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    Summary
    EudraCT Number:2013-004359-18
    Sponsor's Protocol Code Number:CCTU/2012/036
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2015-07-23
    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 number2013-004359-18
    A.3Full title of the trial
    RegenVOX: Phase I/IIa clinical trial of stem cell based tissue engineered partial laryngeal implants in adult patients with end-stage laryngotracheal stenosis with 24 months follow-up
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial of a voicebox implant using tissue engineering
    A.3.2Name or abbreviated title of the trial where available
    RegenVOX:Clinical trial of voicebox implants using tissue engineering
    A.4.1Sponsor's protocol code numberCCTU/2012/036
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01977911
    A.5.4Other Identifiers
    Name:UCL CCTU ID No. Number:2012/036
    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 SponsorUniversity College London (UCL) hereby represented by UCL Comprehensive Clinical Trials Unit(CCTU)
    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 supportMedical Research Council(MRC)
    B.4.2CountryUnited Kingdom
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAllogeneic tissue engineered autologous constituted partial larynx
    D.3.2Product code Tissue engineered partial larynx: TEP-PaL
    D.3.4Pharmaceutical form Implant
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPImplantation
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNo recommended International Nonproprietary Name (INN)
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeN/A
    D.3.9.3Other descriptive nameDecellularised allogeneic laryngeal scaffold
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1 scaffold
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNo recommended International Nonproprietary Name (INN)
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeN/A
    D.3.9.3Other descriptive nameAutologous human bone marrow (hBM) culture isolated and expanded mesenchymal stromal cells (MSCs)
    D.3.9.4EV Substance CodeAS3
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150000 cells/cm2
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Disorder of Upper Respiratory System
    Laryngostenosis
    Tracheal Stenosis
    E.1.1.1Medical condition in easily understood language
    Narrowing of the larynx (voicebox) and upper trachea (windpipe).
    E.1.1.2Therapeutic area Diseases [C] - Ear, nose and throat diseases [C09]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10062034
    E.1.2Term Laryngeal operation
    E.1.2System Organ Class 10042613 - Surgical and medical procedures
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10050816
    E.1.2Term Tracheal stenosis
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10023862
    E.1.2Term Laryngeal stenosis
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    We aim to assess the safety of stem cell based tissue engineered partial voice box replacement implants for the treatment of severe narrowing of the voice box and/or upper windpipe in adults.
    E.2.2Secondary objectives of the trial
    We also aim to assess the potential efficacy of stem cell based tissue engineered partial voice box replacement implants for the treatment of severe narrowing of the voice box and/or upper trachea.

    Our objectives are to assess whether this treatment is effective in terms of significantly improving functions of breathing, speaking and swallowing, improving quality of life for patients and reducing the burden on healthcare systems.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients aged >=18 years with sufficient numbers of Mesenchymal Stromal Cells (MSCs) in their 8-10ml human Bone Marrow (hBM) aspirate procured at screening for the production of adequate cell numbers for TEP-PaL.
    Patients with Myer-Cotton Grade 3 or 4* laryngotracheal stenosis or equivalent morbidity due to traumatic, inflammatory, iatrogenic, neoplastic (benign or low grade malignant) or idiopathic causes who have exhausted conventional therapies.
    (This will be determined by a fully constituted complex airway multidisciplinary team).

    *The Myer-Cotton grading system for mature, firm, circumferential stenosis, confined to the subglottis describes the stenosis based on the per cent relative reduction in cross-sectional area of the subglottis. Four grades of stenosis:
    • grade 1 lesions have less than 50% obstruction
    • grade 2 lesions have 51% to 70% obstruction
    • grade 3 lesions have 71% to 99% obstruction
    • grade 4 lesions have no detectable lumen or complete stenosis
    E.4Principal exclusion criteria
    Pregnancy.
    Those unable to provide informed consent.
    Co-morbid severe chronic obstructive pulmonary disease (COPD) (according to NICE clinical guideline CG101.
    Patients with active / uncontrolled chronic inflammatory conditions such as granulomatosis with polyangitis (formerly known as Wegener’s granulomatosis) and sarcoidosis.
    Any current or previous cancer within 5 years (except non-melanoma skin cancer, adequately treated carcinoma in situ of the uterine cervix, laryngeal malignancy treated locally without local recurrence or metastases or low grade airway tumours such as chondrosarcoma which may be causing airway obstruction.
    Life expectancy less than 5 years unless such limitation is largely due to the airway stenosis to be treated herein (as assessed by consultant trial clinician or referring clinician).
    Concurrent enrolment in any other CTIMP.
    Patients positive for HIV 1, HIV 2, HCV, HBV, syphilis or HTLV.
    E.5 End points
    E.5.1Primary end point(s)
    Safety as defined by mortality and morbidity as measured by occurrence of adverse events / reactions
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the whole study lifetime at each study visit up to 24 months post stage 2 implantation procedure.
    E.5.2Secondary end point(s)
    Efficacy as determined by:
    1. Absence of tracheostomy
    2. Absence of non-absorbable stent
    3. Improvement in mean airway diameter
    4. Improvement in FEV1
    5. Improvement in global quality of life (EQ-5D)
    6. Improvement in voice analysis operavox (VAO) including maximum phonation time (MPT)
    7. Improvement in self assessment of voice handicap (VHI-10)
    8. Improvement in swallowing function (EAT-10)
    9. Improvement in airway, dyspnoea, voice, swallowing index (ADVS index)
    10. Improvement in the penetration-aspiration scale (PAS) as per Video Fluoroscopic Swallow (VFS) or Functional Endoscopic Evaluation of Swallowing(FEES).
    Economic evaluations.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy as determined by:
    1. Absence of tracheostomy (6,12,24 months)
    2. Absence of non-absorbable stent (6,12,24 months)
    3. Mean airway diameter (6,12,24 months)
    4. FEV1 (1,2,6,12,24 months)
    5. Global quality of life (EQ-5D)(1,2,6,12,24 months)
    6. Voice analysis operavox (VAO) including maximum phonation time (MPT)(1,2,6,12,24 months)
    7. Self assessment of voice handicap (VHI-10)(1,2,6,12,24 months)
    8. Swallowing function (EAT-10) (1,2,6,12,24 months)
    9. Airway, dyspnoea, voice, swallowing index (ADVS index) (1,2,6,12,24 months)
    10. PAS as per Video Fluoroscopic Swallow (VFS) or Functional Endoscopic Evaluation of Swallow(FEES)(1wk, and 1,2,6, 12,24 months).
    Economic evaluations
    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 Yes
    E.6.13.1Other scope of the trial description
    Health Economic evaluation
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Early phase trial of an ATIMP surgically implanted in participants with laryngotracheal stenosis
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 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 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
    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 months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state10
    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)
    It is intended that the RegenVOX IMP will be a life-long treatment that will become part of the participant's own body, and eventually be replaced by native tissues. The effect of this trial will therefore be life-long and so participants may require follow up beyond the 24 months follow up period of the trial. The RegenVOX surgical team who perform the implantation operation are best placed to follow the participant up initially after the trial due to the specialist nature of this treatment.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-02-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-07-03
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-05-25
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