Recessive Dystrophic Epidermolysis Bullosa
Conditions
Interventions
Test group:Topical DT01
Sponsors
Xiangya Hospital, Central South University
Eligibility
Sex/Gender
All
Age
2 Years to 60 Years
Inclusion criteria
Inclusion criteria: 1. Able to understand and voluntarily sign the written informed consent form. 2. Aged 2 to 60 years (inclusive of 2 and 60 years), with no gender restrictions. 3. Meets the clinical diagnosis of recessive dystrophic epidermolysis bullosa. 4. Confirmed by genetic testing to have pathogenic mutations in COL7A1, confirmed by immunofluorescence to have reduced or absent COL7A1 protein, and confirmed by immune electron microscopy to have reduced or absent anchoring fibrils. 5. The same patient has at least two skin wounds (to be treated with DT-101 formulation and placebo respectively) that meet the following criteria: a. Location: Similar in size (>=3cm²), located in similar anatomical regions, and similar in appearance; b. Appearance: Clean, with sufficient granulation tissue, well-vascularized, and free of infection; c. Location of the wound area: Located on the anterior and/or lateral trunk and/or upper limbs and/or lower limbs, protected from frequent trauma or friction. Excluded areas include the face and areas close to mucous membranes (urogenital, oral, or anal mucosa). 6. The investigator believes that the subject and caregiver are able to understand the study, comply with the study procedures, and are willing to return to the hospital for all necessary follow-ups. 7. Males and females of reproductive potential must use a reliable method of contraception throughout the study period and for 3 months after the last administration of DT-101. 8. Female subjects of childbearing age have a negative pregnancy test at the initial visit.
Exclusion criteria
Exclusion criteria: 1. Inability to visit the hospital as required by the study schedule due to various reasons. 2. Any disease or condition determined by the investigator that may interfere with the assessment of the safety and efficacy of the study treatment, as well as the subject’s compliance with study visits/procedures. 3. The presence of other diseases that are expected to complicate participation and/or compromise the safety of this technique, such as active infection with human immunodeficiency virus (HIV), hepatitis B (as determined by screening for hepatitis B surface antigen), or hepatitis C (as determined by a positive test for hepatitis C antibody or a positive result of hepatitis C polymerase chain reaction (PCR) analysis). 4. Active infection in the area to be treated. 5. Evidence of systemic infection. 6. Known allergy to any component of the product. 7. Existing evidence or history of squamous cell carcinoma in the area to be treated. 8. Active drug or alcohol addiction as determined by the investigator. 9. The subject is actively receiving chemotherapy or immunotherapy at Visit 1 (Week 1). 10. Allergy to topical anesthetics (lidocaine/prilocaine cream). 11. Use of any chemical or biological investigational product for the specific treatment of RDEB within the past 3 months. 12. Previous receipt of investigational gene or cell therapy for specific wounds. 13. Receipt of skin grafting within the past 3 months. 14. Subjects who have taken systemic antibiotics within 7 days. 15. Pregnant or breastfeeding women. 16. Clinically significant abnormalities as determined by the investigator, including but not limited to hemoglobin <=60 g/L, albumin <=20 g/L, etc. 17. Other reasons determined by the investigator that make the subject unsuitable for participation in this trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Wound healing status after treatment; | — |
Secondary
| Measure | Time frame |
|---|---|
| Assess the safety of formulation DT-101; | — |
Countries
China
Contacts
Public ContactJuan Su
Xiangya Hospital, Central South University
Outcome results
None listed