Epidermolysis BullosaPhase 3NCT04227106
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
A Phase 3 study testing EB-101 in people with Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, run by Abeona Therapeutics, Inc at 2 sites in United States.
Trial summary
The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- What is being tested?
- EB-101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesUnited States
- Study dates
- 10 Jan 2020to 18 Oct 2022
- Phase
- Phase 3
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Clinical diagnosis of RDEB;
- Age 6 years or older, willing and able to give consent/assent;
- If under the age of 18, guardian(s) is/are willing and able to give consent;
- Positive expression of the non-collagenous region 1 of the type 7 collagen protein (NC1+) in the skin;
- Two confirmed RDEB C7 mutations with recessive inheritance patterns (or confirmation that parents don't have any evidence of dominant disease);
- At least 40 cm2 areas of chronically wounded area on the trunk and/or extremities suitable for EB-101 application (open erosions);
Show all 15Show fewer
- Able to undergo adequate anesthesia during EB-101 application;
- Must have at least two matched, eligible wound sites (one pair);
- Wound sites must:
- Have an area ≥20 cm2,
- Present for ≥6 months, and
- Stage 2 wound;
- Women of childbearing potential must use a reliable birth control method throughout the duration of the study and for 6 months post treatment;
- Negative pregnancy test;
- Must be on stable pain medication regimen at least 30 days prior to Screening
Who cannot join
- Medical instability limiting ability to travel to the study site;
- The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as active infection with human immunodeficiency virus (HIV), hepatitis B or hepatitis C;
- Evidence of immune response to C7 by indirect immunofluorescence (IIF);
- Evidence of systemic infection;
- Current evidence or a history of squamous cell carcinoma (SCC) in the area that will undergo EB-101 application;
- Active drug or alcohol addiction;
Show all 13Show fewer
- Hypersensitivity to vancomycin or amikacin;
- Receipt of chemical or biological study product for the specific treatment of RDEB in the past 3 months;
- Positive pregnancy test or breast-feeding;
- Clinically significant medical or laboratory abnormalities as determined by the Principal Investigator;
- Inability to properly follow protocol and protect keratinocyte sheet sites, as determined by the Principal Investigator;
- Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in RDEB patients. These abnormalities will not exclude a participant; and
- Inability to culture participant's keratinocytes.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Stanford University
Redwood City, California, 94063
University of Massachusetts Medical School
Worcester, Massachusetts, 01605
About this study
Official title: VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Read the detailed description
Recessive dystrophic epidermolysis bullosa (RDEB) is an ultra-rare, severe inherited blistering skin disease caused by the absence of a protein known as type 7 collagen (C7). There is no approved treatment for RDEB. Only supportive care is currently possible.
This open-label, controlled study will evaluate the efficacy and safety of EB-101 for the treatment of large, chronic, RDEB wounds. The study intervention consists of one-time surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites in up to approximately 10-15 participants. A single EB-101 sheet is able to provide healing to a wound area up to approximately 40cm2. Up to 6 (six) EB-101 sheets may be applied to each patient, depending on the area of existing wounds. The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6) as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6). Patient-reported outcomes and safety will also be collected throughout the study.
The primary analysis for efficacy will be assessed when all patients reach Week 24. Safety and efficacy assessments will be conducted at regular intervals and completed when last patient reaches Week 26 post-treatment.
Upon completion of the study period, patients will be monitored annually as per standard of care for up to 15 years.
- Being tested
- EB-101
- Keywords
- chronic wounds
- Medical subject headings
- Epidermolysis Bullosa, Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EB-101Biological
Also called LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]
autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7
| Study group | Role | What they receive |
|---|---|---|
| EB-101 | Experimental | One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds Receives: Biological: EB-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Wound Healing
Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds
Measured: 24 weeks post-treatment
Pain Reduction
Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds
Measured: 24 weeks post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2020
- Study start10 Jan 2020
- Primary completion18 Oct 2022
- Study completion18 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Abeona Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jean Tang, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04227106
- Sponsor's protocol ID
- EB-101-CL-301
- Status last verified
- Apr 2022
- First submitted
- 10 Jan 2020
- Last updated
- 5 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Tang JY, Marinkovich MP, Wiss K, McCarthy D, Truesdale A, Chiou AS, Eid E, McIntyre JK, Bailey I, Furukawa LK, Gorell ES, Harris N, Khosla RK, Peter Lorenz H, Lu Y, Nazaroff J, Grachev ID, Moore AJ. Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. Lancet. 2025 Jul 12;406(10499):163-173. doi: 10.1016/S0140-6736(25)00778-0. Epub 2025 Jun 23.
PubMed 40570869
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- ClinicalTrials.gov ID
- NCT04227106
- Acronym / sponsor ID
- EB-101-CL-301
- Keywords
- chronic wounds
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Abeona Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 10 Jan 2020 (actual)
- Primary completion
- 18 Oct 2022 (actual)
- Study completion
- 18 Oct 2022 (actual)
- First posted
- 13 Jan 2020 (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.
The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Detailed description
Recessive dystrophic epidermolysis bullosa (RDEB) is an ultra-rare, severe inherited blistering skin disease caused by the absence of a protein known as type 7 collagen (C7). There is no approved treatment for RDEB. Only supportive care is currently possible.
This open-label, controlled study will evaluate the efficacy and safety of EB-101 for the treatment of large, chronic, RDEB wounds. The study intervention consists of one-time surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites in up to approximately 10-15 participants. A single EB-101 sheet is able to provide healing to a wound area up to approximately 40cm2. Up to 6 (six) EB-101 sheets may be applied to each patient, depending on the area of existing wounds. The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6) as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6). Patient-reported outcomes and safety will also be collected throughout the study.
The primary analysis for efficacy will be assessed when all patients reach Week 24. Safety and efficacy assessments will be conducted at regular intervals and completed when last patient reaches Week 26 post-treatment.
Upon completion of the study period, patients will be monitored annually as per standard of care for up to 15 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| EB-101 | Experimental | Biological: EB-101One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Clinical diagnosis of RDEB;
- Age 6 years or older, willing and able to give consent/assent;
- If under the age of 18, guardian(s) is/are willing and able to give consent;
- Positive expression of the non-collagenous region 1 of the type 7 collagen protein (NC1+) in the skin;
- Two confirmed RDEB C7 mutations with recessive inheritance patterns (or confirmation that parents don't have any evidence of dominant disease);
Key exclusion criteria (13 listed)
- Medical instability limiting ability to travel to the study site;
- The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as active infection with human immunodeficiency virus (HIV), hepatitis B or hepatitis C;
- Evidence of immune response to C7 by indirect immunofluorescence (IIF);
- Evidence of systemic infection;
- Current evidence or a history of squamous cell carcinoma (SCC) in the area that will undergo EB-101 application;
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 |
|---|---|---|
| EB-101Also: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7 | Biological | EB-101 |
Primary outcome
Wound Healing
Time frame: 24 weeks post-treatment
Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds
Pain Reduction
Time frame: 24 weeks post-treatment
Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2022.
- Derived from this study: PubMed 40570869Tang JY, Marinkovich MP, Wiss K, McCarthy D, Truesdale A, Chiou AS, Eid E, McIntyre JK, Bailey I, Furukawa LK, Gorell ES, Harris N, Khosla RK, Peter Lorenz H, Lu Y, Nazaroff J, Grachev ID, Moore AJ. Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. Lancet. 2025 Jul 12;406(10499):163-173. doi: 10.1016/S0140-6736(25)00778-0. Epub 2025 Jun 23.
- Individual participant dataSharing planned: No
Related trials for Epidermolysis Bullosa
Not yet recruitingPhase 2
Filsuvez in Moderate-to-Severe Epidermolysis Bullosa Simplex
Epidermolysis Bullosa Simplex
RecruitingPhase 2
A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study
Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa
RecruitingPhase 3
Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa
Epidermolysis Bullosa (EB)
RecruitingPhase 2
A Study to Evaluate ENERGI-F703EB Cream in Epidermolysis Bullosa
Epidermolysis Bullosa (EB), Junctional Epidermolysis Bullosa
RecruitingObservational study
A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez
Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional
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.
Jean Tang, MD principal investigator
Stanford University
2 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 EB-101 in people with Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, run by Abeona Therapeutics, Inc at 2 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 15 inclusion and 13 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 5 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Wound Healing (time frame: 24 weeks post-treatment). 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?
On the ClinicalTrials.gov record for NCT04227106, 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 5 Dec 2022.