Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01263379
• Date of registration: 15/12/2010
• Prospective Registration: No
• Primary sponsor: Stanford University
• Public title: Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
• Scientific title: A Phase 1/2A Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
• Date of first enrolment: October 5, 2010
• Target sample size: 10
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT01263379
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Jean Tang, MD, PhD
• Affiliation: Stanford University

Key inclusion & exclusion criteria

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.



Age minimum: 13 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa Dystrophica

Epidermolysis Bullosa

Intervention(s)

Biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets

Primary Outcome(s)

Percentage surface area of wound healing [Time Frame: 3, 6 and 12 months post grafting]

Investigator's assessment of graft [Time Frame: 3, 6 and 12 months post grafting]

Secondary Outcome(s)

duration of type VII collagen production [Time Frame: 12 weeks, 25 weeks, and 52 weeks post grafting]

Secondary ID(s)

eProtocol 14563

IND# 13708

RAC Protocol # 0701-827

R01AR055914

SU-10202010-7130

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Abeona Therapeutics, Inc

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)