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

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

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.

Study Overview

• Status: Terminated
• Conditions: Epidermolysis Bullosa
• Intervention / Treatment: Biological: ABH001; Other: Control wound treatment

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 3

Contacts and Locations

Study Locations

• Austria
  • Salzburg, Austria
    • Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
• Canada
  • Ontario
    • Mississauga, Ontario, Canada
      • Toronto Regional Wound Healing Clinic
  • Quebec
    • Montreal, Quebec, Canada
      • University of Montreal
• France
  • Ile-De France
    • Paris, Ile-De France, France
      • Hôpital Necker-Enfants Malades
• Germany
  • Baden-Wuerttemberg
    • Freiburg, Baden-Wuerttemberg, Germany
      • University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)
• Poland
  • Mazowieckie
    • Warszawa, Mazowieckie, Poland
      • Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna
• Portugal
  • Lisboa, Portugal
    • Hospital Cuf Descobertas
• Spain
  • Madrid, Spain
    • Hospital Universitario La Paz
• United States
  • Arizona
    • Phoenix, Arizona, United States
      • Phoenix Children's Hospital
  • California
    • Redwood City, California, United States
      • Lucile Packard Children's Hospital at Stanford University
    • San Diego, California, United States, 92123
      • Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Denver Children's Hospital
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Virginia
    • Norfolk, Virginia, United States, 23507
      • Virginia Clinical Research, Inc

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Reduction in wound surface area	24 weeks

Secondary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Shire Regenerative Medicine, Inc.
• Investigators: Principal Investigator: Alan Arbuckle, MD, FAAD, FAAP, H&E Enterprises

Study record dates

Study Major Dates

• Study Start: December 1, 2012
• Primary Completion (Actual): November 1, 2013
• Study Completion (Anticipated): November 1, 2013

Study Registration Dates

• First Submitted: December 11, 2012
• First Submitted That Met QC Criteria: December 12, 2012
• First Posted (Estimate): December 13, 2012

Study Record Updates

• Last Update Posted (Estimate): November 21, 2013
• Last Update Submitted That Met QC Criteria: November 20, 2013
• Last Verified: November 1, 2013

More Information

Terms related to this study

• Keywords: epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft
• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa
• Other Study ID Numbers: EB01-ABH001; 2012-001815-21 (EudraCT Number)