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Epidermolysis BullosaPhase 3NCT05725018

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

A Phase 3 study testing EB-101 Surgical application of RDEB wounds in people with Epidermolysis Bullosa and Recessive Dystrophic Epidermolysis Bullosa, run by Abeona Therapeutics, Inc at 2 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen [C7] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older. Read more about this study

Who is it for?
Ages 12 months and older
What is being tested?
EB-101 Surgical application of RDEB wounds
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
2 sitesUnited States
Study dates
2 Apr 2023to 30 Sep 2025
Phase
Phase 3
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 12 months and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Clinical diagnosis of DEB.
  • Age 12 months and older.
  • Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent.
  • (This inclusion criterion was deleted as of Amendment 1.)
  • Confirmation of DEB diagnosis (either dominant [DDEB] or recessive [RDEB] forms) by genetic testing.
  • Able to undergo adequate anesthesia during EB-101 treatment.
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  • All women of childbearing potential must have a negative urine pregnancy test and use a reliable birth control method throughout the duration of the study.
  • On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline).
  • Must have at least one wound site that meets all of the following criteria:
  • An area ≥20 cm2,
  • Present for ≥6 months, and
  • Stage 2 wound defined as an open skin wound with partial thickness loss of dermis that has not extended through the dermis into subcutaneous tissue.
  • For patients with RDEB, they must have had Vyjuvek exposure by the time of screening or positive anti-C7 antibodies at baseline.
  • Patients must be willing to discontinue Vyjuvek and Filsuvez use on EB-101 treated sites until completion of the Week 24 visit.

Who cannot join

  • Medical instability limiting ability to travel to the study site or undergo EB-101 treatment.
  • 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.
  • (This exclusion criterion was deleted as of Amendment 3.)
  • Evidence of systemic infection.
  • Current evidence or a history of SCC in the area that will undergo EB-101 application.
  • Active drug or alcohol addiction.
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  • Hypersensitivity to vancomycin or amikacin.
  • Receipt of chemical or biological investigational therapy for the specific treatment of DEB in the 3 months prior to EB-101 application.
  • Breast-feeding.
  • Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the PI.
  • Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in severe DEB patients, and these abnormalities will not exclude a patient.
  • Unwillingness or inability to provide 4 skin biopsies, or patient's keratinocytes cannot be manufactured for use in EB-101 application.
  • Any other circumstance where the PI believes that the patient may not be appropriate for participation in the study.

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

Other trials in United States

About this study

To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen [C7] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older.

Read the detailed description

A multicenter, open-label, single-arm Phase 3b safety study of one-time surgical application of up to 12 autologous, LZRSE-Col7A1 gene-corrected keratinocyte sheets with C7 expression (EB-101) for the treatment of large, chronic DEB wounds in each of approximately 10-12 patients. Patients enrolled will also contribute biopsies to support manufacturing requirements. All patients will be followed through 24 weeks post-treatment.

Patients will be evaluated at their Screening Visit (D -60 to D -25), a phone call approximately 14 days after screening, and at Baseline (Day -1) prior to treatment on Day 0. Patients will remain in the hospital, or other suitable inpatient medical facility that will allow appropriate immobilization of treated wounds, as determined by the Investigator, for observation for up to 7 days (±2 days) following treatment. They will be evaluated by phone on Day 14; by telehealth visits on Weeks 4, 8, and 18; and by clinic visits on Weeks 12 and 24.

Being tested
EB-101 Surgical application of RDEB wounds
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-101 Surgical application of RDEB woundsBiological

    Also called pz-cel

    EB-101 autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7

    Other Names:

    • LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]
Study groups in NCT05725018
Study groupRoleWhat they receive
EB-101 Surgical application of RDEB woundsExperimentalNew or Previously Treated RDEB Patients
Receives: Biological: EB-101 Surgical application of RDEB wounds

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety Endpoint (number of treatment-related and treatment-emergent adverse events (AEs) and serious adverse events (SAEs)

    The number of treatment-related and treatment-emergent adverse events (TEAEs), treatment-emergent wound adverse events (TEWAEs), and serious adverse events (SAEs), including systemic and wound-specific adverse events.

    Measured: 6 months

  2. Safety Endpoint (number of patients and wounds that have an infection or any related adverse event.)

    The number of patients and wounds that have an infection or any related adverse event (AE).

    Measured: 6 months

  3. Safety Endpoint (number of patients and wounds that result in hospitalization (serious adverse event )

    The number of patients and wounds that result in hospitalization (SAE).

    Measured: 6 months

  4. Safety Endpoint (Incidence of squamous cell carcinoma)

    The incidence of squamous cell carcinoma (SCC).

    Measured: 6 months

  5. Safety Endpoint RCR status

    Replication-competent retrovirus (RCR) status.

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Feb 2023
  2. Study start2 Apr 2023
  3. Primary completion30 Sep 2025 (estimated)
  4. Study completion30 Sep 2025 (estimated)

Resources

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
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05725018
Sponsor's protocol ID
EB-101-CL-302
Status last verified
Jun 2025
First submitted
23 Jan 2023
Last updated
27 Jun 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing EB-101 Surgical application of RDEB wounds 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 14 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: under way, not looking for participants, as of the sponsor's last update on 27 Jun 2025. 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?

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?

Safety Endpoint (number of treatment-related and treatment-emergent adverse events (AEs) and serious adverse events (SAEs) (time frame: 6 months). The study lists 5 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 NCT05725018.

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 27 Jun 2025.

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