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

• Register: EUCTR
• Last refreshed on: 21 October 2013
• Main ID: EUCTR2012-001815-21-ES
• Date of registration: 26/09/2012
• Prospective Registration: Yes
• Primary sponsor: Shire Regenerative Medicine, Inc.
• Public title: A study of the safety and efficacy of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing.
• Scientific title: A Multicenter, Prospective, Randomized, Open-label, Intra-patient Controlled Study of the Efficacy and Safety of ABH001 for the Treatment of Stalled Chronic Cutaneous Wounds Associated with Generalized Epidermolysis Bullosa
• Date of first enrolment: 18/01/2013
• Target sample size: 25
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-001815-21
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Assessment blinded If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Standardised wound care alone Number of treatment arms in the trial: 2

Countries of recruitment

Australia, Austria, Germany, Portugal, Spain, United States

Contacts

• Name: Clinical Trials Information
• Affiliation: Shire Regenerative Medicine, Inc.

• Name: Clinical Trials Information
• Affiliation: Shire Regenerative Medicine, Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
1. Ability of subject (or subject?s legal guardian) to understand the purpose and risks of the study and provide signed and dated informed consent/assent and authorization to use protected health information in accordance with national and local patient regulations.
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 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.
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: 20
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. Taking, or have participated in other clinical studies involving gene therapy, stem cell therapy, recombinant DNA/protein therapy. If received, other investigational drug treatments for EB that have not met the acceptable washout periods of: 1 month for topical treatments, 3 months for prior ABH001 treatment on the study wound, 6 months for small molecules, and 1 year or five half-lives for biologics.
9. History of malignant skin disease.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Epidermolysis bullosa
MedDRA version: 15.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Product Code: ABH001
Pharmaceutical Form: Living tissue equivalent
INN or Proposed INN: Human dermal fibroblasts cultured on a bioresorbable polyglactin mesh
Current Sponsor code: ABH001
Other descriptive name: Human fibroblast-derived dermal substitute, Dermagraft
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)

Main Objective: To evaluate the efficacy of ABH001 in initiating healing of selected cutaneous, stalled wounds in epidermolysis bullosa patients.

Primary end point(s): The primary endpoint is the reduction in wound surface area in ABH001-treated versus control-treated wounds

Secondary Objective: 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, and the duration of, wound size reduction.

Timepoint(s) of evaluation of this end point: 24 weeks

Secondary Outcome(s)

Secondary end point(s): Change from baseline in wound pain and itch.
Patient global impression of change (PGIC).
Clinician global impression of change (CGIC).
Proportion of subjects achieving reduction in wound surface area.
Time to reduction of wound surface area and duration of reduction.
Incidence and severity of adverse events

Timepoint(s) of evaluation of this end point: Week 24

Secondary ID(s)

EB01-ABH001

ABH_EB-001

Source(s) of Monetary Support

Shire Regenerative Medicine, Inc.