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.
Sponsor:
Information provided by (Responsible Party):
Shire Regenerative Medicine, Inc.
ClinicalTrials.gov Identifier:
NCT01749306
First received: December 11, 2012
Last updated: November 20, 2013
Last verified: November 2013
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in wound pain and wound itch [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Patient global impression of change (PGIC) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Clinician global impression of change (CGIC) [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Proportion of subjects achieving reduction in wound surface area [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Time to reduction of wound surface area and duration of reduction [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Durability of wound healing [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
  • Incidence, relatedness and severity of adverse events [ Time Frame: Up to 48 Weeks ] [ Designated as safety issue: Yes ]

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
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
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

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01749306

Locations
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
Austria
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
France
Hôpital Necker-Enfants Malades
Paris, Ile-De France, France
Germany
University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)
Freiburg, Baden-Wuerttemberg, Germany
Poland
Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna
Warszawa, Mazowieckie, Poland
Portugal
Hospital CUF Descobertas
Lisboa, Portugal
Spain
Hospital Universitario La Paz
Madrid, Spain
Sponsors and Collaborators
Shire Regenerative Medicine, Inc.
Investigators
Principal Investigator: Alan Arbuckle, MD, FAAD, FAAP H&E Enterprises
  More Information

No publications provided

Responsible Party: Shire Regenerative Medicine, Inc.
ClinicalTrials.gov Identifier: NCT01749306     History of Changes
Other Study ID Numbers: EB01-ABH001, 2012-001815-21
Study First Received: December 11, 2012
Last Updated: November 20, 2013
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on August 26, 2014