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

NCT01749306 | Terminated Phase 3

• 2 other identifiers: EB01-ABH0012012-001815-21
• Study Type: interventional
• Target: 1
• Locations: 8 countries
• Sites: 14

Brief 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.

Conditions

Epidermolysis Bullosa

Keywords

epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft

Outcome Measures

Primary Outcomes (1)

• Reduction in wound surface area

  24 weeks

Secondary Outcomes (7)

• 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

Study Arms (2)

ABH001

OTHER

ABH001 application plus wound care dressings.

Biological: ABH001

Control

OTHER

Control wound treatment

Other: Control wound treatment

Interventions

ABH001 BIOLOGICAL

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

Also known as: Dermagraft, Allogenic Neonatal Dermal Fibroblasts Seeded on poly(glycolide-co-L-lactide) (PGLLA) Scaffold

ABH001

Control wound treatment OTHER

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

Control

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Male and female subjects.
• Stable nutritional status.
• Subjects with a confirmed diagnosis of generalized Epidermolysis Bullosa (EB)
• Cutaneous wounds meeting the following criteria:
• Anatomical location: arms, legs, thorax, or back above the waistline and below the neck.
• Documented age (duration) of the wound(s).
• 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.
• Negative urine pregnancy test for women of child-bearing potential.
• Female subjects of childbearing potential and male subjects of procreative capacity must agree to use an effective method of contraception.

You may not qualify if:

• Pregnant or nursing women.
• Diagnosis of non-genetic generalized EB.
• Localized, active clinical infection of study wounds.
• 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.
• Known allergy to bovine products.
• Known allergy to silver products.
• Systemic infection at the time of enrolment in the study.
• Currently receiving or have received oral steroid therapy within the previous 4 weeks.
• Taking, or have participated in other clinical studies involving gene therapy, stem cell therapy, recombinant DNA/protein therapy.
• Received ABH001, or other biologic or cell therapy for the treatment of EB in the study wound sites within the previous 3 months.
• Hypersensitivity to any of the therapeutic agents.
• History of malignant skin disease.

Sponsors & Collaborators

• Shire Regenerative Medicine, Inc.: lead

Study Sites (14)

Phoenix Children's Hospital

Phoenix, Arizona, United States

Location

Lucile Packard Children's Hospital at Stanford University

Redwood City, California, United States

Location

Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology

San Diego, California, 92123, United States

Location

Denver Children's Hospital

Aurora, Colorado, 80045, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Virginia Clinical Research, Inc

Norfolk, Virginia, 23507, United States

Location

Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität

Salzburg, Salzburg, Austria

Location

Toronto Regional Wound Healing Clinic

Mississauga, Ontario, Canada

Location

University of Montreal

Montreal, Quebec, Canada

Location

Hôpital Necker-Enfants Malades

Paris, Île-de-France Region, France

Location

University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)

Freiburg im Breisgau, Baden-Wurttemberg, Germany

Location

Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna

Warsaw, Masovian Voivodeship, Poland

Location

Hospital CUF Descobertas

Lisbon, Lisbon District, Portugal

Location

Hospital Universitario La Paz

Madrid, Madrid, Spain

Location

MeSH Terms

Conditions

Epidermolysis Bullosa

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Study Officials

• Alan Arbuckle, MD, FAAD, FAAP

  H&E Enterprises

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 11, 2012
• First Posted: December 13, 2012
• Study Start: December 1, 2012
• Primary Completion: November 1, 2013
• Study Completion: November 1, 2013
• Last Updated: November 21, 2013

Record last verified: 2013-11

Locations

ES (1); US (6); PT (1); AU (1); DE (1); CA (2); FR (1); PL (1)