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

Phase 3

Terminated

• Conditions: Epidermolysis Bullosa
• Interventions: Other: Control wound treatment; Biological: ABH001

• Registration Number: NCT01749306
• Lead Sponsor: Shire Regenerative Medicine, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2012-12
• Study First Submitted: 2012-12-11
• Study First Submitted QC: 2012-12-12
• Study First Posted: 2012-12-13
• Status Verified: 2013-11
• Primary Completion: 2013-11
• Study Completion: 2013-11
• Last Update Submitted: 2013-11-20
• Last Update Posted: 2013-11-21

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 1

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Control	Control wound treatment	Control wound treatment
ABH001	ABH001	ABH001 application plus wound care dressings.

Primary Outcome Measures

Name	Time	Method
Reduction in wound surface area	24 weeks

Secondary Outcome Measures

Name	Time	Method
Change in wound pain and wound itch	24 weeks
Durability of wound healing	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
Incidence, relatedness and severity of adverse events	Up to 48 Weeks
Patient global impression of change (PGIC)	24 weeks

Trial Locations

Locations (14)

Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität; 🇦🇹 Salzburg, Austria

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology; 🇺🇸 San Diego, California, United States

Denver Children's Hospital; 🇺🇸 Aurora, Colorado, United States

Lucile Packard Children's Hospital at Stanford University; 🇺🇸 Redwood City, California, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

University of Montreal; 🇨🇦 Montreal, Quebec, Canada

Virginia Clinical Research, Inc; 🇺🇸 Norfolk, Virginia, United States

Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna; 🇵🇱 Warszawa, Mazowieckie, Poland

Toronto Regional Wound Healing Clinic; 🇨🇦 Mississauga, Ontario, Canada

University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik); 🇩🇪 Freiburg, Baden-Wuerttemberg, Germany

Hôpital Necker-Enfants Malades; 🇫🇷 Paris, Ile-De France, France

Hospital CUF Descobertas; 🇵🇹 Lisboa, Portugal

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain