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 fragility disorder |
enfermedad genética de fragilidad de la piel |
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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 | 19.0 |
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 |
The primary objective is to evaluate the efficacy and safety of SD-101-6.0vs. SD- 101-0.0 (placebo) in patients with Simplex, Recessive Dystrophic, or Junctional non Herlitz Epidermolysis Bullosa. The primary endpoint is the complete closure of the patient's target wound. |
El objetivo principal es evaluar la eficacia y seguridad de SD-101-6.0 frente a SD-101-0.0 (placebo) en pacientes con epidermólisis ampollosa simple, distrófica recesiva y juntural tipo no-Herlitz. El criterio principal de valoración es el cierre completo de la herida específica del paciente en dos meses. |
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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. Informed Consent form signed by the patient or patient's legal representative; also, if the patient is under the age of majority but capable of providing assent, signed assent from the patient. 2. Patient (or caretaker) must be willing to comply with all protocol requirements. 3. Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB. 4. Patient must have 1 target wound (size 10 to 50 cm 2 ). 5. Patients 1 month and older. 6. Target wound must be present for 21 days or more. |
1. Consentimiento informado firmado por el paciente o por el representante legal del paciente; si el paciente es menor de 18 años de edad pero es capaz de dar su asentimiento, asentimiento firmado por el paciente. 2. El paciente (o su cuidador) debe estar dispuesto a cumplir todos los requisitos del protocolo. 3. Diagnóstico de EA simple, distrófica recesiva o juntural tipo no-Herlitz. 4. Los pacientes deben tener una herida específica (10 a 50 cm2 de tamaño). 5. Pacientes de 1 mes o más de edad. 6. La herida específica debe tener una duración de 21 días o más. |
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E.4 | Principal exclusion criteria |
1. Patients who do not meet the entry criteria outlined above. 2. Selected target wound cannot have clinical evidence of local infection. 3. Use of any investigational drug within the 30 days before enrollment. 4. Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment. 5. Use of systemic or topical steroidal therapy within the 30 days before enrollment. (Inhaled steroids and ophthalmic drops containing steroids are allowed) 6. Use of systemic antibiotics within the 7 days before enrollment. 7. Current or former malignancy. 8. Arterial or venous disorder resulting in ulcerated lesions. 9. Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed at screening and every 30 days until the final visit for female patients of childbearing potential) 10. Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception. |
1. Pacientes que no cumplan los criterios de inclusión especificados con anterioridad. 2. La herida específica seleccionada no debe tener signos clínicos de infección local. 3. Uso de cualquier fármaco en fase de investigación en los 30 días previos a la inclusión. 4. Uso de inmunoterapia o quimioterapia citotóxica en los 60 días previos a la inclusión. 5. Uso de tratamiento esteroideo tópico o sistémico en los 30 días previos a la inclusión. (Se permiten los esteroides inhalados o los colirios oftálmicos que contienen esteroides.) 6. Uso de antibióticos sistémicos en los 7 días previos a la inclusión. 7. Neoplasia maligna actual o anterior. 8. Trastorno arterial o venoso que provoca lesiones ulceradas. 9. Embarazo o lactancia durante el estudio. (En el caso de las pacientes en edad fértil, se realizará una prueba de embarazo en orina en la selección y cada 30 días hasta la visita final.) 10. Mujeres en edad fértil que no se abstengan de tener relaciones sexuales y no empleen un método de anticoncepción aceptable. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the complete closure of the patient's target wound. Complete target wound closure is defined as skin reepithelialization without drainage. |
El criterio principal de valoración es el cierre completo de la herida específica del paciente. El cierre completo de la herida específica se define como la reepitelización de la piel, sin drenaje |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The primary efficacy analysis will compare the proportion of patients achieving this endpoint within two months. |
El análisis principal de la eficacia comparará la proporción de pacientes que alcanza este criterio de valoración en dos meses. |
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E.5.2 | Secondary end point(s) |
The secondary endpoints include the estimation of body surface area (BSA) coverage of lesional skin, and assessment of itching, pain, and scarring. |
Los criterios secundarios de valoración incluyen el cálculo de cobertura de SC de piel afectada por lesiones y la evaluación del picor y el dolor. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Median Time to Complete Target Wound Closure Change in lesional skin based on BSA estimates at Month 3, compared to Baseline. Change in itching assessed at Day 7, compared to Baseline. Change in pain assessed at Day 7, compared to Baseline. |
Mediana del tiempo transcurrido hasta el cierre completo de la herida específica. Cambio en la piel afectada por lesiones en base a los cálculos de la SC en el mes 3, en comparación con el periodo basal. Cambio en el picor evaluado el día 7, en comparación con el periodo basal. Cambio en el dolor evaluado el día 7, en comparación con el periodo basal. |
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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 | No |
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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Yes |
E.8.2.3 | Other | No |
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 | 6 |
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 |
Austria |
France |
Italy |
Netherlands |
Poland |
United Kingdom |
United States |
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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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LVLS |
Última visita del último paciente |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |