A study of the safety and efficacy of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing.

Conditions: Epidermolysis bullosa
MedDRA version: 16.0Level: PTClassification code 10014989Term: Epidermolysis bullosaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Registration Number: EUCTR2012-001815-21-AT

Lead Sponsor: Shire Regenerative Medicine, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 26

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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 21
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of ABH001 in initiating healing of selected cutaneous, stalled wounds in epidermolysis bullosa patients.;Secondary Objective: To evaluate change in wound surface area over time.<br>To evaluate durability of wound healing.<br>To evaluate parameters related to subject and clinician reported outcomes for the ABH001-treated wound relative to the Control-treated wound.<br>To document the safety of ABH001 applications in this subject population. <br>To evaluate the time to wound size reduction. ;Primary end point(s): The primary endpoint is the reduction in wound surface area in ABH001-treated versus control-treated wounds. ;Timepoint(s) of evaluation of this end point: 24 weeks

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Change in wound surface area over time.<br>Durability of wound healing<br>Change from baseline in wound pain. <br>Clinician global impression of change (CGIC).<br>Patient global impression of change (PGIC).<br>Change from baseline in wound itch. <br>Proportion of subjects achieving reduction in wound surface area.<br>Time to reduction of wound surface area. <br>Incidence and severity of adverse events;Timepoint(s) of evaluation of this end point: Week 24

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