E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Epidermolysis bullosa |
Epidermólisis bullosa |
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E.1.1.1 | Medical condition in easily understood language |
Genetic skin blistering condition |
Enfermedad cutánea genética ampollosa |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10014989 |
E.1.2 | Term | Epidermolysis bullosa |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the efficacy of ABH001 in initiating healing of selected cutaneous, stalled wounds in epidermolysis bullosa patients. |
Evaluar la eficacia de ABH001 para iniciar la cicatrización de las heridas cutáneas tórpidas en pacientes con epidermólisis bullosa |
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E.2.2 | Secondary objectives of the trial |
To evaluate parameters related to subject and clinician reported outcomes for the ABH001-treated wound relative to the Control-treated wound. To document the safety of ABH001 applications in this subject population. To evaluate the time to, and the duration of, wound size reduction. |
Evaluar parámetros relacionados con los resultados percibidos por el sujeto y el clínico al comparar las heridas tratadas con ABH001 y las heridas tratadas con Control Documentar la seguridad de las aplicaciones de ABH001 en esta población de sujetos Evaluar el tiempo y la duración de la reducción del tamaño de la herida |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Ability of subject (or subject?s legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent/assent and authorization to use protected health information in accordance with national and local patient regulations. 2. Male and female subjects. 3. Stable nutritional status. 4. Subjects with a confirmed diagnosis of generalized Epidermolysis Bullosa and cutaneous wounds meeting the following criteria: a. Anatomical location: arms, legs, thorax, or back above the waistline and below the neck. b. Documented age (duration) of the wound(s). c. One or more wounds capable of potentially meeting the following wound selection criteria at the end of the observation period: i. Two clinically non-infected cutaneous wounds with no clinically meaningful change in wound size during the observation period. ii. Two matched wounds. 5. Negative urine pregnancy test for women of child-bearing potential. 6. Female subjects of childbearing potential and male subjects of procreative capacity must agree to use an effective method of contraception. |
1. Capacidad de comprender el propósito y los riesgos del estudio y de proporcionar un consentimiento/asentimiento firmado y fechado y una autorización para el uso de la información sanitaria protegida de acuerdo con la normativa nacional y local aplicable a los pacientes. 2. Sujetos masculinos y femeninos 3. Estado nutricional estable 4. Sujetos con un diagnóstico confirmado de epidermólisis bullosa generalizada y heridas cutáneas que cumplan los criterios siguientes: a. Ubicación anatómica: brazos, piernas, tórax, espalda por encima de la cintura y por debajo del cuello. b. Antigüedad documentada (duración) de la(s) herida(s) c. Una o más heridas con posibilidades de poder cumplir los siguientes criterios de selección de la herida, el fin del periodo de observación: i. Dos heridas cutáneas clínicamente no infectadas con un cambio del área superficial de la herida durante el periodo de observación ii. Dos heridas equivalentes 5. Prueba de embarazo realizada en orina negativa en el caso de mujeres en edad fértil 6.Los sujetos de sexo femenino en edad fértil y los sujetos de sexo masculino capaces de procrear deben aceptar el uso de un método anticonceptivo eficaz |
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E.4 | Principal exclusion criteria |
1. Pregnant or nursing women. 2. Diagnosis of non-genetic generalized EB. 3. Localized, active clinical infection of study wounds. 4. Diseases or conditions that could interfere with the assessment of safety and efficacy of the study treatment and compliance of the subject with study visits/procedures. 5. Known allergy to bovine products. 6. Known allergy to silver products. 7. Systemic infection at the time of enrolment in the study. 8. Taking, or have participated in other clinical studies involving gene therapy, stem cell therapy, recombinant DNA/protein therapy. If received, other investigational drug treatments for EB that have not met the acceptable washout periods of: 1 month for topical treatments, 3 months for prior ABH001 treatment on the study wound, 6 months for small molecules, and 1 year or five half-lives for biologics. 9. History of malignant skin disease. |
1. Mujeres embarazadas o lactantes 2. Diagnóstico de EB generalizada no-hereditaria 3. Infección clínica activa y localizada de las heridas del estudio 4. Enfermedades o afecciones que podrían,interferir con la evaluación de la seguridad y la eficacia del tratamiento de estudio y con el cumplimiento por parte del sujeto de las visitas o los procedimientos 5. Alergia conocida a productos bovinos 6. Alergia conocida a productos con plata 7. Infección sistémica en el momento de la inclusión en el estudio 8. Estar participando o haber participado en otros estudios clínicos relacionados con la terapia genética, la terapia con células madre, la terapia con ADN/proteínas recombinante(s). Si han recibido otros tratamientos farmacológicos en investigación para la EB, pero no han cumplido los periodos de reposo farmacológico aceptables de: 1 mes para tratamientos tópicos, 3 meses para un tratamiento anterior con ABH001 en la herida del estudio, 6 meses para moléculas pequeñas y 1 año o cinco vidas medias para biofármacos. 9. Historial de neoplasias malignas de la piel |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the reduction in wound surface area in ABH001-treated versus control-treated wounds |
El criterio de valoración principal es la reducción del área superficial de la herida tratada con ABH001 y la herida equivalente tratada con Control. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Change from baseline in wound pain and itch. Patient global impression of change (PGIC). Clinician global impression of change (CGIC). Proportion of subjects achieving reduction in wound surface area. Time to reduction of wound surface area and duration of reduction. Incidence and severity of adverse events |
Cambio en el dolor y el prurito de la herida desde el inicio. Impresión general del paciente de cambio (IGPC). Impresión general del clínico de cambio (IGCC). Proporción de sujetos que alcanzan reducción de la superficie de la herida. Tiempo y duración de la reducción de la superficie de la herida.. Incidencia y gravedad de efectos adversos. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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) | 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 | 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 |
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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 |
Standardised wound care alone |
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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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |