Clinical trial NCT01749306: 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
Overall status: TERMINATED
Phase: PHASE3
Conditions: Epidermolysis Bullosa
Keywords: epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft
Lead sponsor: Shire Regenerative Medicine, Inc.
Sponsor class: INDUSTRY
First posted: 2012-12-13
Enrollment: 1
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Reduction in wound surface area
timeFrame: 24 weeks
Secondary outcomes:
measure: Change in wound pain and wound itch
timeFrame: 24 weeks
measure: Patient global impression of change (PGIC)
timeFrame: 24 weeks
measure: Clinician global impression of change (CGIC)
timeFrame: 24 weeks
measure: Proportion of subjects achieving reduction in wound surface area
timeFrame: 24 weeks
measure: Time to reduction of wound surface area and duration of reduction
timeFrame: 24 weeks
measure: Durability of wound healing
timeFrame: 24 weeks
measure: Incidence, relatedness and severity of adverse events
timeFrame: Up to 48 Weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Redwood City
country: United States
facility: Lucile Packard Children's Hospital at Stanford University
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology
state: California
city: Aurora
country: United States
facility: Denver Children's Hospital
state: Colorado
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Norfolk
country: United States
facility: Virginia Clinical Research, Inc
state: Virginia
city: Salzburg
country: Austria
facility: Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
city: Mississauga
country: Canada
facility: Toronto Regional Wound Healing Clinic
state: Ontario
city: Montreal
country: Canada
facility: University of Montreal
state: Quebec
city: Paris
country: France
facility: Hôpital Necker-Enfants Malades
state: Ile-De France
city: Freiburg
country: Germany
facility: University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)
state: Baden-Wuerttemberg
city: Warszawa
country: Poland
facility: Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna
state: Mazowieckie
city: Lisboa
country: Portugal
facility: Hospital CUF Descobertas
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
Study officials:
affiliation: H&E Enterprises
name: Alan Arbuckle, MD, FAAD, FAAP
role: PRINCIPAL_INVESTIGATOR
References: