Epidermolysis BullosaPhase 4NCT01619670
A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa
A Phase 4 study testing Apligraf in people with Epidermolysis Bullosa and Wound Healing, run by University Hospital, Basel, Switzerland at 1 site in Switzerland.
Trial summary
Epidermolysis Bullosa (EB) is a very rare disease, with a severe impact on the life of the patient and the caregiver. Epidermolysis Bullosa (EB) comprises a group of genetically determined skin fragility disorders characterized by blistering of the skin and mucosae following mild mechanical trauma. Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Epidermolysis Bullosa, Wound Healing
- What is being tested?
- Apligraf
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteSwitzerland
- Study dates
- Jun 2012to Jan 2015
- Phase
- Phase 4
- 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 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 confirmed diagnosis of EB.
- Subject has at least two lesions, if in a situation with two non-adjacent EB lesions, at least 4 cm apart.
- Subject with EB lesions at least 2 cm2 present for at least 3weeks. 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
- 1. 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.
- 2. Subject with uncontrolled diabetes mellitus (glycosylated HbA1C > 10%), cancer (biopsy confirmed active malignancy), or positive HIV test.
- 3. 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.
- 4. 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.
- 5. Subject who is currently on or has received topical steroidal therapy within 30 days before screening. Inhaled steroids are allowed.
- 6. Subject with the presence of acute infections in the areas intended for treatment.
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- 7. Known hypersensitivity to bovine collagen or to the components of the Apligraf agarose shipping medium.
- 8. Subject who is lactating or pregnant (hCG positive as determined by lab testing).
- 9. Subject enrolled in any wound or investigational device study for any disease within the past four weeks.
- 10. Subject who has received an investigational drug or biological treatment within three months.
- 11. 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.
- 12. 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
1 site in 1 country.
About this study
Official title: A Prospective Single Center Within Subject Controlled Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa
Epidermolysis Bullosa (EB) is a very rare disease, with a severe impact on the life of the patient and the caregiver. Epidermolysis Bullosa (EB) comprises a group of genetically determined skin fragility disorders characterized by blistering of the skin and mucosae following mild mechanical trauma. There is no specific proven treatment for any form of EB, and the mainstay of clinical management is based on protection and avoidance of provoking factors. Chronic nonhealing erosions and ulcers have been treated with conventional split-thickness skin grafts. Alternatively some patients may benefit from the use of autologous or allogeneic cultured keratinocyte grafts.
Read the detailed description
Apligraf is a living bilayered cell therapy product. Apligraf is constructed of Type I bovine collagen. The mechanism of action of Apligraf is still unknown. No clinical evidence of rejection of Apligraf was observed when placed on acute or chronic wounds. Apligraf has been suggested to act as a "smart" material for wound healing by interacting with the surrounding environment to promote healing. It provides components with multiple actions, interacts with wounds in biological and physical ways, and appears to adapt to the wound environment and probably produces numerous pro-healing cytokines. The purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with epidermolysis bullosa. Apligraf will be evaluated for efficacy and safety compared to a conventional nonadherent dressing. The study is a pivotal, single center, within subject controlled observational trial in which the rate of wound healing, recurrence of EB lesions, subject report of pain will be compared to wounds treated with Control treatment. Subjects between 2 and 65 years of age, inclusive, with epidermolysis bullosa lesions will be screened for this study. For each subject at least two designated treatment sites will be selected. In case of two comparable treatment sites the most right side will receive Apligraf and the most left side will receive control. Efficacy will be assessed by clinical observations, wound tracings and photographs.
- Conditions
- Epidermolysis Bullosa, Wound Healing
- Being tested
- Apligraf
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ApligrafDevice
non adhesive layer
| Study group | Role | What they receive |
|---|---|---|
| no intervention | No intervention | standard wound care |
| Apligraf | Active comparator | non adhesive layer with apligraf Receives: Device: Apligraf |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of wounds
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 study week 12
Secondary outcomes (1)
Time
Time until 100 % epithelialization of wound tissue with the absence of drainage (i.e.complete wound closure).
Measured: until 100% epithelialization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2012 (estimated)
- Study startJun 2012
- Primary completionJan 2015
- Study completionJan 2015
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.
- More trials run by University Hospital, Basel, SwitzerlandThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Basel, Switzerland
- Sponsor type
- University, hospital or other
- Collaborators
- Organogenesis
- Responsible party
- Sponsor
- Investigators
- Andreas Arnold, MD, Principal investigator, University Hospital Basel, Dep. Dermatology and Venereology, Basel/Switzerland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01619670
- Sponsor's protocol ID
- EKBB 235/11
- Status last verified
- Mar 2015
- First submitted
- 12 Jun 2012
- Last updated
- 10 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Single Center Within Subject Controlled Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT01619670
- Acronym / sponsor ID
- EKBB 235/11
- Conditions
- Epidermolysis Bullosa, Wound Healing
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 3 participants (actual)
- Lead sponsor
- University Hospital, Basel, SwitzerlandUniversity, hospital or other
- Collaborators
- Organogenesis
- Study start
- Jun 2012
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 14 Jun 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.
Epidermolysis Bullosa (EB) is a very rare disease, with a severe impact on the life of the patient and the caregiver. Epidermolysis Bullosa (EB) comprises a group of genetically determined skin fragility disorders characterized by blistering of the skin and mucosae following mild mechanical trauma. There is no specific proven treatment for any form of EB, and the mainstay of clinical management is based on protection and avoidance of provoking factors. Chronic nonhealing erosions and ulcers have been treated with conventional split-thickness skin grafts. Alternatively some patients may benefit from the use of autologous or allogeneic cultured keratinocyte grafts.
Detailed description
Apligraf is a living bilayered cell therapy product. Apligraf is constructed of Type I bovine collagen. The mechanism of action of Apligraf is still unknown. No clinical evidence of rejection of Apligraf was observed when placed on acute or chronic wounds. Apligraf has been suggested to act as a "smart" material for wound healing by interacting with the surrounding environment to promote healing. It provides components with multiple actions, interacts with wounds in biological and physical ways, and appears to adapt to the wound environment and probably produces numerous pro-healing cytokines. The purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with epidermolysis bullosa. Apligraf will be evaluated for efficacy and safety compared to a conventional nonadherent dressing. The study is a pivotal, single center, within subject controlled observational trial in which the rate of wound healing, recurrence of EB lesions, subject report of pain will be compared to wounds treated with Control treatment. Subjects between 2 and 65 years of age, inclusive, with epidermolysis bullosa lesions will be screened for this study. For each subject at least two designated treatment sites will be selected. In case of two comparable treatment sites the most right side will receive Apligraf and the most left side will receive control. Efficacy will be assessed by clinical observations, wound tracings and photographs.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| no intervention | No intervention | –standard wound care |
| Apligraf | Active comparator | Device: Apligrafnon adhesive layer with apligraf |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Subject is between 2 and 65 years of age.
- Subject with clinical confirmed diagnosis of EB.
- Subject has at least two lesions, if in a situation with two non-adjacent EB lesions, at least 4 cm apart.
- Subject with EB lesions at least 2 cm2 present for at least 3weeks. 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 (12 listed)
- 1. 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.
- 2. Subject with uncontrolled diabetes mellitus (glycosylated HbA1C > 10%), cancer (biopsy confirmed active malignancy), or positive HIV test.
- 3. 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.
- 4. 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.
- 5. Subject who is currently on or has received topical steroidal therapy within 30 days before screening. Inhaled steroids are allowed.
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 |
|---|---|---|
| Apligrafnon adhesive layer | Device | Apligraf |
Primary outcome
Proportion of wounds
Time frame: through study week 12
Proportion of wounds first achieving 100 % epithelialization of tissue with the absence of drainage (i.e. complete wound closure) through study week 12.
1 secondary outcome
Time
Time frame: until 100% epithelialization
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 10 Mar 2015.
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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.
Andreas Arnold, MD principal investigator
University Hospital Basel, Dep. Dermatology and Venereology, Basel/Switzerland
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Apligraf in people with Epidermolysis Bullosa and Wound Healing, run by University Hospital, Basel, Switzerland at 1 site in Switzerland.
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 8 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 10 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Proportion of wounds (time frame: through study week 12). 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 10 Mar 2015.