Epidermolysis BullosaNCT01454687
Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes
A study testing Grafting of Autologous Cultured Revertant Keratinocytes in people with Epidermolysis Bullosa, run by Stanford University.
Trial summary
The term epidermolysis bullosa (EB) is used to describe a group of genetic skin diseases associated with skin weakness, blisters, and chronic wounds. "Revertant mosaicism" means that there are two genetically different populations of cells due to spontaneous mutations. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- Grafting of Autologous Cultured Revertant Keratinocytes
- Type of treatment
- Surgery or procedure
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2011to Apr 2014
- Phase
- Interventional
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of EB (simplex, junctional or dystrophic)
- Areas of revertant skin that has been confirmed by biopsy
- 18 years or older subject willing and able to give consent
- Confirmation of EB diagnosis by immunofluorescence (IF), electron microscopy (EM), and genetic testing confirming mutation
- At least 100 to 200 cm2 of open erosions on the trunk and/or extremities suitable for skin grafting
- Able to undergo adequate anesthesia to allow grafting procedures to take place
Who cannot join
- Medical instability limiting ability to travel to Stanford University Medical Center
- The presence of medical illness expected to complicate participation and/or compromise the safety of this technique
- Active infection with HIV, hepatitis B, or hepatitis C
- Active infection in the area that will undergo grafting
- Evidence of a systemic infection
- Current evidence or a history of skin cancer in the area that will undergo grafting
Show all 10Show fewer
- Active drug or alcohol addiction
- Hypersensitivity to vancomycin or amikacin
- Receipt of chemical or biological study product for the specific treatment ofEB in the past six months
- Positive pregnancy test or breast-feeding
About this study
The term epidermolysis bullosa (EB) is used to describe a group of genetic skin diseases associated with skin weakness, blisters, and chronic wounds. "Revertant mosaicism" means that there are two genetically different populations of cells due to spontaneous mutations. Some EB patients have normal, non-fragile skin patches which may be areas of revertant mosaicism. In the revertant areas, the proteins function normally, like non-EB skin. In this study, we plan to culture cells from the revertant areas and graft them on to the wounded areas.
- Conditions
- Epidermolysis Bullosa
- Being tested
- Grafting of Autologous Cultured Revertant Keratinocytes
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Grafting of Autologous Cultured Revertant KeratinocytesProcedure or surgery
Grafting of two to four epidermal sheets 40cm2 - 50cm2 onto wounded areas
What is being measured
The primary outcome is the main question the study is designed to answer.
Expression of the correct protein at the basement membrane zone
Measured: Week 52
Engraftment and healing of wounds with genetically revertant keratinocytes
Measured: Week 52
Secondary outcomes (4)
Engraftment and healing of wounds with genetically revertant keratinocytes
Measured: Week 8-12
Engraftment and healing of wounds with genetically revertant keratinocytes
Measured: Week 25
Expression of correct protein at the basement membrane zone
Measured: Week 8-12
Expression of the correct protein at the basement membrane zone
Measured: Week 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2011 (estimated)
- Study startOct 2011
- Primary completionApr 2014
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 Stanford UniversityThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alfred Lane, Stanford University
- Investigators
- Alfred Lane, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01454687
- Sponsor's protocol ID
- IRB # 22005
- Status last verified
- Apr 2014
- First submitted
- 13 Oct 2011
- Last updated
- 11 Apr 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes
- ClinicalTrials.gov ID
- NCT01454687
- Acronym / sponsor ID
- IRB # 22005
- Conditions
- Epidermolysis Bullosa
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Oct 2011
- Primary completion
- Apr 2014 (actual)
- First posted
- 19 Oct 2011 (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 term epidermolysis bullosa (EB) is used to describe a group of genetic skin diseases associated with skin weakness, blisters, and chronic wounds. "Revertant mosaicism" means that there are two genetically different populations of cells due to spontaneous mutations. Some EB patients have normal, non-fragile skin patches which may be areas of revertant mosaicism. In the revertant areas, the proteins function normally, like non-EB skin. In this study, we plan to culture cells from the revertant areas and graft them on to the wounded areas.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Clinical diagnosis of EB (simplex, junctional or dystrophic)
- Areas of revertant skin that has been confirmed by biopsy
- 18 years or older subject willing and able to give consent
- Confirmation of EB diagnosis by immunofluorescence (IF), electron microscopy (EM), and genetic testing confirming mutation
- At least 100 to 200 cm2 of open erosions on the trunk and/or extremities suitable for skin grafting
Key exclusion criteria (10 listed)
- Medical instability limiting ability to travel to Stanford University Medical Center
- The presence of medical illness expected to complicate participation and/or compromise the safety of this technique
- Active infection with HIV, hepatitis B, or hepatitis C
- Active infection in the area that will undergo grafting
- Evidence of a systemic infection
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 |
|---|---|---|
| Grafting of Autologous Cultured Revertant KeratinocytesGrafting of two to four epidermal sheets 40cm2 - 50cm2 onto wounded areas | Procedure or surgery | – |
Primary outcome
Expression of the correct protein at the basement membrane zone
Time frame: Week 52
Engraftment and healing of wounds with genetically revertant keratinocytes
Time frame: Week 52
4 secondary outcomes
Engraftment and healing of wounds with genetically revertant keratinocytes
Time frame: Week 8-12
Engraftment and healing of wounds with genetically revertant keratinocytes
Time frame: Week 25
Expression of correct protein at the basement membrane zone
Time frame: Week 8-12
Expression of the correct protein at the basement membrane zone
Time frame: Week 25
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2014.
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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.
Alfred Lane, MD principal investigator
Stanford University
Questions about this trial
What is this clinical trial studying?
A study testing Grafting of Autologous Cultured Revertant Keratinocytes in people with Epidermolysis Bullosa, run by Stanford University.
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 6 inclusion and 10 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: withdrawn, as of the sponsor's last update on 11 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Expression of the correct protein at the basement membrane zone (time frame: Week 52). The study lists 2 primary outcomes in all. 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 11 Apr 2014.