E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cicatricial conjunctivitis associated with Lyell's syndrome, Stevens-Johnson syndrome and mucous membrane pemphigoid with ocular involvement. |
Conjuntivitis cicatriciales asociadas a síndrome de Lyell, síndrome Stevens- Johnson y Penfigoide de las membranas mucosas con afectación ocular. |
|
E.1.1.1 | Medical condition in easily understood language |
Conjunctivitis |
Conjuntivitis |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Eye Diseases [C11] |
MedDRA Classification |
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 |
To evaluate the safety and tolerability of the administration of allogenic mesenchymal cells derived from adipose tissue in diabetic patients with critical ischemia of the lower limbs. |
evaluar la seguridad y tolerabilidad de la administración de células mesenquimales troncales adultas alogénicas de tejido adiposo expandidas en pacientes diabéticos con isquemia crítica de miembros inferiores |
|
E.2.2 | Secondary objectives of the trial |
-To evaluate the preliminary efficacy of the treatment. - To evaluate the quality of life after the administration of the treatment. - To evaluate immunological changes |
- Evaluar la eficacia preliminar del tratamiento - Evaluar los cambios en la calidad de vida de los pacientes - Evaluar cambios inmunológicos |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Men and women over 18 years of age. 2. Diagnosis of ocular pemphigoid in Foster stages I-IIcIIIb (2) or diagnosis of recurrent chronic or episodic inflammation accompanied by cicatricial conjunctivitis of the mucous membranes with ocular involvement after the acute phase of Stevens-Johnson Syndrome or Lyell Syndrome . 3. In the case of women of childbearing age, who are willing to use an effective contraceptive method during the period of participation in the study 4. Consent to participate and signature of the informed consent |
1. Hombres y mujeres mayores de 18 años. 2. Diagnóstico de penfigoide ocular en estadíos I-IIcIIIb (2) de Foster o diagnóstico de inflamación crónica o episódica recurrente acompañada de conjuntivitis cicatricial de las membranas mucosas con afectación ocular tras la fase aguda de un Síndrome de Stevens-Johnson o Síndrome de Lyell. 3. En caso de mujeres en edad fértil, que estén dispuestas a utilizar un método anticonceptivo eficaz durante el periodo de participación en el estudio 4. Consentimiento para participar y firma del consentimiento informado |
|
E.4 | Principal exclusion criteria |
1. Signs of active infection on the ocular surface. 2. History of neoplasms in the last 5 years. except for epithelial basal or squamous cell carcinoma 3. Allergy to local anesthetics 4. Patients who have participated in another clinical trial with medication during the 90 days prior to signing the IC 5. Medical or psychiatric illness of any kind that, in the opinion of the investigator, may be a reason for exclusion from the study. 6. Congenital or acquired immunodeficiencies. 7. Major surgery or serious trauma of the subject in the semester prior to signing the IC. 8. Pregnant or lactating women. 9. Impossibility or refusal to carry out the follow-up required in the study by the patient |
1. Signos de infección activa en la superficie ocular. 2. Historial de neoplasias en los últimos 5 años. a excepción de carcinoma epitelial de células basales o escamosas 3. Alergia a anestésicos locales 4. Pacientes que hayan participado en otro ensayo clínico con medicamento durante los 90 días previos a la firma del CI 5. Enfermedad médica o psiquiátrica de cualquier tipo que, en opinión del investigador, pueda suponer un motivo de exclusión del estudio. 6. Inmunodeficiencias congénitas o adquiridas. 7. Cirugía mayor o traumatismo grave del sujeto en el semestre anterior a la firma del CI. 8. Mujeres embarazadas o lactantes. 9. Imposibilidad o negación a realizar el seguimiento requerido en el estudio por parte del paciente |
|
E.5 End points |
E.5.1 | Primary end point(s) |
According to the primary objective of the study, the following variables are defined: o Percentage and type of complications arising from treatment: o Complications during anesthesia. o Complications during cell implantation. o Peri and postoperative complications. |
- Porcentaje y tipo de complicaciones debidas al tratamiento. - Complicaciones durante la anestesia. - Complicaciones durante el implante celular. - Complicaciones peri y post operatorias |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
At 7 days (and 21 days in the 2-dose group), 4 weeks, 12w, 24w and 52 weeks |
A los 7 días (y 21 días en grupo 2 dosis), 4 semanas 12s, 24s y 52 semanas |
|
E.5.2 | Secondary end point(s) |
-Evaluate the preliminary efficacy of the treatment in terms of: o Improvement of signs (scarring conjunctivitis rating scale) o Symptom improvement (OSDI questionnaire) o VA improvement (EDTRS)
-Assess the changes in quality of life with respect to baseline using the specific NEI-VFQ questionnaire 25
-Determine the evolution of the conjunctival flora of patients after being treated by applying ASC to the ocular surface and evaluate the effect of the treatment (to see if there is any type of immunomodulation) |
Evaluar la eficacia preliminar del tratamiento en términos de: o Mejoría de los signos (escala de valoración de conjuntivitis cicatricial) o Mejoría de los síntomas (cuestionario OSDI) o Mejoría de la AV (EDTRS)
Evaluar los cambios en la calidad de vida respecto a la situación basal mediante el cuestionario específico NEI-VFQ 25
Determinar la evolución de la flora conjuntival de los pacientes tras ser tratados mediante la aplicación ASC en la superficie ocular y evaluar el efecto del tratamiento (para ver si hay algún tipo de inmunomodulación) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
At 7 days (and 21 days in the 2-dose group), 4 weeks, 12w, 24w and 52 weeks |
A los 7 días (y 21 días en grupo 2 dosis), 4 semanas 12s, 24s y 52 semanas |
|
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 | Yes |
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 | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
DOSE RANGING STUDY, WITH 2 CONSECUTIVE COHORTS, ONE SINGLE DOSE AND ONE 2 DOSES |
|
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 | Yes |
E.8.2.3.1 | Comparator description |
SAME MEDICINAL PRODUCT, ONE MORE DOSE |
|
E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 8 |
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
|
Last patient Last visit |
última visita del último paciente |
|
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 | 20 |
E.8.9.1 | In the Member State concerned days | |