RDEBNCT05708677
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
An observational study of RDEB, run by Abeona Therapeutics, Inc at 2 sites in United States.
Trial summary
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the 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
- RDEB
- What is being tested?
- EB-101
- How many people
- 22 participants
- Where
- 2 sitesUnited States
- Study dates
- 9 Feb 2021to Aug 2036
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
Study population: Patient with previous EB-101 treatment
Who may be able to join
- Willing and able to give consent/assent;
- If under the age of 18, guardian(s) is/are willing and able to give consent;
- Prior study treatment with EB-101.
Who cannot join
- Inability to properly follow protocol as determined by the Principal Investigator
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites
Stanford University
Redwood City, California, 94063
University of Massachusetts Medical School
Worcester, Massachusetts, 01605
About this study
Official title: A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Read the detailed description
This is an open-label, long-term, follow-up study in participants from prior interventional trials involving surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites. Up to 22 participants will be enrolled in this study, with follow-up through at least 5 years post treatment. Patient-reported outcomes and safety will be assessed throughout the study, and where applicable, compared to Baseline of the preceding interventional EB-101 trial. Upon completion, participants will transfer to an annual monitoring program for additional 10 years of phone visits.
- Conditions
- RDEB
- Being tested
- EB-101
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EB-101Biological
Also called pz-cel
This is non-interventional and follows patients treated with EB-101
| Study group | Role | What they receive |
|---|---|---|
| Long-Term Extension | This study will follow one group of participants who were enrolled in a previous EB-101 study. Receives: Biological: EB-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Wound Closure
Proportion of RDEB wounds with healing ≥50%, ≥75%, and 100% at all clinic visits up to Month 54 as determined by direct investigator assessment.
Measured: 5 years
Pain Severity Scale (Wong-Baker FACES)
Change in pain assessed by Wong-Baker FACES scale at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Measured: 5 years
Itch Severity Scale
Longitudinal change of Itch severity scores assessed at all clinic visits up to Week 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Measured: 5 years
Zarit Burden Interview Scale
Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 4, with the higher scores being the worse outcome.
Measured: 5 years
Quality of Life in Epidermolysis Bullosa
Quality of Life in Epidermolysis Bullosa (QOLEB) responses will be assessed at all clinic visits up to Month 54.
Measured: 5 years
Caregiver Global Impression of Change for Pain Scale
Caregiver Global Impression of Change for Pain (CrGI-Pain) scores assessed at all clinic visits up to Month 54. The minimum score value is 1 and the maximum score value is 7, with the higher scores being the worse outcome.
Measured: 5 years
Wound Infection and Adverse Events
The number of participants and wounds that have an infection or any related adverse event.
Measured: 5 years
The number of treatment-emergent adverse events.
The number of treatment-emergent adverse events.
Measured: 5 years
Serious Adverse Events
The number of participants and wounds that result in hospitalization (serious adverse event [SAE]).
Measured: 5 years
Incidence of squamous cell carcinoma.
Incidence of squamous cell carcinoma.
Measured: 5 years
Replication-competent retrovirus (RCR) status
Replication-competent retrovirus (RCR) status
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2023
- Study start9 Feb 2021
- Primary completionFeb 2036 (estimated)
- Study completionAug 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RDEBStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 22 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Abeona Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Angela Iheanacho, MS, Study director, Abeona Therapeutics, IncSarah Abdelwahab, MD, Study director, Abeona Therapeutics, Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05708677
- Sponsor's protocol ID
- EB-101-LT-001
- Status last verified
- Mar 2026
- First submitted
- 23 Jan 2023
- Last updated
- 17 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- ClinicalTrials.gov ID
- NCT05708677
- Acronym / sponsor ID
- EB-101-LT-001
- Conditions
- RDEB
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 22 participants (estimated)
- Lead sponsor
- Abeona Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 9 Feb 2021 (actual)
- Primary completion
- Feb 2036 (estimated)
- Study completion
- Aug 2036 (estimated)
- First posted
- 1 Feb 2023 (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.
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Detailed description
This is an open-label, long-term, follow-up study in participants from prior interventional trials involving surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites. Up to 22 participants will be enrolled in this study, with follow-up through at least 5 years post treatment. Patient-reported outcomes and safety will be assessed throughout the study, and where applicable, compared to Baseline of the preceding interventional EB-101 trial. Upon completion, participants will transfer to an annual monitoring program for additional 10 years of phone visits.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Long-Term Extension | – | Biological: EB-101This study will follow one group of participants who were enrolled in a previous EB-101 study. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patient with previous EB-101 treatment
Key inclusion criteria (3 listed)
- Willing and able to give consent/assent;
- If under the age of 18, guardian(s) is/are willing and able to give consent;
- Prior study treatment with EB-101.
Key exclusion criteria (1 listed)
- Inability to properly follow protocol as determined by the Principal Investigator
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: pz-celThis is non-interventional and follows patients treated with EB-101 | Biological | Long-Term Extension |
Primary outcome
Wound Closure
Time frame: 5 years
Proportion of RDEB wounds with healing ≥50%, ≥75%, and 100% at all clinic visits up to Month 54 as determined by direct investigator assessment.
Pain Severity Scale (Wong-Baker FACES)
Time frame: 5 years
Change in pain assessed by Wong-Baker FACES scale at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Itch Severity Scale
Time frame: 5 years
Longitudinal change of Itch severity scores assessed at all clinic visits up to Week 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.
Zarit Burden Interview Scale
Time frame: 5 years
Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 4, with the higher scores being the worse outcome.
Quality of Life in Epidermolysis Bullosa
Time frame: 5 years
Quality of Life in Epidermolysis Bullosa (QOLEB) responses will be assessed at all clinic visits up to Month 54.
Caregiver Global Impression of Change for Pain Scale
Time frame: 5 years
Caregiver Global Impression of Change for Pain (CrGI-Pain) scores assessed at all clinic visits up to Month 54. The minimum score value is 1 and the maximum score value is 7, with the higher scores being the worse outcome.
Wound Infection and Adverse Events
Time frame: 5 years
The number of participants and wounds that have an infection or any related adverse event.
The number of treatment-emergent adverse events.
Time frame: 5 years
The number of treatment-emergent adverse events.
Serious Adverse Events
Time frame: 5 years
The number of participants and wounds that result in hospitalization (serious adverse event [SAE]).
Incidence of squamous cell carcinoma.
Time frame: 5 years
Incidence of squamous cell carcinoma.
Replication-competent retrovirus (RCR) status
Time frame: 5 years
Replication-competent retrovirus (RCR) status
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2026.
- Individual participant dataSharing planned: Undecided
Related trials for RDEB
Not yet recruitingPhase 4
Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds
Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB)
RecruitingObservational study
A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
Recessive Dystrophic Epidermolysis Bullosa
Not yet recruiting
Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
Recessive Dystrophic Epidermolysis Bullosa, Dysphagia
Not yet recruitingPhase 1/2
Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
Epidermolysis Bullosa (EB), Epidermolysis Bullosa Acquisita
RecruitingPhase 1/2
Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
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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.
Angela Iheanacho, MS study director
Abeona Therapeutics, Inc
Sarah Abdelwahab, MD study director
Abeona Therapeutics, Inc
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of RDEB, 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 3 inclusion and 1 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: enrolling by invitation, as of the sponsor's last update on 17 Jul 2026. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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 Closure (time frame: 5 years). The study lists 11 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 17 Jul 2026.