organised by the sponsor of the trial.
    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 Decision2009-03-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-02-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-08-24
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