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

NCT ID: NCT01749306

Last Updated: 2013-11-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-12-31

Study Completion Date

2013-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Epidermolysis Bullosa

Keywords

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ABH001

ABH001 application plus wound care dressings.

Group Type OTHER

ABH001

Intervention Type BIOLOGICAL

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

Control

Control wound treatment

Group Type OTHER

Control wound treatment

Intervention Type 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

Interventions

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ABH001

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

Intervention Type BIOLOGICAL

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

Intervention Type OTHER

Other Intervention Names

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

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shire Regenerative Medicine, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Alan Arbuckle, MD, FAAD, FAAP

Role: PRINCIPAL_INVESTIGATOR

H&E Enterprises

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

Lucile Packard Children's Hospital at Stanford University

Redwood City, California, United States

Site Status

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

San Diego, California, United States

Site Status

Denver Children's Hospital

Aurora, Colorado, United States

Site Status

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

Chicago, Illinois, United States

Site Status

Virginia Clinical Research, Inc

Norfolk, Virginia, United States

Site Status

Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität

Salzburg, Salzburg, Austria

Site Status

Toronto Regional Wound Healing Clinic

Mississauga, Ontario, Canada

Site Status

University of Montreal

Montreal, Quebec, Canada

Site Status

Hôpital Necker-Enfants Malades

Paris, Île-de-France Region, France

Site Status

University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)

Freiburg im Breisgau, Baden-Wurttemberg, Germany

Site Status

Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna

Warsaw, Masovian Voivodeship, Poland

Site Status

Hospital CUF Descobertas

Lisbon, Lisbon District, Portugal

Site Status

Hospital Universitario La Paz

Madrid, Madrid, Spain

Site Status

Countries

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United States Austria Canada France Germany Poland Portugal Spain

Other Identifiers

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2012-001815-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

EB01-ABH001

Identifier Type: -

Identifier Source: org_study_id