Trial Outcomes & Findings for Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (NCT NCT01263379)
NCT ID: NCT01263379
Last Updated: 2023-08-22
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
12 participants
Primary outcome timeframe
3, 6, 12 and 24 months post grafting
Results posted on
2023-08-22
Participant Flow
12 participants signed informed consent, 7 were allocated to the treatment arm.
Participant milestones
| Measure |
LEAES Treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
|---|---|
|
Overall Study
STARTED
|
7
|
|
Overall Study
COMPLETED
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Baseline characteristics by cohort
| Measure |
LEAES Treatment
n=7 Participants
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
7 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3, 6, 12 and 24 months post graftingPopulation: The 7 participants who were determined to be eligible to receive LEAES cell sheets received 6 cell sheets to 6 wounds, each which were analyzed in this study as the primary endpoint.
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.
Outcome measures
| Measure |
LEAES Treatment
n=42 wounds
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
6 Months Post LEAES Treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
12 Months Post LEAES Treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
|---|---|---|---|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
3 months · Global Score of 100% to 75%
|
35 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
3 months · Global Score of 74% to 50%
|
5 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
3 months · Global Score of 49% or less
|
2 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
6 months · Global Score of 100% to 75%
|
28 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
6 months · Global Score of 74% to 50%
|
3 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
6 months · Global Score of 49% or less
|
11 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
12 months · Global Score of 100% to 75%
|
19 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
12 months · Global Score of 74% to 50%
|
11 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
12 months · Global Score of 49% or less
|
12 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
24 months · Global Score of 100% to 75%
|
26 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
24 months · Global Score of 74% to 50%
|
5 wounds
|
—
|
—
|
|
Number of Wounds by Healing Category Per Investigator Visual Assessment
24 months · Global Score of 49% or less
|
11 wounds
|
—
|
—
|
PRIMARY outcome
Timeframe: 3, 6 and 12 months post graftingPopulation: The Canfield Imaging System was implemented after the first 2 participants received LEAES. Tattoos were not used when placing tattoos for these first 2 participants. Therefore, measurements using the Canfield Imaging System was not possible.
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
Outcome measures
| Measure |
LEAES Treatment
n=30 Wounds
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
6 Months Post LEAES Treatment
n=30 Wounds
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
12 Months Post LEAES Treatment
n=30 Wounds
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
|---|---|---|---|
|
Percentage Surface Area of Wound Healing
GT04, Wound A
|
100 percentage of wound area
|
100 percentage of wound area
|
100 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT04, Wound B
|
100 percentage of wound area
|
100 percentage of wound area
|
100 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT04, Wound C
|
100 percentage of wound area
|
100 percentage of wound area
|
100 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT04, Wound D
|
100 percentage of wound area
|
100 percentage of wound area
|
97.5 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT04, Wound E
|
100 percentage of wound area
|
100 percentage of wound area
|
100 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT04, Wound Z - induced
|
100 percentage of wound area
|
35 percentage of wound area
|
90 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT05, Wound A
|
96 percentage of wound area
|
90.6 percentage of wound area
|
78.6 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT05, Wound B
|
98 percentage of wound area
|
81.6 percentage of wound area
|
66.5 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT05, Wound C
|
91.5 percentage of wound area
|
71 percentage of wound area
|
92 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT05, Wound D
|
93.5 percentage of wound area
|
85.5 percentage of wound area
|
87.5 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT05, Wound E
|
96 percentage of wound area
|
74.6 percentage of wound area
|
75.7 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT05, Wound Z - induced
|
100 percentage of wound area
|
100 percentage of wound area
|
100 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT07, Wound A
|
93.5 percentage of wound area
|
96 percentage of wound area
|
70 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT07, Wound B
|
89.8 percentage of wound area
|
99 percentage of wound area
|
26 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT07, Wound C
|
99 percentage of wound area
|
32 percentage of wound area
|
2 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT07, Wound D
|
96.67 percentage of wound area
|
100 percentage of wound area
|
—
|
|
Percentage Surface Area of Wound Healing
GT07, Wound E
|
89.45 percentage of wound area
|
94.5 percentage of wound area
|
100 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT07, Wound F
|
99 percentage of wound area
|
97 percentage of wound area
|
95 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT08, Wound A
|
70 percentage of wound area
|
54 percentage of wound area
|
12 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT08, Wound B
|
98.7 percentage of wound area
|
81 percentage of wound area
|
48 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT08, Wound C
|
100 percentage of wound area
|
55 percentage of wound area
|
44 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT08, Wound D
|
82 percentage of wound area
|
73 percentage of wound area
|
29 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT08, Wound E
|
100 percentage of wound area
|
84 percentage of wound area
|
10 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT08, Wound F
|
70 percentage of wound area
|
48.8 percentage of wound area
|
31 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT09, Wound A
|
92 percentage of wound area
|
100 percentage of wound area
|
85 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT09, Wound B
|
100 percentage of wound area
|
100 percentage of wound area
|
100 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT09, Wound C
|
79 percentage of wound area
|
74 percentage of wound area
|
78.56 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT09, Wound D
|
100 percentage of wound area
|
82 percentage of wound area
|
78.67 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT09, Wound E
|
100 percentage of wound area
|
100 percentage of wound area
|
98.7 percentage of wound area
|
|
Percentage Surface Area of Wound Healing
GT09, Wound F
|
92 percentage of wound area
|
76 percentage of wound area
|
78.6 percentage of wound area
|
SECONDARY outcome
Timeframe: 3 months, 6 months, 12 months, 24 months post-graftingPopulation: The 7 participants who were determined to be eligible to receive LEAES cell sheets received 6 cell sheets to 6 wounds, each which were analyzed in this study as the primary endpoint.
Skin biopsies were obtained to evaluate expression of type VII collagen, NC2 epitope, using immuno-electron microscopy and immuno-fluorescent light microscopy.
Outcome measures
| Measure |
LEAES Treatment
n=7 Participants
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
6 Months Post LEAES Treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
12 Months Post LEAES Treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
|---|---|---|---|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
3 months · Positive for NC2
|
7 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
3 months · Negative for NC2
|
0 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
3 months · Not done
|
0 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
6 months · Positive for NC2
|
5 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
6 months · Negative for NC2
|
2 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
6 months · Not done
|
0 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
12 months · Positive for NC2
|
2 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
12 months · Negative for NC2
|
4 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
12 months · Not done
|
1 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
24 months · Positive for NC2
|
2 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
24 months · Negative for NC2
|
1 Participants
|
—
|
—
|
|
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
24 months · Not done
|
4 Participants
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 3 months, 6 months, 12 months and 24 months post graftingPopulation: The 7 participants who were determined to be eligible to receive LEAES cell sheets received 6 cell sheets to 6 wounds, each which were analyzed in this study as the primary endpoint.
Skin biopsies were obtained to observe physical development of the anchoring fibrils using electron microscopy
Outcome measures
| Measure |
LEAES Treatment
n=7 Participants
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
6 Months Post LEAES Treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
12 Months Post LEAES Treatment
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
|
|---|---|---|---|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
3 months · Positive for AFs
|
6 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
3 months · Negative for AFs
|
1 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
3 months · Not done
|
0 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
6 months · Positive for AFs
|
5 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
6 months · Negative for AFs
|
2 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
6 months · Not done
|
0 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
12 months · Positive for AFs
|
3 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
12 months · Negative for AFs
|
3 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
12 months · Not done
|
1 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
24 months · Positive for AFs
|
1 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
24 months · Negative for AFs
|
1 Participants
|
—
|
—
|
|
Number of Participants With Presence of Anchoring Fibrils (AF)
24 months · Not done
|
5 Participants
|
—
|
—
|
Adverse Events
LEAES Treatment
Serious events: 2 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
LEAES Treatment
n=7 participants at risk
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
|---|---|
|
Skin and subcutaneous tissue disorders
Neoplasms benign, malignant and unspecified, squamous cell carcinoma
|
28.6%
2/7 • Number of events 4 • 2 years post receiving LEAES cell sheets
|
Other adverse events
| Measure |
LEAES Treatment
n=7 participants at risk
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
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.
|
|---|---|
|
Skin and subcutaneous tissue disorders
Wound infection
|
85.7%
6/7 • Number of events 10 • 2 years post receiving LEAES cell sheets
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
42.9%
3/7 • Number of events 5 • 2 years post receiving LEAES cell sheets
|
|
Skin and subcutaneous tissue disorders
Pain
|
28.6%
2/7 • Number of events 2 • 2 years post receiving LEAES cell sheets
|
|
Skin and subcutaneous tissue disorders
Wound complication
|
14.3%
1/7 • Number of events 1 • 2 years post receiving LEAES cell sheets
|
|
Surgical and medical procedures
Postoperative hemorrhage
|
14.3%
1/7 • Number of events 1 • 2 years post receiving LEAES cell sheets
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place