Epidermolysis BullosaNCT02670837
Study of Cellutome System for Treatment of Individual Lesions in EB Pts
A study testing Cellutome Epidermal Harvesting System in people with Epidermolysis Bullosa, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
Few but persistent wounds often remain even after successful hematopoietic cell transplantation for systemic genodermatosis epidermolysis bullosa (EB). The investigators propose local wound therapy using epidermal skin grafting from the same donor that provided the hematopoietic graft, or from the same EB individual… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- Cellutome Epidermal Harvesting System
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Aug 2016to 3 Apr 2024
- Phase
- Interventional
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient (Recipient)
- Diagnosis of Dystrophic Epidermolysis Bullosa (DEB) or Junctional Epidermolysis Bullosa (JEB) with at least one wound, visibly free from infection (or previously treated) and meets the eligibility for Arm A or Arm B based on the skin graft source:
- Cell harvest from previous hematopoietic cell transplantation (HCT) donor (Arm A) - not applicable if Arm B
- At least 6 months after hematopoietic cell transplantation with donor chimerism
- Peripheral blood donor chimerism should be measured within 21 days of grafting and be >/= 5% and stable. Stability of chimerism will be determined by the protocol team and based on 3 peripheral blood chimerism values at least 1 month apart.
- No history of pre-BMT autoimmune cytopenias
Show all 19Show fewer
- Off immune suppressive therapy
- Original transplant donor is available and willing to be the epidermis donor
- Self-donation (Arm B) - not applicable if Arm A
- Proven somatic reversion
- Site for skin grafting free of cellulitis and any other clinically evident abnormalities
- Meets donor eligibility
- Insurance pre-authorization for procedure, if applicable
- Voluntary written consent (patient or parent/guardian for minors with assent) prior to any research related procedures or treatment.
- Skin Graft Donor (either hematopoietic cell transplantation donor for the EB patient [Arm A] or EB patient herself/himself [Arm B])
- Age > 2 years (based on prior safety testing of the device)
- Healthy on physical examination in the opinion of the evaluating provider
- Negativity for Hepatitis B and C, HIV, and HTLV1/2 within 30 days of donation
- Voluntary written consent (donor or parent/guardian for minors with assent) prior to any research related procedures
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Masonic Cancer Center and Medical Center
Minneapolis, Minnesota, 55455
About this study
Official title: Study of Epidermal Grafting Using the CelluTome Epidermal Harvesting System for the Treatment of Individual Lesions in Persons With Epidermolysis Bullosa [MT2015-36]
Few but persistent wounds often remain even after successful hematopoietic cell transplantation for systemic genodermatosis epidermolysis bullosa (EB). The investigators propose local wound therapy using epidermal skin grafting from the same donor that provided the hematopoietic graft, or from the same EB individual with a mosaic (naturally gene corrected) skin. In both cases permissive immune system and skin chimerism is expected to enable long-term epidermal engraftment and wound healing. The investigators will use FDA approved vacuum device (CelluTome®, Regulation number 878.4820) that enables scar-free harvesting of epidermis and its transfer on a non-adherent silicone dressing (Adaptic) to the recipient as a wound dressing.
- Conditions
- Epidermolysis Bullosa
- Being tested
- Cellutome Epidermal Harvesting System
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cellutome Epidermal Harvesting SystemDevice
| Study group | Role | What they receive |
|---|---|---|
| Graft from HCT donor | Experimental | Cells are harvested from a donor using Cellutome, then transferred via Adaptic dressing to the recipient's wound with up to 3 donor harvest sites/treated wound sites on day 0. Receives: Device: Cellutome Epidermal Harvesting System |
| Self donor from intact skin patch | Experimental | Cells are harvested from the subject using Cellutome, then transferred via Adaptic dressing to that subject's wound with up to 3 harvest sites/treated wound sites on day 0. Receives: Device: Cellutome Epidermal Harvesting System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Grafts Successfully Treated
If the body surface area affected by the wound is at least 50%
lower at 12 weeks relative to baseline, the graft will be considered
successful.
Measured: 12 weeks after grafting
Secondary outcomes (5)
Participants With Lesion Free Skin
Participants without Squamous cell carcinoma (SCC) at graft site. Wound reassessments will be performed via photographs and follow-up visits.
Measured: 1 year after grafting
Longevity of Grafted Skin
Percentage of patients who have had a 6 week period of lesion free skin by the time they are 1 year post grafting. Wound reassessments will be performed via photographs and follow-up visits.
Measured: 1 year after grafting
Percentage Change of a Patient's IScorEB Assessment Score
Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.
Measured: Baseline and 6 weeks
Percentage Change of a Patient's IScoreEB Assessment Score
Measure percent of changes in quality of life (QOL) through pain, itching, and general QOL IScorEB questionnaire. Scores can range from 16 to 112. The QOLS scores are summed so that a higher score indicates higher quality of life.
Measured: Baseline and 12 weeks
Scar-free Healing of the Body Sites of the Donor
Percentage of donors with no evidence of non-healed skin. Wound reassessments will be performed via photographs and follow-up visits.
Measured: 1 year after grafting
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2016 (estimated)
- Study start4 Aug 2016
- Primary completion16 Mar 2023
- Study completion3 Apr 2024
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christen Ebens, MD, MPH, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02670837
- Sponsor's protocol ID
- 2015LS154
- Status last verified
- Apr 2024
- First submitted
- 8 Jan 2016
- Last updated
- 9 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Publications
Ebens CL, McGrath JA, Riedl JA, Keith AR, Lilja G, Rusch S, Keene DR, Tufa SF, Riddle MJ, Shanley R, Van Heest AE, Tolar J. Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds. Br J Dermatol. 2021 Jun;184(6):1161-1169. doi: 10.1111/bjd.19503. Epub 2020 Dec 14.
PubMed 32866988
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Epidermal Grafting Using the CelluTome Epidermal Harvesting System for the Treatment of Individual Lesions in Persons With Epidermolysis Bullosa [MT2015-36]
- ClinicalTrials.gov ID
- NCT02670837
- Acronym / sponsor ID
- 2015LS154
- Conditions
- Epidermolysis Bullosa
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 4 Aug 2016 (actual)
- Primary completion
- 16 Mar 2023 (actual)
- Study completion
- 3 Apr 2024 (actual)
- First posted
- 2 Feb 2016 (estimated)
- Results
- Posted 12 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Few but persistent wounds often remain even after successful hematopoietic cell transplantation for systemic genodermatosis epidermolysis bullosa (EB). The investigators propose local wound therapy using epidermal skin grafting from the same donor that provided the hematopoietic graft, or from the same EB individual with a mosaic (naturally gene corrected) skin. In both cases permissive immune system and skin chimerism is expected to enable long-term epidermal engraftment and wound healing. The investigators will use FDA approved vacuum device (CelluTome®, Regulation number 878.4820) that enables scar-free harvesting of epidermis and its transfer on a non-adherent silicone dressing (Adaptic) to the recipient as a wound dressing.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Graft from HCT donor | Experimental | Device: Cellutome Epidermal Harvesting SystemCells are harvested from a donor using Cellutome, then transferred via Adaptic dressing to the recipient's wound with up to 3 donor harvest sites/treated wound sites on day 0. |
| Self donor from intact skin patch | Experimental | Device: Cellutome Epidermal Harvesting SystemCells are harvested from the subject using Cellutome, then transferred via Adaptic dressing to that subject's wound with up to 3 harvest sites/treated wound sites on day 0. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (19 listed)
- Patient (Recipient)
- Diagnosis of Dystrophic Epidermolysis Bullosa (DEB) or Junctional Epidermolysis Bullosa (JEB) with at least one wound, visibly free from infection (or previously treated) and meets the eligibility for Arm A or Arm B based on the skin graft source:
- Cell harvest from previous hematopoietic cell transplantation (HCT) donor (Arm A) - not applicable if Arm B
- Self-donation (Arm B) - not applicable if Arm A
- Insurance pre-authorization for procedure, if applicable
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Cellutome Epidermal Harvesting System | Device | Graft from HCT donor, Self donor from intact skin patch |
Primary outcome
Percentage of Grafts Successfully Treated
Time frame: 12 weeks after grafting
If the body surface area affected by the wound is at least 50% lower at 12 weeks relative to baseline, the graft will be considered successful.
5 secondary outcomes
Participants With Lesion Free Skin
Time frame: 1 year after grafting
Longevity of Grafted Skin
Time frame: 1 year after grafting
Percentage Change of a Patient's IScorEB Assessment Score
Time frame: Baseline and 6 weeks
Percentage Change of a Patient's IScoreEB Assessment Score
Time frame: Baseline and 12 weeks
Scar-free Healing of the Body Sites of the Donor
Time frame: 1 year after grafting
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32866988Ebens CL, McGrath JA, Riedl JA, Keith AR, Lilja G, Rusch S, Keene DR, Tufa SF, Riddle MJ, Shanley R, Van Heest AE, Tolar J. Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds. Br J Dermatol. 2021 Jun;184(6):1161-1169. doi: 10.1111/bjd.19503. Epub 2020 Dec 14.
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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.
Christen Ebens, MD, MPH principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Cellutome Epidermal Harvesting System in people with Epidermolysis Bullosa, run by Masonic Cancer Center, University of Minnesota at 1 site 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 19 inclusion and 0 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: finished, as of the sponsor's last update on 9 Apr 2024. 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 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Mar 2024). 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?
Percentage of Grafts Successfully Treated (time frame: 12 weeks after grafting). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02670837, which also lists 1 linked publication.
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 9 Apr 2024.