Epidermolysis Bullosa, JunctionalPhase 3NCT00587223
Safety and Efficacy of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa (EB)
A Phase 3 study testing Apligraf and Standard dressing regimen in people with Epidermolysis Bullosa, Junctional and Epidermolysis Bullosa Dystrophica, run by Organogenesis at 5 sites in United States.
Trial summary
The purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with dystrophic or junctional epidermolysis bullosa. Apligraf will be evaluated for efficacy and safety compared to a conventional nonadherent dressing. Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica
- What is being tested?
- Apligraf and Standard dressing regimen
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 5 sitesUnited States
- Study dates
- Dec 2007to Dec 2008
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject is between 2 and 65 years of age.
- Subject with clinical and histological diagnosis of junctional or dystrophic EB with documented electron microscopy or immunofluorescence microscopy.
- Subject has at least two non-adjacent EB lesions, at least 4 cm apart. The two selected EB lesions must be relatively matched in terms of diagnosis, location, level of erosion and size. Lesions may be on the same limb.
- Subject with dystrophic or junctional EB lesions between 10-44 cm2 present for at least 6 weeks. For the purposes of this study, a lesion is defined as a wound resulting from a post blister erosion.
- Subject who is a female of child-bearing potential (females >10 years of age) must have a documented negative urine or serum pregnancy test. Sexually active females must be practicing a medically proven form of contraception during the course of the study period.
- Subject or legal guardian must have read, understood and signed an institutional review board (IRB) approved Informed Consent Form or Assent Form.
- Subject and/or legal guardian must be able and willing to follow study procedures and instructions.
Who cannot join
- Subject with lesions only on the soles, posterior thigh or gluteus maximus.
- Subject whose lesion has healed 20% or greater in area from post debridement (if applicable) Baseline Screen (Visit 1) to post debridement Day 0 (Visit 2) as determined by wound tracings.
- Subject with uncontrolled diabetes mellitus (glycosylated HbA1C > 10%), cancer (biopsy confirmed active malignancy), or positive HIV test.
- Subject is a child (<18 years of age) who is currently receiving or has received oral steroid therapy exceeding a total daily dose of 0.5mg/kg for more than two weeks, radiation or other immuno-suppressive therapy which would interfere with wound healing within the past four weeks.
- Subject is an adult (>18 years of age) who is currently receiving or has received chronic high dose steroid therapy exceeding a total daily dose of 20mg for more than two weeks, radiation or other immuno-suppressive therapy which would interfere with wound healing within the past four weeks.
- Subject who is currently on or has received topical steroidal therapy within 30 days before screening. Inhaled steroids are allowed.
Show all 17Show fewer
- Subject with the presence of acute infections in the areas intended for treatment.
- Subject with a history of squamous cell carcinoma.
- Known hypersensitivity to bovine collagen or to the components of the Apligraf agarose shipping medium.
- Subject who is lactating or pregnant (hCG positive as determined by lab testing).
- Subject is a child (<18 years of age) with liver (ALT/SGPT, ALP, AST/SGOT, bilirubin, total protein, LDH) and/or renal function (BUN and creatinine) tests > 2 x upper limit of normal (ULN). Albumin < 2.0 mg/dL.
- Subject is an adult (>18 years of age) with liver (ALT/SGPT, ALP, AST/SGOT, bilirubin, total protein, LDH) and/or renal function (BUN and creatinine) tests > 2 x upper limit of normal (ULN). Albumin < 2.0 mg/dL.
- Subject enrolled in any wound or investigational device study for any disease within the past four weeks.
- Subject who has received an investigational drug or biological treatment within three months.
- Subject previously treated with Apligraf, Dermagraft or any other cell therapy at the target sites.
- Subject with a history of alcohol or substance abuse within the previous year, which could interfere with study compliance such as inability to attend scheduled study visits or compliance with home dressing changes.
- Subject who, in the opinion of the investigator, for any reason other than those listed above, will not be able to complete the study per protocol.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Children's Hospital of Cincinnati
Cincinnati, Ohio, 45229
University of Texas
Houston, Texas, 77030-1341
University of Miami - Miller School of Medicine
Miami, Florida, 33125
Columbia University Medical Center
New York, New York, 10032
Stanford University School of Medicine
Stanford, California, 94305-5168
About this study
Official title: A Prospective, Multicenter, Within Subject Controlled Study to Evaluate the Effect of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa
The purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with dystrophic or junctional epidermolysis bullosa. Apligraf will be evaluated for efficacy and safety compared to a conventional nonadherent dressing. A matched-pair design will be used to evaluate Apligraf treatment versus conventional treatment in 68 study pairs.
- Being tested
- Apligraf, Standard dressing regimen
- Medical subject headings
- Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ApligrafDevice
Up to 3 applications: Day 0, Month 1, Month 2.
Standard dressing regimenOther
Dressing regimen will be comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Apligraf (a living bilayered cell therapy product) Receives: Device: Apligraf |
| 2 | Active comparator | Dressing regimen comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer. Receives: Other: Standard dressing regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Wounds First Achieving 100% Epithelialization of Tissue With the Absence of Drainage (i.e. Complete Wound Closure) Through Study Week 12
Measured: Through 12 weeks
Secondary outcomes (5)
Time Until Complete Closure
Measured: through 12 weeks
Rate of Complete Wound Closure Over Time
Measured: through 12 weeks
Recurrence of Epidermolysis Bullosa (EB) Lesions
Measured: through 12 months
Reduction of Intensity of Pain
Measured: through 12 weeks
Proportion of Wounds Experiencing an Adverse Event
Measured: through 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2008 (estimated)
- Study startDec 2007
- Primary completionDec 2008
- Study completionDec 2008
Why it stopped: Insufficient patient enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Epidermolysis Bullosa, JunctionalStudies 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
- Organogenesis
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Elizabeth Alvarez- Connelly, MD, Principal investigator, University of MiamiDamien Bates, MD, PhD, FRACS (Plast.), Study director, Organogenesis Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00587223
- Sponsor's protocol ID
- 06-EB-001-AG
- Status last verified
- Jun 2010
- First submitted
- 21 Dec 2007
- Last updated
- 29 Jun 2010
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Within Subject Controlled Study to Evaluate the Effect of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT00587223
- Acronym / sponsor ID
- 06-EB-001-AG
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Insufficient patient enrollment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- OrganogenesisIndustry (pharma, biotech, devices)
- Study start
- Dec 2007
- Primary completion
- Dec 2008 (actual)
- Study completion
- Dec 2008 (actual)
- First posted
- 7 Jan 2008 (estimated)
- Results
- Posted 22 Jun 2010
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 use of Apligraf for the treatment of nonhealing wounds in subjects with dystrophic or junctional epidermolysis bullosa. Apligraf will be evaluated for efficacy and safety compared to a conventional nonadherent dressing. A matched-pair design will be used to evaluate Apligraf treatment versus conventional treatment in 68 study pairs.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Device: ApligrafApligraf (a living bilayered cell therapy product) |
| 2 | Active comparator | Other: Standard dressing regimenDressing regimen comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer. |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Subject is between 2 and 65 years of age.
- Subject with clinical and histological diagnosis of junctional or dystrophic EB with documented electron microscopy or immunofluorescence microscopy.
- Subject has at least two non-adjacent EB lesions, at least 4 cm apart. The two selected EB lesions must be relatively matched in terms of diagnosis, location, level of erosion and size. Lesions may be on the same limb.
- Subject with dystrophic or junctional EB lesions between 10-44 cm2 present for at least 6 weeks. For the purposes of this study, a lesion is defined as a wound resulting from a post blister erosion.
- Subject who is a female of child-bearing potential (females >10 years of age) must have a documented negative urine or serum pregnancy test. Sexually active females must be practicing a medically proven form of contraception during the course of the study period.
Key exclusion criteria (17 listed)
- Subject with lesions only on the soles, posterior thigh or gluteus maximus.
- Subject whose lesion has healed 20% or greater in area from post debridement (if applicable) Baseline Screen (Visit 1) to post debridement Day 0 (Visit 2) as determined by wound tracings.
- Subject with uncontrolled diabetes mellitus (glycosylated HbA1C > 10%), cancer (biopsy confirmed active malignancy), or positive HIV test.
- Subject is a child (<18 years of age) who is currently receiving or has received oral steroid therapy exceeding a total daily dose of 0.5mg/kg for more than two weeks, radiation or other immuno-suppressive therapy which would interfere with wound healing within the past four weeks.
- Subject is an adult (>18 years of age) who is currently receiving or has received chronic high dose steroid therapy exceeding a total daily dose of 20mg for more than two weeks, radiation or other immuno-suppressive therapy which would interfere with wound healing within the past four weeks.
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 |
|---|---|---|
| ApligrafUp to 3 applications: Day 0, Month 1, Month 2. | Device | 1 |
| Standard dressing regimenDressing regimen will be comprised of a primary nonadherent dressing, nonstick gauze and standard dressing retainer | Other | 2 |
Primary outcome
Proportion of Wounds First Achieving 100% Epithelialization of Tissue With the Absence of Drainage (i.e. Complete Wound Closure) Through Study Week 12
Time frame: Through 12 weeks
5 secondary outcomes
Time Until Complete Closure
Time frame: through 12 weeks
Rate of Complete Wound Closure Over Time
Time frame: through 12 weeks
Recurrence of Epidermolysis Bullosa (EB) Lesions
Time frame: through 12 months
Reduction of Intensity of Pain
Time frame: through 12 weeks
Proportion of Wounds Experiencing an Adverse Event
Time frame: through 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2010.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Elizabeth Alvarez- Connelly, MD principal investigator
University of Miami
Damien Bates, MD, PhD, FRACS (Plast.) study director
Organogenesis Inc.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Apligraf and Standard dressing regimen in people with Epidermolysis Bullosa, Junctional and Epidermolysis Bullosa Dystrophica, run by Organogenesis at 5 sites in United States.
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 7 inclusion and 17 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 29 Jun 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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?
Yes. Results are posted in the registry (first posted 22 Jun 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Proportion of Wounds First Achieving 100% Epithelialization of Tissue With the Absence of Drainage (i.e. Complete Wound Closure) Through Study Week 12 (time frame: Through 12 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 29 Jun 2010.