Dystrophic Epidermolysis BullosaPhase 2NCT05157958
Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET in Subjects With Dystrophic Epidermolysis Bullosa
A Phase 2 study testing ALLO-ASC-SHEET in people with Dystrophic Epidermolysis Bullosa, run by Anterogen Co., Ltd. at 1 site in United States.
Trial summary
After confirming eligibility, a single subject with four selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, three target lesions for ALLO-ASC-SHEET and the other target for Vehicle control, and which lesion to apply which IP treatment will be determined randomly at the time of enrollment… Read more about this study
- Who is it for?
- Ages 4 to 60 years
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- What is being tested?
- ALLO-ASC-SHEET
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 1 May 2023to 28 Apr 2026
- Phase
- Phase 2
- 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 4 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subject diagnosed as dystrophic epidermolysis bullosa confirmed by clinical criteria and one of the following:
- Immunostaining test: patients who have reduced or no type 7 collagen in staining degree of immunofluorescence. Other antigens (laminin-332, type 17 collagen, plectin, integrin α6β4, type 5 and type 14 keratin, etc.) are normal in immune-staining.
- COL7A1 mutational analysis: confirmation of COL7A1 genetic mutation.
- Subject with skin ulcer lesions of dystrophic epidermolysis bullosa meet the following criteria, on the screening start day (Visit 1) and treatment start day (enrollment day) (Visit 3):
- Subject has two skin ulcer lesions judged as comparable to compare the safety and efficacy by investigator during screening period and prior to the IP application (enrollment day).
- Two skin ulcer lesions meeting criteria stated in 2a) should be sized 5-20 cm2 (inclusive)
- Subject who has two comparable target skin ulcer lesions, and each lesions with a change of size equal to or less than ±50% at treatment day (Visit 3) compared to that of screening day (Visit 1)
- Subject who has no clinical evidence of infection related signs/symptoms, or visible necrosis in the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied).
Who cannot join
- Subject who requires antibiotics due to bacterial infection on skin of the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied).
- Female subjects: pregnant woman (indicated by serum hCG test result at screening), breast-feeding patient, all sexually active patient, with child bearing potential in case of female*, who is not willing to contracept** during the clinical trial.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
University of Miami Dermatology Clinical Trials Unit
Miami, Florida, 33125
About this study
Official title: Double Blind, Randomized, Phase II Clinical Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET Versus Vehicle Control in Dystrophic Epidermolysis Bullosa (DEB) Patients
After confirming eligibility, a single subject with four selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, three target lesions for ALLO-ASC-SHEET and the other target for Vehicle control, and which lesion to apply which IP treatment will be determined randomly at the time of enrollment using pre-designed block randomization scheme.
Read the detailed description
Primary efficacy endpoint will be assessed during and after 12th IP applications.
- Conditions
- Dystrophic Epidermolysis Bullosa
- Being tested
- ALLO-ASC-SHEET, Vehicle Control
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALLO-ASC-SHEETBiological
Weekly administration
Vehicle ControlOther
Weekly administration
| Study group | Role | What they receive |
|---|---|---|
| ALLO-ASC-SHEET | Experimental | Allogeneic mesenchymal stem cells
Dressing for Dystrophic Epidermolysis Bullosa wound Receives: Biological: ALLO-ASC-SHEET |
| Conventional Therapy | Active comparator | Hydrogel Sheet
Matching control Receives: Other: Vehicle Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
Incidence, severity, relationship of adverse event
Measured: First application to week 37
Secondary outcomes (1)
Proportion of complete wound closure
Proportion of subject with target skin ulcer area meeting the definition of wound closure during and at the completion of 12 weeks treatment
Measured: First application to week 17
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2021
- Study start1 May 2023
- Primary completion1 Oct 2025 (estimated)
- Study completion28 Apr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dystrophic 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Anterogen Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05157958
- Sponsor's protocol ID
- ALLO-ASC-DEB-201
- Status last verified
- Apr 2024
- First submitted
- 2 Dec 2021
- Last updated
- 22 Apr 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double Blind, Randomized, Phase II Clinical Study to Evaluate Safety and Efficacy of ALLO-ASC-SHEET Versus Vehicle Control in Dystrophic Epidermolysis Bullosa (DEB) Patients
- ClinicalTrials.gov ID
- NCT05157958
- Acronym / sponsor ID
- ALLO-ASC-DEB-201
- Conditions
- Dystrophic Epidermolysis Bullosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Anterogen Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 1 May 2023 (actual)
- Primary completion
- 1 Oct 2025 (estimated)
- Study completion
- 28 Apr 2026 (estimated)
- First posted
- 15 Dec 2021 (actual)
- 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.
After confirming eligibility, a single subject with four selected target lesions will receive both ALLO-ASC-SHEET and Vehicle control, three target lesions for ALLO-ASC-SHEET and the other target for Vehicle control, and which lesion to apply which IP treatment will be determined randomly at the time of enrollment using pre-designed block randomization scheme.
Detailed description
Primary efficacy endpoint will be assessed during and after 12th IP applications.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALLO-ASC-SHEET | Experimental | Biological: ALLO-ASC-SHEETAllogeneic mesenchymal stem cells Dressing for Dystrophic Epidermolysis Bullosa wound |
| Conventional Therapy | Active comparator | Other: Vehicle ControlHydrogel Sheet Matching control |
Who may qualify
- Ages
- 4 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Subject diagnosed as dystrophic epidermolysis bullosa confirmed by clinical criteria and one of the following:
- Subject with skin ulcer lesions of dystrophic epidermolysis bullosa meet the following criteria, on the screening start day (Visit 1) and treatment start day (enrollment day) (Visit 3):
- Subject who has two comparable target skin ulcer lesions, and each lesions with a change of size equal to or less than ±50% at treatment day (Visit 3) compared to that of screening day (Visit 1)
- Subject who has no clinical evidence of infection related signs/symptoms, or visible necrosis in the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied).
Key exclusion criteria (2 listed)
- Subject who requires antibiotics due to bacterial infection on skin of the target skin ulcer area (area including ulcer lesion and surrounding area where the IP is to be applied).
- Female subjects: pregnant woman (indicated by serum hCG test result at screening), breast-feeding patient, all sexually active patient, with child bearing potential in case of female*, who is not willing to contracept** during the clinical trial.
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 |
|---|---|---|
| ALLO-ASC-SHEETWeekly administration | Biological | ALLO-ASC-SHEET |
| Vehicle ControlWeekly administration | Other | Conventional Therapy |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: First application to week 37
Incidence, severity, relationship of adverse event
1 secondary outcome
Proportion of complete wound closure
Time frame: First application to week 17
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2024.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ALLO-ASC-SHEET in people with Dystrophic Epidermolysis Bullosa, run by Anterogen Co., Ltd. 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 8 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 22 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fernanda Bellodi Schmidt (82-2-2014-0391, antstudy@anterogen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: First application to week 37). 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 22 Apr 2024.