Epidermolysis BullosaPhase 3NCT01749306
A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
A Phase 3 study testing ABH001 in people with Epidermolysis Bullosa, run by Shire Regenerative Medicine, Inc. at 14 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- ABH001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 14 sitesin 8 countries
- Study dates
- Dec 2012to Nov 2013
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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:
- Two clinically non-infected cutaneous wounds with no clinically meaningful change in wound size during the observation period.
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
14 sites in 8 countries.
14 sites in 8 countries
United States6 sites
Denver Children's Hospital
Aurora, Colorado, 80045
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Virginia Clinical Research, Inc
Norfolk, Virginia, 23507
Phoenix Children's Hospital
Phoenix, Arizona
Lucile Packard Children's Hospital at Stanford University
Redwood City, California
Rady Children's Hospital - San Diego/UCSD, Pediatric and Adolescent Dermatology
San Diego, California, 92123
Canada2 sites
Toronto Regional Wound Healing Clinic
Mississauga, Ontario
University of Montreal
Montreal, Quebec
Austria1 site
Landeskrankenhaus Salzburg-Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
Salzburg, State of Salzburg
Germany1 site
University of Freiburg (Studiensekretariat Hautklinik Universitätsklinikum Freiburg - Hautklinik)
Freiburg im Breisgau, Baden-Wurttemberg
Poland1 site
Warszawki Uniwersytet Medyczny Katedra I Klinika Dermatologiczna
Warsaw, Masovian Voivodeship
About this study
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
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
- Being tested
- ABH001, Control wound treatment
- Keywords
- epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABH001Biological
Also called Dermagraft, Allogenic Neonatal Dermal Fibroblasts Seeded on poly(glycolide-co-L-lactide) (PGLLA) Scaffold
ABH001 applications topically every 4 weeks (±1 week) with protocol-specified dressings until wound healed or up to 44 weeks
Control wound treatmentOther
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
| Study group | Role | What they receive |
|---|---|---|
| ABH001 | Other | ABH001 application plus wound care dressings. Receives: Biological: ABH001 |
| Control | Other | Control wound treatment Receives: Other: Control wound treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in wound surface area
Measured: 24 weeks
Secondary outcomes (7)
Change in wound pain and wound itch
Measured: 24 weeks
Patient global impression of change (PGIC)
Measured: 24 weeks
Clinician global impression of change (CGIC)
Measured: 24 weeks
Proportion of subjects achieving reduction in wound surface area
Measured: 24 weeks
Time to reduction of wound surface area and duration of reduction
Measured: 24 weeks
Durability of wound healing
Measured: 24 weeks
Incidence, relatedness and severity of adverse events
Measured: Up to 48 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2012 (estimated)
- Study startDec 2012
- Primary completionNov 2013
- Study completionNov 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire Regenerative Medicine, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alan Arbuckle, MD, FAAD, FAAP, Principal investigator, H&E Enterprises
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01749306
- Sponsor's protocol ID
- EB01-ABH001
- Other IDs
- 2012-001815-21
- Status last verified
- Nov 2013
- First submitted
- 11 Dec 2012
- Last updated
- 21 Nov 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01749306
- Acronym / sponsor ID
- EB01-ABH001
- Conditions
- Epidermolysis Bullosa
- Keywords
- epidermolysis bullosa, chronic wound, wound healing, dermal repair, fibroblasts, Dermagraft
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Shire Regenerative Medicine, Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2012
- Primary completion
- Nov 2013 (actual)
- Study completion
- Nov 2013 (estimated)
- First posted
- 13 Dec 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ABH001 | Other | Biological: ABH001ABH001 application plus wound care dressings. |
| Control | Other | Other: Control wound treatmentControl wound treatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- 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:
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ABH001Also: Dermagraft, Allogenic Neonatal Dermal Fibroblasts Seeded on poly(glycolide-co-L-lactide) (PGLLA) ScaffoldABH001 applications topically every 4 weeks (±1 week) with protocol-specified dressings until wound healed or up to 44 weeks | Biological | ABH001 |
| Control wound treatmentControl 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 | Other | Control |
Primary outcome
Reduction in wound surface area
Time frame: 24 weeks
7 secondary outcomes
Change in wound pain and wound itch
Time frame: 24 weeks
Patient global impression of change (PGIC)
Time frame: 24 weeks
Clinician global impression of change (CGIC)
Time frame: 24 weeks
Proportion of subjects achieving reduction in wound surface area
Time frame: 24 weeks
Time to reduction of wound surface area and duration of reduction
Time frame: 24 weeks
Durability of wound healing
Time frame: 24 weeks
Incidence, relatedness and severity of adverse events
Time frame: Up to 48 Weeks
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2013.
Related trials for Epidermolysis Bullosa
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alan Arbuckle, MD, FAAD, FAAP principal investigator
H&E Enterprises
14 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ABH001 in people with Epidermolysis Bullosa, run by Shire Regenerative Medicine, Inc. at 14 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 21 Nov 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada, Austria, France, Germany and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reduction in wound surface area (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Nov 2013.