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

Epidermolysis Bullosa Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01749306
• Other study ID #: EB01-ABH001
• Secondary ID: 2012-001815-21
• Status: Terminated
• Phase: Phase 3
• First received: December 11, 2012
• Last updated: November 20, 2013
• Start date: December 2012
• Est. completion date: November 2013

Study information

• Verified date: November 2013
• Source: Shire Regenerative Medicine, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 1
• Est. completion date: November 2013
• Est. primary completion date: November 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. 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

2. Male and female subjects.

3. Stable nutritional status.

4. Subjects with a confirmed diagnosis of generalized Epidermolysis Bullosa (EB)

5. Cutaneous wounds meeting the following criteria:

1. Anatomical location: arms, legs, thorax, or back above the waistline and below the neck.

2. Documented age (duration) of the wound(s).

3. 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.

6. Negative urine pregnancy test for women of child-bearing potential.

7. Female subjects of childbearing potential and male subjects of procreative capacity must agree to use an effective method of contraception.

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 within the previous 4 weeks.

9. Taking, or have participated in other clinical studies involving gene therapy, stem cell therapy, 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. Hypersensitivity to any of the therapeutic agents.

12. History of malignant skin disease.

Related Conditions & MeSH terms

• Epidermolysis Bullosa

Intervention

Biological:
• ABH001
ABH001 applications topically every 4 weeks (±1 week) with protocol-specified dressings until wound healed or up to 44 weeks

Other:
• Control wound treatment
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

Locations

Country	Name	City	State
Austria	Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität	Salzburg
Canada	Toronto Regional Wound Healing Clinic	Mississauga	Ontario
Canada	University of Montreal	Montreal	Quebec
France	Hôpital Necker-Enfants Malades	Paris	Ile-De France
Germany	University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)	Freiburg	Baden-Wuerttemberg
Poland	Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna	Warszawa	Mazowieckie
Portugal	Hospital CUF Descobertas	Lisboa
Spain	Hospital Universitario La Paz	Madrid
United States	Denver Children's Hospital	Aurora	Colorado
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Virginia Clinical Research, Inc	Norfolk	Virginia
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	Lucile Packard Children's Hospital at Stanford University	Redwood City	California
United States	Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Shire Regenerative Medicine, Inc.

Countries where clinical trial is conducted

United States,  Austria,  Canada,  France,  Germany,  Poland,  Portugal,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Reduction in wound surface area		24 weeks
Secondary	Change in wound pain and wound itch		24 weeks
Secondary	Patient global impression of change (PGIC)		24 weeks
Secondary	Clinician global impression of change (CGIC)		24 weeks
Secondary	Proportion of subjects achieving reduction in wound surface area		24 weeks
Secondary	Time to reduction of wound surface area and duration of reduction		24 weeks
Secondary	Durability of wound healing		24 weeks
Secondary	Incidence, relatedness and severity of adverse events		Up to 48 Weeks