A Phase 3 interventional study of EB-101 Surgical application of RDEB wounds in Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa and RDEB, sponsored by Abeona Therapeutics, Inc. Active, not recruiting at 2 sites in United States. Open to participants aged 12 Months and older. Per ClinicalTrials.gov, last updated 2025-06-27.
Sponsored by Abeona Therapeutics, Inc · Phase 3, Interventional, and Treatment
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.
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.
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 21 are open to participants now.
This study's planned enrollment of 12 is above the median of 9 across 55 interventional studies indexed under Epidermolysis Bullosa Dystrophica.
Browse Epidermolysis Bullosa Dystrophica studies →Abeona Therapeutics, Inc is the lead sponsor of 11 studies on the registry; 3 are open to participants now.
Compared within Epidermolysis Bullosa Dystrophica, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Epidermolysis Bullosa. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Must have at least one wound site that meets all of the following criteria:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
New or Previously Treated RDEB Patients
Biological: EB-101 Surgical application of RDEB wounds
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 \[LEAES\]
Also known as: pz-cel
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.
Time frame: 6 months
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).
Time frame: 6 months
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).
Time frame: 6 months
Safety Endpoint (Incidence of squamous cell carcinoma)
The incidence of squamous cell carcinoma (SCC).
Time frame: 6 months
Safety Endpoint RCR status
Replication-competent retrovirus (RCR) status.
Time frame: 6 months
Proportion of RDEB wounds with ≥50%, ≥75%, and complete
Proportion of RDEB wounds with ≥50%, ≥75%, and complete (i.e., re epithelialization with no drainage or erosion and presence of only minor crusting) healing at Weeks 12 and 24, as determined by direct Investigator assessment.
Time frame: 6 months
Extent of pain reduction assessed by the Wong-Baker FACES scale
Extent of pain reduction assessed by the Wong-Baker FACES scale (for patients ages ≥6 years old) at Weeks 8, 12, and 24.
Time frame: 6 months
Longitudinal change in scores of Worst Itch Numeric Rating Scale
Longitudinal change in scores of Worst Itch Numeric Rating Scale (WI-NRS; for patients ages ≥6 years old) scores assessed at Weeks 8, 12, and 24
Time frame: 6 months
Longitudinal change of Zarit Burden Interview Short Form
Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at Weeks 12 and 24 (for all ages).
Time frame: 6 months
Change in Quality of Life in Epidermolysis Bullosa
Change in Quality of Life in Epidermolysis Bullosa (QOLEB) scores assessed at Week 24 compared with pretreatment Baseline scores (completed by patients ≥11 years old or with guidance for patients 8 to 10 years old). If patient is \<8 years old, QOLEB should be completed by a legal guardian.
Time frame: 6 months
Change in Caregiver Global Impression of Pain
Change in Caregiver Global Impression of Pain (CrGI-Pain) scores assessed at Weeks 8, 12, and 24 compared with pretreatment Baseline (for all ages).
Time frame: 6 months
Comparison of how non-collagenous region 1 of the collagen VII molecule
Comparison of how non-collagenous region 1 of the collagen VII molecule (NC1) status relates to wound healing and safety profile (if applicable).
Time frame: 6 months
Comparison of how circulating C7 antibody status relates to wound healing and safety profile
Comparison of how circulating C7 antibody status relates to wound healing and safety profile (if applicable).
Time frame: 6 months
Plan to share: No
No publications or documents are linked to this record.
Started Apr 2023 (actual). Primary completion is estimated for Sep 2025, 29 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.
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Epidermolysis Bullosa Dystrophica →
Abeona Therapeutics, Inc