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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01749306
Recruitment Status : Terminated
First Posted : December 13, 2012
Last Update Posted : November 21, 2013
Information provided by (Responsible Party):
Shire Regenerative Medicine, Inc.

December 11, 2012
December 13, 2012
November 21, 2013
December 2012
November 2013   (Final data collection date for primary outcome measure)
Reduction in wound surface area [ Time Frame: 24 weeks ]
Same as current
Complete list of historical versions of study NCT01749306 on Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
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.

Not Provided
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Epidermolysis Bullosa
  • 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
  • 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
  • ABH001
    ABH001 application plus wound care dressings.
    Intervention: Biological: ABH001
  • Control
    Control wound treatment
    Intervention: Other: Control wound treatment
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
November 2013
November 2013   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
Child, Adult, Older Adult
Contact information is only displayed when the study is recruiting subjects
Austria,   Canada,   France,   Germany,   Poland,   Portugal,   Spain,   United States
2012-001815-21 ( EudraCT Number )
Not Provided
Not Provided
Not Provided
Shire Regenerative Medicine, Inc.
Shire Regenerative Medicine, Inc.
Not Provided
Principal Investigator: Alan Arbuckle, MD, FAAD, FAAP H&E Enterprises
Shire Regenerative Medicine, Inc.
November 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP