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

This study has been terminated.
Information provided by (Responsible Party):
Shire Regenerative Medicine, Inc. Identifier:
First received: December 11, 2012
Last updated: November 20, 2013
Last verified: November 2013

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.

Condition Intervention Phase
Epidermolysis Bullosa
Biological: ABH001
Other: Control wound treatment
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Shire Regenerative Medicine, Inc.:

Primary Outcome Measures:
  • Reduction in wound surface area [ Time Frame: 24 weeks ]

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

Enrollment: 1
Study Start Date: December 2012
Estimated Study Completion Date: November 2013
Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: Dermagraft, Allogenic Neonatal Dermal Fibroblasts Seeded on poly(glycolide-co-L-lactide) (PGLLA) Scaffold
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


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
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Please refer to this study by its identifier: NCT01749306

United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States
United States, California
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, United States, 92123
United States, Colorado
Denver Children's Hospital
Aurora, Colorado, United States, 80045
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Virginia
Virginia Clinical Research, Inc
Norfolk, Virginia, United States, 23507
Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
Salzburg, Austria
Canada, Ontario
Toronto Regional Wound Healing Clinic
Mississauga, Ontario, Canada
Canada, Quebec
University of Montreal
Montreal, Quebec, Canada
Hôpital Necker-Enfants Malades
Paris, Ile-De France, France
University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)
Freiburg, Baden-Wuerttemberg, Germany
Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna
Warszawa, Mazowieckie, Poland
Hospital CUF Descobertas
Lisboa, Portugal
Hospital Universitario La Paz
Madrid, Spain
Sponsors and Collaborators
Shire Regenerative Medicine, Inc.
Principal Investigator: Alan Arbuckle, MD, FAAD, FAAP H&E Enterprises
  More Information

Responsible Party: Shire Regenerative Medicine, Inc. Identifier: NCT01749306     History of Changes
Other Study ID Numbers: EB01-ABH001
2012-001815-21 ( EudraCT Number )
Study First Received: December 11, 2012
Last Updated: November 20, 2013

Keywords provided by Shire Regenerative Medicine, Inc.:
epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft

Additional relevant MeSH terms:
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous processed this record on April 24, 2017