E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Genetic skin fragility disorder |
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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 | 20.0 |
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 compare 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. |
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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 cm2). 5. Patients 1 month and older. 6. Target wound must be present for 21 days or more. |
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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. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Endpoints The primary efficacy endpoints for this study are • Time to complete target wound closure within 3 months • The proportion of patients experiencing complete closure of their target wound within 3 months |
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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 3 months. |
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E.5.2 | Secondary end point(s) |
Key Secondary Efficacy Endpoints The secondary measures of efficacy include: • Proportion of patients experiencing complete closure of their target wound within 2 months • Proportion of patients experiencing complete closure of their target wound within 1 month • Change in lesional skin based on BSAI at Month 3, compared to Baseline •Change in Total Body Wound Burden based on BSAI 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 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
The secondary measures of efficacy include: • Change in lesional skin based on BSAI 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. •Proportion of patients experiencing complete would closure at 2 and 1 months. •Total body wound burden at Month 3 |
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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
| 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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 |
Australia |
Austria |
France |
Germany |
Israel |
Italy |
Lithuania |
Netherlands |
Poland |
Romania |
Serbia |
Spain |
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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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 | 3 |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |