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    Summary
    EudraCT Number:2019-004939-24
    Sponsor's Protocol Code Number:BPF-001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-02-04
    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 ConcernedUK - MHRA
    A.2EudraCT number2019-004939-24
    A.3Full title of the trial
    Phase I/II open-label study to assess the safety, tolerability and efficacy of a novel tissue engineered airway product, consisting of expanded autologous bone marrow (BM) derived Mesenchymal Stromal Cells (MSC) seeded on to a decellularised allogeneic patch of an airway scaffold in subjects with clinically significant bronchopleural fistula.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/II, open label study to assess the safety and efficacy of expanded autologous bone marrow (BM) derived Mesenchymal Stromal Cells (MSC) seeded on to decellularised allogeneic patch of an airway scaffold in subjects with clinically significant Bronchopleural Fistula.
    A.4.1Sponsor's protocol code numberBPF-001
    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 SponsorCell and Gene Therapy Catapult
    B.1.3.4CountryUnited Kingdom
    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 supportVideregen
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCell and Gene Therapy Catapult
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address12th floor, Tower Wing, Guys Hospital
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE1 9RT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+442037289500
    B.5.6E-mailregulatory@ct.catapult.org.uk
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameTissue engineered autologous cell seeded allogeneic airway scaffold
    D.3.4Pharmaceutical form Living tissue equivalent
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPImplantation
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Bronchopleural fistula (BPF)
    E.1.1.1Medical condition in easily understood language
    A bronchopleural fistula is an abnormal passageway between the large airways in the lungs and pleural cavity.
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10053481
    E.1.2Term Bronchopleural fistula
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • Safety: To assess the safety of the engineered airway tissue product during 3 months (90 days) post defect repair
    • Efficacy: To determine BPF closure at 3 months post defect repair.
    E.2.2Secondary objectives of the trial
    • To assess impact on the subject’s Quality of Life (QoL) of the engineered airway tissue
    product patch at 3, 6, 9,12, 24 and 36 months following defect repair.
    • To assess the viability and the integrity of the repair area visually via bronchoscopy at 6,
    9, 12 months. Integrity assessed by visual appearance and no clinical signs of leaks.
    • To assess safety of the TE airway patch at 6, 9,12, 24 and 36 months following defect
    repair.
    • Incidence of interventions at the operative sites up to 6, 9, 12, 24 and 36 months.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male or female subjects 18 years or older.
    • Documented diagnoses of BPF through imaging and bronchoscopic examination.
    • BPF which involves the tracheobronchial junction or proximal bronchus.
    • Subjects who have failed primary repair.
    • Subjects who have no evidence of any primary or recurrent cancer (not limited to the surgical site) at the time of pre-operative screening as evidenced by CT and/or targeted biopsy (except for controlled or controllable basal cell carcinoma.
    • Subjects who have signed and dated written informed consent to participate in the study.
    • Females of childbearing potential (i.e. not surgically sterilised or post-menopausal for at least 2 years) must have a negative serum or urine pregnancy test.
    • Male and female subjects of childbearing potential (i.e. not surgically sterilised or post-menopausal for at least 2 years) must use forms of highly effective methods of contraception, which are defined as hormonal methods of contraception (oral, injection or implant), barrier methods (condom or occlusive cap (diaphragm or cervical/vault caps)) with spermicidal foam/gel/cream/film/suppository, or true abstinence for 1 month following surgery.
    • Subjects who have produced viable cells from BMA.
    E.4Principal exclusion criteria
    • Subjects who have received previous treatment with another Advanced Technology Medicinal Product (ATMP).
    • Subjects with ECOG performance status of 3 or 4.
    • Subjects deemed not suitable for surgery by the MDT.
    • Uncontrolled diabetes, defined as HbA1c levels above 7.0 %.
    • Any medical condition contraindicating the ability to tolerate general anaesthetic in the judgement of a consultant anaesthetist.
    • Subjects who have a severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or in the judgment of the investigator, would make the subject inappropriate for entry into this trial.
    • Subjects with clinically significant renal and liver impairment.
    • Subjects undergoing an immunosuppression regimen or suffering from a primary immunodeficiency syndrome.
    • Subjects with any known hypersensitivity to the culture and transport media compounds.
    • Subjects with current or recurrent disease that could affect the administration, the action or disposition of the investigational product, or clinical or laboratory assessments.
    • Subjects with clinically relevant or recent (within 2 years) history of substance abuse, including alcohol.
    • Subject who has participated in any other interventional clinical trial within previous 30 days of the start of this study.
    • Subjects with known presence of Human Immunodeficiency Virus (HIV) antibody, Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody.
    • Subject who, in the investigator’s judgement, is unlikely to complete all protocol required study visits or procedures, including follow-up visits, or comply with the study requirements for participation
    • Subjects with any medical condition, that in the investigator’s judgement, is likely to interfere with assessment of safety or efficacy of study treatment.
    • Subject who is pregnant.
    • Subjects with cancer (except for controlled or controllable basal cell carcinoma).
    .
    E.5 End points
    E.5.1Primary end point(s)
    • Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF implant during the 3 months post-defect repair.
    • Efficacy: BPF closure assessed by visual appearance and no clinical signs of leaks.
    E.5.1.1Timepoint(s) of evaluation of this end point
    • Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF
    implant during the 3 months post-defect repair.
    • Efficacy: BPF closure at 3 months assessed by visual appearance and no clinical signs
    of leaks.
    E.5.2Secondary end point(s)
    • Impact as assessed in change from Baseline in Physiological and QoL scores for each
    subject at 3, 6, 9, 12, 24 and up to 36 months post defect repair (physiological and
    QoLs): QoL EQ-5D, 6-minute walking test (6MWT), ambulatory O2 measurements.
    • Viability of the repair area and integration with vascular supply at 6, 9, 12 months.
    Adverse events related to the BPF implant procedures reported after 3, 6, 9, 12, 24
    and 36 months.
    • Interventions at the operative sites up to 6, 9, 12, 24 and 36 months.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Efficacy: closure of BPF with no need for further surgical closure. The trial will be considered to have demonstrated efficacy if 2 or more subjects meet the primary endpoint.
    • Safety and tolerability:
    - AEs including pre-defined postoperative AEs of special interest up to 30 ± 3 days post implantation
    - Related AEs reported after 3, 6, 9, 12, 24 and 36 months
    • Viability and the integrity of the repair area (closure) visually via bronchoscopy at 6, 9, 12, 24 and 36 months:
    - Integrity assessed by visual appearance of no visible leaks
    - Structural closure of the defect and visual health of the defect area and surrounding
    • Absence of other surgical interventions at 6, 9, 12, 24 and 36 months
    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) Yes
    E.7.1.1First administration to humans Yes
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    The subjects will act as their own internal historical control (-D56 to -D1)
    E.8.2.4Number of treatment arms in the trial1
    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 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
    The study will end when the last subject has undergone their last trial assessment, is withdrawn, or is
    lost to follow-up (whichever occurs last).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 5
    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 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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    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
    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 Decision2020-05-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-02
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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