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    Summary
    EudraCT Number:2010-021463-32
    Sponsor's Protocol Code Number:IC-01-02-02-007
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2010-11-30
    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 number2010-021463-32
    A.3Full title of the trial
    Skeletal muscle-derived cell implantation for the treatment of fecal incontinence: a multicenter, randomized, double-blind, placebocontrolled, parallel-group, dose-finding clinical study
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The aim of this clinical study is to show the clinical efficacy and safety of SMDCs in patients suffering from FI, and to find the optimal cell count for the functional regeneration of the external anal sphincter.
    A.3.2Name or abbreviated title of the trial where available
    STEFFI
    A.4.1Sponsor's protocol code numberIC-01-02-02-007
    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 SponsorInnovacell Biotechnologie AG
    B.1.3.4CountryAustria
    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 supportInnovacell Biotechnologie AG - Life Science Center
    B.4.2CountryAustria
    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 nameautologous Skeletal Muscle Derived Cells
    D.3.2Product code aSMDC
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeaSMDC
    D.3.9.3Other descriptive nameautologous Skeletal Muscle Derived Cells
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number4000000 to 6000000
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberEMEA/CAT/674819/2009
    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.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 nameautologous Skeletal Muscle-Derived Cells
    D.3.2Product code aSMDC
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntramuscular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeaSMDC
    D.3.9.3Other descriptive nameautologous Skeletal Muscle Derived Cells
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number40000000 to 60000000
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberEMEA/CAT/674819/2009
    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 placeboSolution for injection
    D.8.4Route of administration of the placeboIntramuscular use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fecal incontinence in female and male patients with external anal sphincter weakness or sphincter damage.
    E.1.1.1Medical condition in easily understood language
    Fecal incontinence is the involuntary loss of flatus and/or solid or liquid stool or the inability to control the discharge of fecal matter through the anus.
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10016296
    E.1.2Term Fecal incontinence
    E.1.2System Organ Class 100000167639
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this study is to find the optimal cell count for functional regeneration of the external anal sphincter.
    E.2.2Secondary objectives of the trial
    Efficacy variables:
    -Changes in frequency of incontinence episodes until Day 90 compared to the baseline
    -Change in the Wexner score at day 90 and day 180 compared to baseline
    -Change in the Visual Analogue Scale at day 90 and day 180 compared to baseline
    -Frequency of response measured as a reduction of the frequency of incontinence episodes by more than 20 %, 50%, 75% and 90%, under treatment compared to the baseline
    -Frequency of patients in remission
    -Changes in the anorectal manometry data at day 180 compared to baseline
    -Patient’s assessment based on the QoL questionnaire score
    -Investigator’s assessment by the CGI score
    -Change in the volume or thickness of external anal sphincter assessed by 3D sonography
    -Examinations based on the incontinence diary records
    -Pharmacoeconomic evaluation

    Safety variables:
    -Physical examination
    -Vital signs
    -Standard hematology, blood chemistry, and urinalysis
    -Pregnancy test
    -Concomitant medication
    -AEs and SAEs
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients of > or = 18 years of age,
    2. Patients suffering from FI for more than 6 months, which is confirmed at screening by relevant medical history and anorectal examination,
    3. Patients with Wexner score >9,
    4. Patients with no indications against a surgery under anesthesia,
    5. Patients willing and able to comply with the study procedures,
    6. Patients who are mentally competent and able to understand all study requirements,
    7. Patients must agree to read and sign the Informed Consent (IC) form prior to any study-related procedures,
    8. Female patients of childbearing potential willing to use acceptable methods of contraception (birth control pills, barriers, or abstinence).

    Interim Inclusion Criterion:
    Patients with a minimum of 3 incontinence episodes per week measured at visit -1 (diary distributed at screening visit)
    E.4Principal exclusion criteria
    1. Patients with pathological findings (excluding sphincter damage) based on rectoscopy and ultrasound at the screening visit,
    2. Patients who have undergone any anorectal surgery within the last 6 months prior to screening visit,
    3. Patients with maximal one overlap repair in total,
    4. Patients with more than 2 anorectal surgical procedures, (but maximal one overlap repair in total) e.g.
    - primary repair after delivery and one overlap repair later-on,
    - in- and explantation of a permanent neurostimulation system,
    5. Patients with overlap repair and associated early atrophy of external anal sphincter,
    6. Patients with a history of artificial anal sphincter surgery,
    7. Patients with trans- or perianal injection of any bulking products,
    8. Patients with a malignant disease not in remission for 5 years or more,
    9. Patients who had undergone radiation therapy,
    10.Patients who had undergone chemotherapy,
    11. Patients with compromised immune system and/or rheumatic disease,
    12. Patients under immunosuppressive therapy,
    13. Patients with a diagnosis of chronic inflammatory bowel disease,
    14. Patients with recurrent anal fistula disease,
    15. Patients with chronic diarrhea,
    16. Patients suffering from a disease which has not been
    resolved within a timeframe prior to screening as follows:
    fever and/or diarrhea of unknown reasons (4 weeks), HAV
    (4 months), toxoplasmosis (6 months), osteomyelitis, Qfever, rheumatic fever, tuberculosis, or Salmonella infections (2 years), and malaria (4 years).
    17. Patients who, according to the clinical judgment of the investigator, are not suitable for inclusion due to acute anal sphincter injury including obstetric and other trauma, acute disc prolapse, or neurological diseases (spinal cord injury, multiple sclerosis, Parkinson’s disease, stroke, etc.),
    18. Patients with uncontrolled diabetes mellitus type I or II, or suffering from diabetic peripheral neuropathic pain,
    19. Patients diagnosed with human immunodeficiency virus (HIV), acute or chronic viral hepatitis HCV, acute or chronic viral hepatitis HBV, active Syphilis, HTLV (tested upon risk assessment by investigator),
    20. Patients diagnosed with any kind of skeletal muscle disease and/or neuronal disorders,
    21. Patients with known hypersensitivity to any component of the product (autologous cells, ringer’s lactate, human serum albumin, DMSO, bovine proteins, fibroblast growth factor [FGF]),
    22. Patients with clinically relevant abnormal laboratory values, any persistent chronic bacterial infections as well as local infections as indicated by a high level of the C-reactive protein (CRP) of >35 mg/l and confirmed by bacteriological analysis, or with any bleeding disorder,
    23. Patients who, according to the clinical judgment of the investigator, are not suitable for this study,
    24. Patients who are currently participating or have participated in another clinical trial (testing a medical device or drug) within 30 days prior to the study begin or have previously participated in the current clinical study,
    25. Patients who are pregnant, lactating, or intending pregnancy in the near future, and those of childbearing potential who are not willing to use acceptable methods of contraception (birth control pills, barriers, or abstinence) or who have a positive pregnancy test (only to be performed in women of childbearing potential),
    26. Patients dependent from the sponsor, CRO, or the investigator (e.g. employees, relatives, etc.).
    27. Patients deprived of their liberty by a judicial or administrative decision, patients admitted to a hospital, social institution or who are under a measure of legal protection, patients hospitalized without consent or who are in an emergency situation.
    28. Patients with severe myocardial disorders, irregular pulse or a pacemaker,
    29. Patients with implantations of metal components in the electrical stimulation treatment area


    Interim Exclusion Criterion:
    Patients with persistent bacterial infections confirmed by clinical signs and positive results in bacteriological testing at visit-1 (Salmonella (Typhus), F. tularensis (Tularaemia), M. leprae (Leprosy), Brucella, Rickettsia).
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the current study is the frequency of incontinence episodes occurred under treatment (from baseline until Day 180) compared to the baseline period (day -28 to day 0), in each treatment group.
    E.5.1.1Timepoint(s) of evaluation of this end point
    - 2 weeks before the biopsy (day -84 to day -70),
    - 4 weeks before the implantation (day -28 to day 0),
    - 4 weeks after the implantation (day 1 to 29),
    - and 4 weeks before V3 (day 62 to 90) and V4 (day 152 to 180)
    E.5.2Secondary end point(s)
    Efficacy variables:
    -Changes in frequency of incontinence episodes until Day 90 compared to the baseline
    -Change in the Wexner score at day 90 and day 180 compared to baseline
    -Change in the Visual Analogue Scale at day 90 and day 180 compared to baseline
    -Frequency of response measured as a reduction of the frequency of incontinence episodes by more than 20 %, 50%, 75% and 90%, under treatment compared to the baseline
    -Frequency of patients in remission
    -Changes in the anorectal manometry data at day 180 compared to baseline
    -Patient’s assessment based on the QoL questionnaire score
    -Investigator’s assessment by the CGI score
    -Change in the volume or thickness of external anal sphincter assessed by 3D sonography
    -Examinations based on the incontinence diary records
    -Pharmacoeconomic evaluation

    Safety variables:
    -Physical examination
    -Vital signs
    -Standard hematology, blood chemistry, and urinalysis
    -Pregnancy test
    -Concomitant medication
    -AEs and SAEs
    E.5.2.1Timepoint(s) of evaluation of this end point
    Screening (visit -2), visit -1, visit 0, visit 1, visit 2, visit 3 and visit 4
    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 Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    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.8.2.4Number of treatment arms in the trial3
    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 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 No
    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
    Sponsor made the decision to stop the study based on the results of on 6 and 12 month analysis for futility within the 24 months follow-up phase (3 rd of August 2017). Continuing with the trial would not contribute to additional safety or efficacy information compared to the results obtained at 6 and 12 months.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months21
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months21
    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 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: 227
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 25
    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 state32
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 252
    F.4.2.2In the whole clinical trial 252
    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)
    The inclusion in a separate follow-up study for additional 24 months is planned for all patients from this phase IIb study.
    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 Decision2011-02-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-12-13
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2017-08-04
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