E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Perianal fistulas in Crohn's Disease Patients |
Fístulas perianales en enfermos de Crohn |
|
E.1.1.1 | Medical condition in easily understood language |
Lesions (fistulas) around the annus in patients with chronic inflamatory bowel disease (Crohn) |
Heridas (fistulas) alrededor del ano en pacientes con enfermedad crónica inflamatoria del intestino (Crohn) |
|
E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutic techniques [E02] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10068659 |
E.1.2 | Term | Perianal fistula |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011401 |
E.1.2 | Term | Crohn's disease |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
|
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 |
Main objective: To evaluate the safety and feasibility of intralesional platelet-rich plasma and fibrin clot from autologous origin, processed with PRGF-System tecnology (BTI), for the treatment of perianal fistulas in Crohn's disease. |
Objetivo principal: Evaluar la seguridad y factibilidad de la terapia de la enfermedad fistulosa perianal en enfermos de Crohn mediante de plasma rico en plaquetas y coagulo de fibrina de origen autólogo procesado mediante la tecnología PRGF-System (BTI) |
|
E.2.2 | Secondary objectives of the trial |
Secondary objectives: To evaluate the efficacy in terms of ratio of complex perianal fistula closure, decrease in the number of draining fistulas after two consecutive visits, and percentage of subjects with healed fistula (measured by MRI). |
Objetivos secundarios: Evaluar la posible eficacia en cuanto a cierre de fístulas perianales complejas, reducción en el número de fístulas de drenaje durante dos visitas consecutivas, y porcentaje de sujetos con cicatrización de la fístula (medida por RM). |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Age 18 or over / Confirmed diagnosis of Crohn's disease a year before the inclusion date / Presence of perianal fistula / Identifiable internal orifice / Demonstration activity of the fistulizing perianal pathology based on clinical (including PDAI) and radiological (pelvic MRI and endoanal ultrasound) criteria / Presence of 3 or less fistulous tracts / No inflamatory activity in the rectum or proctitis (confirmed by colonoscopy) / Written informed consent |
Edad mayor de 18 años/ Diagnóstico confirmado de enfermedad de Crohn un año antes de la fecha de inclusión/ Presencia de patología fistulizante perianal/Orificio interno identificable/ Demostración de actividad de la patología fistulizante perianal en base a criterios clínicos (incluyendo PDAI) y radiológicos (RM pélvica y ecografía endoanal)/ Presencia de 3 ó menos trayectos fistulosos/Ausencia de actividad inflamatoria rectal o proctitis (confirmada mediante colonoscopia) / Consentimiento informado obtenido por escrito |
|
E.4 | Principal exclusion criteria |
Superficial fistulas whose treatment of choice is fistulotomy / Presence of more than 3 fistulous tracts / Rectourethral and rectovaginal fistulas / Endoanal abscesses larger than 2 cm detected by physical examination or imaging (pelvic MRI endoanal ultrasound) / Current anorectal tumors / Clinically significant anal fissures or stenosis which prevent the rectoscopy or similar procedures / Impossibility of performing MRI for any reason (prosthesis, contrast allergy, claustrophobia ...) / Impossibility of performing endoanal ultrasound for any reason / Clinically inactive fistulizing disease (including PDAI) / Pregnant women or women in the first 6 months post-partum / Chemotherapy performed in the 6 months prior to study inclusion/ Bleeding diathesis or concurrent anticoagulant therapy / Prior radiation with evidence of radiation injury in the treatment area / Participation in any clinical trial during the 3 months prior to the screening visit / Other serious conditions or bio-psycho-social factors that may predict lack of compliance with study procedures / Major surgery or serious trauma of the subject in the previous semester / Congenital or acquired immunodeficiencies / Presence of surgical threads, unless they can be removed before starting treatment / Severe proctitis (striking friability, spontaneous bleeding, multiple erosions, deep ulcers), judging by the sigmoidoscopy / Malignancy in remission for less than a years before study inclusion, with the exception of basal cell carcinoma (BCC). |
Fístulas superficiales cuyo tratamiento de elección sea fistulotomía / Presencia de más de 3 trayectos fistulosos/Fístulas rectovaginales y rectouretrales/Presencia de abscesos de más de 2 cms detectados por exploración física o técnicas de imagen (RM pélvica o ecografía endoanal)/Tumores anorrectales actuales / Fisuras o estenosis anales clínicamente significativas que impidan la realización de rectoscopia o procedimientos similares / Imposibilidad de realización de RM por cualquier motivo que la contraindique (prótesis, alergia al contraste, claustrofobia?) / Imposibilidad de realización de ecografía endoanal por cualquier motivo que la contraindique / Pacientes con patología fistulizante clínicamente inactiva (incluyendo PDAI)/ Mujeres embarazadas o en los 6 meses postparto/Quimioterapia durante los 6 meses anteriores al estudio /Diátesis hemorrágica o terapiaanticoagulante actual /Radiación previa con evidencia de lesión por radiación en el área a tratar/Participación en cualquier otro estudio clínico durante los 3 meses anteriores a la visita pre-estudio/Pacientes con otros trastornos graves o factores bio-psico-sociales que hagan previsible la falta de cumplimiento con los procedimientos del estudio /Cirugía mayor o traumatismo grave del sujeto en el semestre anterior/Sujetos con inmunodeficiencias congénitas o adquiridas/Presencia de sedales, salvo que se puedan retirar antes de iniciar el tratamiento/ Presencia de proctitis grave (friabilidad llamativa, sangrado espontáneo, erosiones múltiples, úlceras profundas) a juzgar por la rectosigmoidoscopia/ Malignidades en remisión durante menos de un año antes de la inclusión en el estudio, con la excepción del carcinoma basocelular (CBC). |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of IMP-related adverse events during a follow-up period of 12 months post-treatment. |
Incidencia de acontecimientos adversos relacionados con el producto en investigación durante un periodo de seguimiento de 12 meses postadministración. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Weeks 0, 2, 12, 22, 24, 36, 46 and 48. |
Semanas 0, 2, 12, 22, 24, 36, 46 and 48. E.5.2 |
|
E.5.2 | Secondary end point(s) |
1. Ratio of complex perianal fistula closure in Crohn's disease patients, after 24 and 48 weeks of follow-up. 2. Number of draining fistulas after two consecutive visits at weeks 22, 24 and 46, 48. 3. Percentage of subjects with healed fistula (measured by MRI) after 12 and 24 weeks of follow-up. |
1. Ratio de cierre de fístulas complejas en enfermedad de Crohn perianal después de 24 y 48 semanas de seguimiento. 2. Número de fístulas de drenaje durante dos visitas consecutivas en las semanas 22, 24 y 46, 48 de seguimiento. 3. Porcentaje de sujetos con cicatrización de la fístula (medida por RM) tras 12 y 24 semanas de seguimiento. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Weeks 0, 2, 12, 22, 24, 36, 46 and 48. |
Semanas 0, 2, 12, 22, 24, 36, 46 and 48. |
|
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 | No |
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.8.2.4 | Number of treatment arms in the trial | 1 |
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
|
LVLS |
Ultimo paciente, última visita |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |