Back to resultsA Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
Primary Purpose
Epidermolysis Bullosa
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ABH001
Control wound treatment
Sponsored by
About this trial
This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Sites / Locations
- Phoenix Children's Hospital
- Lucile Packard Children's Hospital at Stanford University
- Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology
- Denver Children's Hospital
- Ann & Robert H. Lurie Children's Hospital of Chicago
- Virginia Clinical Research, Inc
- Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
- Toronto Regional Wound Healing Clinic
- University of Montreal
- Hôpital Necker-Enfants Malades
- University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)
- Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna
- Hospital CUF Descobertas
- Hospital Universitario La Paz
Arms of the Study
Arm 1
Arm 2
Arm Type
Other
Other
Arm Label
ABH001
Control
Arm Description
ABH001 application plus wound care dressings.
Control wound treatment
Outcomes
Primary Outcome Measures
Reduction in wound surface area
Secondary Outcome Measures
Change in wound pain and wound itch
Patient global impression of change (PGIC)
Clinician global impression of change (CGIC)
Proportion of subjects achieving reduction in wound surface area
Time to reduction of wound surface area and duration of reduction
Durability of wound healing
Incidence, relatedness and severity of adverse events
Full Information
NCT ID
NCT01749306
First Posted
December 11, 2012
Last Updated
November 20, 2013
Sponsor
Shire Regenerative Medicine, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01749306
Brief Title
A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2013
Overall Recruitment Status
Terminated
Study Start Date
December 2012 (undefined)
Primary Completion Date
November 2013 (Actual)
Study Completion Date
November 2013 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire Regenerative Medicine, Inc.
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epidermolysis Bullosa
Keywords
epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
1 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ABH001
Arm Type
Other
Arm Description
ABH001 application plus wound care dressings.
Arm Title
Control
Arm Type
Other
Arm Description
Control wound treatment
Intervention Type
Biological
Intervention Name(s)
ABH001
Other Intervention Name(s)
Dermagraft, Allogenic Neonatal Dermal Fibroblasts Seeded on poly(glycolide-co-L-lactide) (PGLLA) Scaffold
Intervention Description
ABH001 applications topically every 4 weeks (±1 week) with protocol-specified dressings until wound healed or up to 44 weeks
Intervention Type
Other
Intervention Name(s)
Control wound treatment
Intervention Description
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
Primary Outcome Measure Information:
Title
Reduction in wound surface area
Time Frame
24 weeks
Secondary Outcome Measure Information:
Title
Change in wound pain and wound itch
Time Frame
24 weeks
Title
Patient global impression of change (PGIC)
Time Frame
24 weeks
Title
Clinician global impression of change (CGIC)
Time Frame
24 weeks
Title
Proportion of subjects achieving reduction in wound surface area
Time Frame
24 weeks
Title
Time to reduction of wound surface area and duration of reduction
Time Frame
24 weeks
Title
Durability of wound healing
Time Frame
24 weeks
Title
Incidence, relatedness and severity of adverse events
Time Frame
Up to 48 Weeks
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alan Arbuckle, MD, FAAD, FAAP
Organizational Affiliation
H&E Enterprises
Official's Role
Principal Investigator
Facility Information:
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
Country
United States
Facility Name
Lucile Packard Children's Hospital at Stanford University
City
Redwood City
State/Province
California
Country
United States
Facility Name
Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Denver Children's Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Virginia Clinical Research, Inc
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
City
Salzburg
Country
Austria
Facility Name
Toronto Regional Wound Healing Clinic
City
Mississauga
State/Province
Ontario
Country
Canada
Facility Name
University of Montreal
City
Montreal
State/Province
Quebec
Country
Canada
Facility Name
Hôpital Necker-Enfants Malades
City
Paris
State/Province
Ile-De France
Country
France
Facility Name
University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)
City
Freiburg
State/Province
Baden-Wuerttemberg
Country
Germany
Facility Name
Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna
City
Warszawa
State/Province
Mazowieckie
Country
Poland
Facility Name
Hospital CUF Descobertas
City
Lisboa
Country
Portugal
Facility Name
Hospital Universitario La Paz
City
Madrid
Country
Spain
12. IPD Sharing Statement
Learn more about this trial
A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
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