A Phase 1/2 interventional study of LZRSE-Col7A1 Engineered Autologous Epidermal Sheets in Epidermolysis Bullosa Dystrophica and Epidermolysis Bullosa, sponsored by Abeona Therapeutics, Inc. Completed at 1 site in United States. Open to participants aged 13 Years and older. Per ClinicalTrials.gov, last updated 2023-08-22.
Sponsored by Abeona Therapeutics, Inc · Phase 1/2, Interventional, and Treatment
This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen. The corrected cells will be transplanted back to the patient.
The research project involves gene transfer into keratinocytes, which are the majority of the cells in the outer layer of skin. In this gene transfer trial we plan to biopsy some skin tissue, grow the cells in a skin cell culture (sterile dishes with special fluid that allows cells to grow and multiply) and then infect the cells with a virus that we have genetically engineered to insert the correct type VII collagen gene. The cells should then make type VII collagen.
The process of inserting the correct type VII collagen gene into cells is called "gene transfer." The virus used is called a "retrovirus." The virus is made so that it only delivers the type VII collagen gene and it should not spread to other parts of the body. During the study we will check for growth of the virus.
After cells have received gene transfer, we will grow the cells in culture into a sheet of cells that look like a plastic film. We plan to graft the sheet to wounds. Grafting means we will take cells from the culture and stitch them to the patient's skin.
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 21 are open to participants now.
This study's 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.
Counted across the registry records on this site, refreshed daily.
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.
Exclusion Criteria:
abnormalities (Grade 2 or higher on the National Cancer Institute [NCI] toxicity scale) on laboratory tests performed prior to grafting, except for the following specific exclusionary laboratory threshold results, subject to approval or exemption by the EB physician:
Clinically significant abnormalities (Grade 2 or higher on the NCI toxicity scale) identified through medical history and physical examination on Day 0, with the following exceptions:
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.
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.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
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.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
Biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
This trial will create a graft, which we call "LEAES", of the patient's own skin that has been genetically engineered in our lab to express this missing protein.
Also known as: LEAES
Number of Wounds by Healing Category Per Investigator Visual Assessment
The graft site was clinically evaluated by the investigator with a global score of: 1) 100% to 75% healed, 2) 74% to 50% healed, 3) 49% or less healed with 100% meaning completely healed.
Time frame: 3, 6, 12 and 24 months post grafting
Percentage Surface Area of Wound Healing
Percentage of wound area will be obtained using the Canfield system. Changes in dimensions between visits as well as changes in dimensions from baseline will be recorded. Dimensions of untreated wounded skin will be used for comparison
Time frame: 3, 6 and 12 months post grafting
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
Skin biopsies were obtained to evaluate expression of type VII collagen, NC2 epitope, using immuno-electron microscopy and immuno-fluorescent light microscopy.
Time frame: 3 months, 6 months, 12 months, 24 months post-grafting
Number of Participants With Presence of Anchoring Fibrils (AF)
Skin biopsies were obtained to observe physical development of the anchoring fibrils using electron microscopy
Time frame: 3 months, 6 months, 12 months and 24 months post grafting
| Milestone | LEAES Treatment |
|---|---|
| Started | 7 |
| Completed | 7 |
| Not completed | 0 |
The graft site was clinically evaluated by the investigator with a global score of: 1) 100% to 75% healed, 2) 74% to 50% healed, 3) 49% or less healed with 100% meaning completely healed.
| wounds | LEAES Treatment |
|---|---|
| 3 months — Global Score of 100% to 75% | 35 |
| 3 months — Global Score of 74% to 50% | 5 |
| 3 months — Global Score of 49% or less | 2 |
| 6 months — Global Score of 100% to 75% | 28 |
| 6 months — Global Score of 74% to 50% | 3 |
| 6 months — Global Score of 49% or less | 11 |
| 12 months — Global Score of 100% to 75% | 19 |
| 12 months — Global Score of 74% to 50% | 11 |
| 12 months — Global Score of 49% or less | 12 |
| 24 months — Global Score of 100% to 75% | 26 |
| 24 months — Global Score of 74% to 50% | 5 |
| 24 months — Global Score of 49% or less | 11 |
Percentage of wound area will be obtained using the Canfield system. Changes in dimensions between visits as well as changes in dimensions from baseline will be recorded. Dimensions of untreated wounded skin will be used for comparison
| percentage of wound area | 3 Months Post LEAES Treatment | 6 Months Post LEAES Treatment | 12 Months Post LEAES Treatment |
|---|---|---|---|
| GT04, Wound A | 100 | 100 | 100 |
| GT04, Wound B | 100 | 100 | 100 |
| GT04, Wound C | 100 | 100 | 100 |
| GT04, Wound D | 100 | 100 | 97.5 |
| GT04, Wound E | 100 | 100 | 100 |
| GT04, Wound Z - induced | 100 | 35 | 90 |
| GT05, Wound A | 96 | 90.6 | 78.6 |
| GT05, Wound B | 98 | 81.6 | 66.5 |
| GT05, Wound C | 91.5 | 71 | 92 |
| GT05, Wound D | 93.5 | 85.5 | 87.5 |
| GT05, Wound E | 96 | 74.6 | 75.7 |
| GT05, Wound Z - induced | 100 | 100 | 100 |
| GT07, Wound A | 93.5 | 96 | 70 |
| GT07, Wound B | 89.8 | 99 | 26 |
| GT07, Wound C | 99 | 32 | 2 |
| GT07, Wound D | 96.67 | 100 | — |
| GT07, Wound E | 89.45 | 94.5 | 100 |
| GT07, Wound F | 99 | 97 | 95 |
| GT08, Wound A | 70 | 54 | 12 |
| GT08, Wound B | 98.7 | 81 | 48 |
| GT08, Wound C | 100 | 55 | 44 |
| GT08, Wound D | 82 | 73 | 29 |
| GT08, Wound E | 100 | 84 | 10 |
| GT08, Wound F | 70 | 48.8 | 31 |
| GT09, Wound A | 92 | 100 | 85 |
| GT09, Wound B | 100 | 100 | 100 |
| GT09, Wound C | 79 | 74 | 78.56 |
| GT09, Wound D | 100 | 82 | 78.67 |
| GT09, Wound E | 100 | 100 | 98.7 |
| GT09, Wound F | 92 | 76 | 78.6 |
Skin biopsies were obtained to evaluate expression of type VII collagen, NC2 epitope, using immuno-electron microscopy and immuno-fluorescent light microscopy.
| Participants | LEAES Treatment |
|---|---|
| 3 months — Positive for NC2 | 7 |
| 3 months — Negative for NC2 | 0 |
| 3 months — Not done | 0 |
| 6 months — Positive for NC2 | 5 |
| 6 months — Negative for NC2 | 2 |
| 6 months — Not done | 0 |
| 12 months — Positive for NC2 | 2 |
| 12 months — Negative for NC2 | 4 |
| 12 months — Not done | 1 |
| 24 months — Positive for NC2 | 2 |
| 24 months — Negative for NC2 | 1 |
| 24 months — Not done | 4 |
Skin biopsies were obtained to observe physical development of the anchoring fibrils using electron microscopy
| Participants | LEAES Treatment |
|---|---|
| 3 months — Positive for AFs | 6 |
| 3 months — Negative for AFs | 1 |
| 3 months — Not done | 0 |
| 6 months — Positive for AFs | 5 |
| 6 months — Negative for AFs | 2 |
| 6 months — Not done | 0 |
| 12 months — Positive for AFs | 3 |
| 12 months — Negative for AFs | 3 |
| 12 months — Not done | 1 |
| 24 months — Positive for AFs | 1 |
| 24 months — Negative for AFs | 1 |
| 24 months — Not done | 5 |
Collected over 2 years post receiving LEAES cell sheets. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| LEAES Treatment | 0/7 (0%) | 2/7 (28.6%) | 7/7 (100%) |
| Event | LEAES Treatment |
|---|---|
| Neoplasms benign, malignant and unspecified, squamous cell carcinomaSkin and subcutaneous tissue disorders | 2/7 |
| Event | LEAES Treatment |
|---|---|
| Wound infectionSkin and subcutaneous tissue disorders | 6/7 |
| PruritusSkin and subcutaneous tissue disorders | 3/7 |
| PainSkin and subcutaneous tissue disorders | 2/7 |
| Wound complicationSkin and subcutaneous tissue disorders | 1/7 |
| Postoperative hemorrhageSurgical and medical procedures | 1/7 |
| Age, Categorical(Participants) | LEAES Treatment |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 7 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | LEAES Treatment |
|---|---|
| Female | 2 |
| Male | 5 |
| Ethnicity (NIH/OMB)(Participants) | LEAES Treatment |
|---|---|
| Hispanic or Latino | 3 |
| Not Hispanic or Latino | 4 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | LEAES Treatment |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 7 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | LEAES Treatment |
|---|---|
| United States | 7 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Results will be submitted to scientific journals for publication and presented at scientific meetings.
Supporting information: Study protocol
Time frame: Study protocol is attached to this submission and will be available per ClinicalTrials.gov timeline.
This study is completed, as verified in Aug 2023. You cannot join it, but the record below documents what was studied.
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Epidermolysis Bullosa Dystrophica →
Abeona Therapeutics, Inc