Clinical Trials
Catherine Curtin, MD
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Eligibility
Inclusion Criteria:
1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
2. 13 years old or older and willing and able to give assent/consent
3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
4. NC1[+] and mAb LH24 antibody staining negative
5. RDEB type VII collagen mutations in subject and carrier parents confirmed
6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
suitable for skin grafting
7. Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion Criteria:
1. Medical instability limiting ability to travel to Stanford University Medical Center
2. The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with HIV, hepatitis B or
hepatitis C, as determined by hepatitis B surface antigen screening, detection of
hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction
(PCR) analysis.
3. Antibodies to type VII collagen associated antigens
4. Active infection in the area that will undergo grafting
5. Evidence of systemic infection
6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
grafting
7. Active drug or alcohol addiction
8. Hypersensitivity to vancomycin or amikacin
9. Receipt of chemical or biological study product for the specific treatment of RDEB in
the past six months
10. Positive pregnancy test or breast-feeding
11. Clinically significant 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:
- Albumin < 2.5 g/dL
- Leukocytes > 20K/uL
- Hemoglobin < 7.5 g/dL. Low hemoglobin will be treated at the discretion of the
investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
12. 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
Ages Eligible for Study
13 Years - N/A
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Kylie Loutit, MS
650-724-1982
Not Recruiting
Dyani Gaudilliere, MD
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Eligibility
Inclusion Criteria:
1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
2. 13 years old or older and willing and able to give assent/consent
3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
4. NC1[+] and mAb LH24 antibody staining negative
5. RDEB type VII collagen mutations in subject and carrier parents confirmed
6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
suitable for skin grafting
7. Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion Criteria:
1. Medical instability limiting ability to travel to Stanford University Medical Center
2. The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with HIV, hepatitis B or
hepatitis C, as determined by hepatitis B surface antigen screening, detection of
hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction
(PCR) analysis.
3. Antibodies to type VII collagen associated antigens
4. Active infection in the area that will undergo grafting
5. Evidence of systemic infection
6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
grafting
7. Active drug or alcohol addiction
8. Hypersensitivity to vancomycin or amikacin
9. Receipt of chemical or biological study product for the specific treatment of RDEB in
the past six months
10. Positive pregnancy test or breast-feeding
11. Clinically significant 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:
- Albumin < 2.5 g/dL
- Leukocytes > 20K/uL
- Hemoglobin < 7.5 g/dL. Low hemoglobin will be treated at the discretion of the
investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
12. 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
Ages Eligible for Study
13 Years - N/A
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Kylie Loutit, MS
650-724-1982
Not Recruiting
David Kahn, MD
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Eligibility
Inclusion Criteria:
1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
2. 13 years old or older and willing and able to give assent/consent
3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
4. NC1[+] and mAb LH24 antibody staining negative
5. RDEB type VII collagen mutations in subject and carrier parents confirmed
6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
suitable for skin grafting
7. Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion Criteria:
1. Medical instability limiting ability to travel to Stanford University Medical Center
2. The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with HIV, hepatitis B or
hepatitis C, as determined by hepatitis B surface antigen screening, detection of
hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction
(PCR) analysis.
3. Antibodies to type VII collagen associated antigens
4. Active infection in the area that will undergo grafting
5. Evidence of systemic infection
6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
grafting
7. Active drug or alcohol addiction
8. Hypersensitivity to vancomycin or amikacin
9. Receipt of chemical or biological study product for the specific treatment of RDEB in
the past six months
10. Positive pregnancy test or breast-feeding
11. Clinically significant 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:
- Albumin < 2.5 g/dL
- Leukocytes > 20K/uL
- Hemoglobin < 7.5 g/dL. Low hemoglobin will be treated at the discretion of the
investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
12. 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
Ages Eligible for Study
13 Years - N/A
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Kylie Loutit, MS
650-724-1982
Not Recruiting
Gordon Lee, MD
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Eligibility
Inclusion Criteria:
1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
2. 13 years old or older and willing and able to give assent/consent
3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
4. NC1[+] and mAb LH24 antibody staining negative
5. RDEB type VII collagen mutations in subject and carrier parents confirmed
6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
suitable for skin grafting
7. Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion Criteria:
1. Medical instability limiting ability to travel to Stanford University Medical Center
2. The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with HIV, hepatitis B or
hepatitis C, as determined by hepatitis B surface antigen screening, detection of
hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction
(PCR) analysis.
3. Antibodies to type VII collagen associated antigens
4. Active infection in the area that will undergo grafting
5. Evidence of systemic infection
6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
grafting
7. Active drug or alcohol addiction
8. Hypersensitivity to vancomycin or amikacin
9. Receipt of chemical or biological study product for the specific treatment of RDEB in
the past six months
10. Positive pregnancy test or breast-feeding
11. Clinically significant 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:
- Albumin < 2.5 g/dL
- Leukocytes > 20K/uL
- Hemoglobin < 7.5 g/dL. Low hemoglobin will be treated at the discretion of the
investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
12. 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
Ages Eligible for Study
13 Years - N/A
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Kylie Loutit, MS
650-724-1982
Not Recruiting
H. Peter Lorenz, MD
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Eligibility
Inclusion Criteria:
1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
2. 13 years old or older and willing and able to give assent/consent
3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
4. NC1[+] and mAb LH24 antibody staining negative
5. RDEB type VII collagen mutations in subject and carrier parents confirmed
6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
suitable for skin grafting
7. Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion Criteria:
1. Medical instability limiting ability to travel to Stanford University Medical Center
2. The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with HIV, hepatitis B or
hepatitis C, as determined by hepatitis B surface antigen screening, detection of
hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction
(PCR) analysis.
3. Antibodies to type VII collagen associated antigens
4. Active infection in the area that will undergo grafting
5. Evidence of systemic infection
6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
grafting
7. Active drug or alcohol addiction
8. Hypersensitivity to vancomycin or amikacin
9. Receipt of chemical or biological study product for the specific treatment of RDEB in
the past six months
10. Positive pregnancy test or breast-feeding
11. Clinically significant 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:
- Albumin < 2.5 g/dL
- Leukocytes > 20K/uL
- Hemoglobin < 7.5 g/dL. Low hemoglobin will be treated at the discretion of the
investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
12. 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
Ages Eligible for Study
13 Years - N/A
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Kylie Loutit, MS
650-724-1982
Not Recruiting
Arash Momeni, MD
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Eligibility
Inclusion Criteria:
1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
2. 13 years old or older and willing and able to give assent/consent
3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
4. NC1[+] and mAb LH24 antibody staining negative
5. RDEB type VII collagen mutations in subject and carrier parents confirmed
6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
suitable for skin grafting
7. Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion Criteria:
1. Medical instability limiting ability to travel to Stanford University Medical Center
2. The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with HIV, hepatitis B or
hepatitis C, as determined by hepatitis B surface antigen screening, detection of
hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction
(PCR) analysis.
3. Antibodies to type VII collagen associated antigens
4. Active infection in the area that will undergo grafting
5. Evidence of systemic infection
6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
grafting
7. Active drug or alcohol addiction
8. Hypersensitivity to vancomycin or amikacin
9. Receipt of chemical or biological study product for the specific treatment of RDEB in
the past six months
10. Positive pregnancy test or breast-feeding
11. Clinically significant 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:
- Albumin < 2.5 g/dL
- Leukocytes > 20K/uL
- Hemoglobin < 7.5 g/dL. Low hemoglobin will be treated at the discretion of the
investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
12. 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
Ages Eligible for Study
13 Years - N/A
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Kylie Loutit, MS
650-724-1982
Not Recruiting
Dung Nguyen, MD
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
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.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Eligibility
Inclusion Criteria:
1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
2. 13 years old or older and willing and able to give assent/consent
3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
4. NC1[+] and mAb LH24 antibody staining negative
5. RDEB type VII collagen mutations in subject and carrier parents confirmed
6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
suitable for skin grafting
7. Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion Criteria:
1. Medical instability limiting ability to travel to Stanford University Medical Center
2. The presence of medical illness expected to complicate participation and/or compromise
the safety of this technique, such as active infection with HIV, hepatitis B or
hepatitis C, as determined by hepatitis B surface antigen screening, detection of
hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction
(PCR) analysis.
3. Antibodies to type VII collagen associated antigens
4. Active infection in the area that will undergo grafting
5. Evidence of systemic infection
6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
grafting
7. Active drug or alcohol addiction
8. Hypersensitivity to vancomycin or amikacin
9. Receipt of chemical or biological study product for the specific treatment of RDEB in
the past six months
10. Positive pregnancy test or breast-feeding
11. Clinically significant 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:
- Albumin < 2.5 g/dL
- Leukocytes > 20K/uL
- Hemoglobin < 7.5 g/dL. Low hemoglobin will be treated at the discretion of the
investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
12. 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the
EB physician.
Ages Eligible for Study
13 Years - N/A
Genders Eligible for Study
All
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Kylie Loutit, MS
650-724-1982
Not Recruiting