A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to evaluate the efficacy and safety of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing. It is hypothesized that ABH001 may initiate and continue wound healing in patients with epidermolysis bullosa.

Official Title
-----------------
A Multicenter, Prospective, Randomized, Open-label, Intra-subject Controlled Study of the Efficacy and Safety of ABH001 for the Treatment of Stalled Chronic Cutaneous Wounds Associated With Generalized Epidermolysis Bullosa

Conditions
-----------------
Epidermolysis Bullosa

Intervention / Treatment
-----------------
* Biological: ABH001
* Other: Control wound treatment


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: The 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 Male and female subjects. Stable nutritional status. Subjects with a confirmed diagnosis of generalized Epidermolysis Bullosa (EB) Cutaneous wounds meeting the following criteria: Anatomical location: arms, legs, thorax, or back above the waistline and below the neck. Documented age (duration) of the wound(s). 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. Negative urine pregnancy test for women of child-bearing potential. Female subjects of childbearing potential and male subjects of procreative capacity must agree to use an effective method of contraception. Exclusion Criteria: Pregnant or nursing women. Diagnosis of non-genetic generalized EB. Localized, active clinical infection of study wounds. 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. Known allergy to bovine products. Known allergy to silver products. Systemic infection at the time of enrolment in the study. Currently receiving or have received oral steroid therapy within the previous 4 weeks. Taking, or have participated in other clinical studies involving gene therapy, stem cell therapy, recombinant DNA/protein therapy. Received ABH001, or other biologic or cell therapy for the treatment of EB in the study wound sites within the previous 3 months. Hypersensitivity to any of the therapeutic agents. History of malignant skin disease.
Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: ABH001<br>ABH001 application plus wound care dressings. | Biological: ABH001<br>* ABH001 applications topically every 4 weeks (±1 week) with protocol-specified dressings until wound healed or up to 44 weeks<br>* Other names: Dermagraft, Allogenic Neonatal Dermal Fibroblasts Seeded on poly(glycolide-co-L-lactide) (PGLLA) Scaffold;|
| Other: Control<br>Control wound treatment | Other: Control wound treatment<br>* Control wound care with protocol-specified dressings every 4 weeks (±1 week) up to 20 weeks with optional cross-over to ABH001 for additional 20 weeks<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Reduction in wound surface area |  | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in wound pain and wound itch |  | 24 weeks | 
| Patient global impression of change (PGIC) |  | 24 weeks | 
| Clinician global impression of change (CGIC) |  | 24 weeks | 
| Proportion of subjects achieving reduction in wound surface area |  | 24 weeks | 
| Time to reduction of wound surface area and duration of reduction |  | 24 weeks | 
| Durability of wound healing |  | 24 weeks | 
| Incidence, relatedness and severity of adverse events |  | Up to 48 Weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft