E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of complex perianal fistulas in patients without inflammatory bowel disease. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10002156 |
E.1.2 | Term | Anal fistula |
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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 |
Compare the efficacy of ASCs versus ASCs plus fibrin adhesive versus fibrin adhesive for closure of complex perianal fistulas not associated to inflammatory bowel disease. Fistula closure is defined as absence of suppuration of the fistula through the external orifice, spontaneously and by pressure, and complete re-epithelization of the external orifice in the clinical evaluation and absence of collections >2 cm. directly related to the fistula tract treated, as measured by MRI |
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E.2.2 | Secondary objectives of the trial |
1. Evaluate the changes over time in the clinical complexity of the fistula in patients treated with ASCs, ASCs plus fibrin adhesive, and fibrin adhesive alone. 2. Compare the efficacy of ASCs vs ASCs plus fibrin adhesive and fibrin adhesive alone, for clinical evaluated closure of complex perianal fistulas not associated to inflammatory bowel disease. 3.Evaluate the safety in patients treated with ASCs vs ASCs plus fibrin adhesive versus fibrin adhesive alone. 4.Evaluate the quality of life of patients treated with ASCs in comparison to those treated wit ASCs plus fibrin adhesive and with fibrin adhesive alone (SF36 score). 5. Evaluate the degree of fecal incontinence (if any) using Wexner clinical scale (Wexner incontinence score) in patients treated with ASCs in comparison to those treated wit ASCs plus fibrin adhesive and with fibrin adhesive alone 6.Evaluate the number of surgeries avoided in patients treated with ASCs compared to those treated with fibrin adhesive alone |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
a.Patient with a complex perianal fistula amenable to local treatment with ASCs, as judged by the investigator after a clinical examination and MRI have been conducted. b.Patient of any sex aged 18 years or older c.Women of childbearing age should have a negative serum or urine pregnancy test and not be lactating before study entry. Both men and women should use adequate birth control methods as defined by the investigator. d.Seton presence is allowed at the time of study entry and until cells (in groups receiving ASCs) or fibrin adhesive are implanted. e.No condition that may prevent the patient from following the study procedures until 26 weeks of follow?up are completed is foreseen. f.Patient should give his/her signed, written informed consent.
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E.4 | Principal exclusion criteria |
a.Patient has been diagnosed with inflammatory bowel disease, as documented by radiology/endoscopy/histopathology. b.Patient has a rectovaginal fistula. c.Patient is a pregnant or lactating woman d.Patient has acute sepsis at the time of study entry e.A liposuction to draw al least 100 cc3 of fat from the abdominal wall is not technically feasible, or does not want this procedure to be performed. f.Patient needs surgery in the perianal region for reasons other than fistulas at the time of study entry, or is expected to possibly need any type of surgery in that area in the next 26 weeks. Abscess drainage is permitted. g.Presence of two or more complex perianal fistulas. h.Patient has collections >2 cm in MRI. If such collections exist, the patient may participate in the study if the investigating surgeon performs a complete toilette of the area, draining collections, and the absence of such collections or other collections >2 cm is shown in a subsequent MRI. i.Patient is allergic to local anesthetics or Gadolinium (MRI contrast agent)
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion or patients in whom the external openings of the treated perianal fistula are closed clinically evaluated (absence of suppuration through the external opening of the fistula, spontaneously and by pressure, and the complete re-epithelization of the external opening) and also by MRI (absence of collections >2cm associated to the fistulous tract) after the ASC treatment cycle (1 + 1) with a dose of approximately 20 million ASCs in the first administration and 40 millions in the second administration, compared to the proportion of patients treated with ASCs plus fibrin adhesive or with fibrin adhesive alone, 24 weeks after receiving the first dose of study medication (26 in patients with a second dose.) |
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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 | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
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) | Yes |
E.8.2.2 | Placebo | Yes |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 10 |
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 | Information not present in EudraCT |
E.8.7 | Trial 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
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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 | 18 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 18 |