E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10016296 |
E.1.2 | Term | Fecal incontinence |
E.1.2 | System Organ Class | 100000004856 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Assessment of safety and efficacy of study treatment |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients who participated in the phase IIb STEFFI study (IC-01-02-02-007) and were randomized to placebo 2. Patients must agree to read and sign the Informed Consent (IC) form prior to any study-related procedures 3. Patients willing and able to comply with the study procedures 4. Patients who are mentally competent and able to understand all study requirements 5. Female patients of childbearing potential willing to use appropriate methods of contraception. Women considered of childbearing potential shall only be included in the study after a confirmed menstrual period and a negative pregnancy test.
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E.4 | Principal exclusion criteria |
1. Patients who, according to the clinical judgment of the investigator, are not suitable for this study 2. 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 3. Patients who are currently participating or have participated in another clinical study (test-ing a medical device or drug) within 30 days prior to screening Visit 1 4. Patients dependent from the sponsor, CRO, or the investigator (e.g. employees, relatives, etc.) 5. Female patients who are pregnant, lactating, or intending pregnancy in the near future and female patients of childbearing potential who are not willing to use appropriate methods of contraception up to Visit 5 or who have a positive pregnancy test (only to be performed in women of childbearing potential) 6. Patients who underwent any anorectal surgery within 6 months before screening visit 7. Patients with indications against a surgery under anesthesia 8. Patients with a malignant disease not in remission for 5 years or more 9. Patients who have undergone radiation therapy of the bowel and pelvis 10. Patients who have undergone chemotherapy within last 5 years prior to study enrolment and/or chemotherapy related neuropathy of the bowel and pelvis 11. Patients with compromised immune system and/or rheumatic disease, and patients under immunosuppressive therapy 12. Patients suffering from a disease which has not been resolved within 4 weeks prior to screening including fever and/or diarrhea of unknown reasons (4 weeks) 13. 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]), gentamycin) 14. Patients diagnosed with human immunodeficiency virus (HIV), acute or chronic viral hepa-titis HCV, acute or chronic viral hepatitis HBV, active Syphilis or HTLV (tested upon risk assessment by investigator) 15. Patients diagnosed with any kind of skeletal muscle disease and/or neuronal disorders 16. Patients with severe myocardial disorders, irregular pulse or a pacemaker 17. Patients with implantations of metal components in the electrical stimulation treatment area 18. Patients with uncontrolled diabetes mellitus type I or II, or suffering from diabetic peripheral neuropathic pain 19. Patients with clinically relevant abnormal laboratory values judged by the responsible investigator as relevant for the study treatment |
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E.5 End points |
E.5.1 | Primary end point(s) |
Changes in frequency of incontinence episodes as measured by the incontinence diary records prior to Visit 5 compared to the baseline period from diary records prior to biopsy. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Visit 5 (3 months after implantation) versus baseline ( 3 weeks after screening) |
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E.5.2 | Secondary end point(s) |
1.Suitability to use an e-diary for incontinence recording 2.Safety (Physical examination, vital signs, standard hematology, blood chemistry) 3.Other (Pregnancy, concomitant medication) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. Visit 5 (3 months after implantation versus Visit 2) 2. Visit 1 (screening), 2 (21 days after screening at biopsy), 3 (day of implantation), 4 (1 day after implantation), 5 (3 months after implantation) 3. Visit 1 (screening), 2 (21 days after screening at biopsy), 3 (day of implantation), 4 (1 day after implantation), 5 (3 months after implantation) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial 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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |