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 blistering condition |
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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 | 16.0 |
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. |
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E.2.2 | Secondary objectives of the trial |
To evaluate change in wound surface area over time. To evaluate durability of wound healing. 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 wound size reduction. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subject or legal guardian must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent or Assent Form and must be able to and willing to follow study procedures and instructions. 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 wound selection criteria at the end of the observation period: i. Clinically non-infected cutaneous wounds with no clinically meaningful change in wound size during the observation period. ii. Matched wounds/wound areas. 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. |
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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. Currently receiving or have received oral steroid therapy exceeding a total daily dose of 0.5 mg/kg (hydrocortisone equivalent potency) for more than 2 weeks, radiation or other immune-suppressive therapy within the previous 4 weeks. 9. Participating or having participated in other investigational clinical studies, involving gene therapy, stem cell therapy and/or recombinant DNA/protein therapy. 10. Received ABH001, or other biologic or cell therapy for the treatment of EB in the study wound sites within the previous 3 months 11. Documented or suspected hypersensitivity to any of the therapeutic agents, including anaesthetic drugs. 12. History of malignant skin disease. |
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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. |
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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 in wound surface area over time. Durability of wound healing Change from baseline in wound pain. Clinician global impression of change (CGIC). Patient global impression of change (PGIC). Change from baseline in wound itch. Proportion of subjects achieving reduction in wound surface area. Time to reduction of wound surface area. Incidence and severity of adverse events |
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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 | 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 |
Canada |
France |
Germany |
Poland |
Portugal |
Spain |
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 | 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 |