dystrophic epidermolysis bullosa
Conditions
Interventions
WG1025-phase I:External Application
1 application in week1 and 2 applications during week2~26
placebo-phase II:External Application
2 application during week1~26
WG1025-phase II:External Application,External Application
WG1025-phase II extension:External Application, External Application
Sponsors
Dermatology Hospital of Southern Medical University
Eligibility
Sex/Gender
All
Age
2 Years to 100 Years
Inclusion criteria
Inclusion criteria: 1. Subjects clinically diagnosed as DEB; 2. Confirmation of DEB diagnosis (DDEB or RDEB) through genetic testing including COL7A1; 3. >= 2 years old at the time of signing the ICF; 4. Fertile men or women must use a reliable contraceptive method for the entire study period up to 3 months (90 days) after the last use of WG1025 (see Appendix 1); 5. Wounds that meet the following criteria (a and b for Phase II studies and b for phase I studies): (1) Location: at least 1 pair of wound pairs of subjects with similar anatomical symmetry or similar parts or symmetrical or similar parts of the same anatomical region with similar appearance; (2) Appearance: red base, good blood supply, clean wound, no infection; 6. As determined by the researcher, have the ability and willingness to understand the test, follow the test procedure, and return to the research center to complete the required visitors; 7. Subject or subject's legal guardian/legal representative/impartial witness must read, understand, and sign the ICF and must be able and willing to follow study procedures and instructions.
Exclusion criteria
Exclusion criteria: 1. Unable to return to the study site; 2. The presence of a disease or condition that, in the investigator's judgment, may interfere with the assessment of the safety and efficacy of the study treatment and the subject's compliance with the study follow-up/procedure; 3. Patients who received chemotherapy or immunotherapy (other than dupriuzumab injection) within 1 month before screening or within 5 half-lives of the drugs they received (whichever is older); 4. Evidence of current squamous cell carcinoma or history of squamous cell carcinoma; 5. Investigator judges that the subjects are drug abusers or drug and alcohol addicts; 6. Participants in interventional clinical trials within the past 3 months (90 days); 7. Allergic to local anesthesia (lidocaine/proclorcaine cream); 8. Subjects who have previously received skin grafting; 9. Pregnant or lactating women; 10. During screening, the wounds assessed by the investigator were not suitable for receiving the study drug administration; 11. Allergic to the investigational drug, including its excipients, gauze or dressing; 12. Subjects with active infections requiring treatment (except for stable inactive infections) and/or those with uncontrollable local wounds are excluded during the screening process; 13. Have serious cardiovascular disease, and the investigator determines that it is not suitable to participate in the clinical research; 14. Subjects with severe anemia (hemoglobin 3 times the upper limit of normal (ULN); or (2) Serum bilirubin > 3 × ULN; 16.Subjects with abnormal renal function: (1) Urea or urea nitrogen > 1.5 × ULN; or (2) Serum creatinine > 2 × ULN; 17. Subjects with active hepatitis B, active hepatitis C, positive human immunodeficiency virus antibody or positive anti-syphilis spirochete specific antibody; patients with stable hepatitis B who have received treatment [HBV DNA < 2000 IU/ml] can be included in the study. 18. Subjects with active or past autoimmune diseases that may recur (such as systemic lupus erythematosus, rheumatoid arthritis, vasculitis, etc.); However, subjects with clinically stable autoimmune thyroiditis disease or with autoimmune skin diseases that do not require systemic treatment, such as psoriasis, may be included. 19. Subjects who donate blood or lose large amounts of blood (= 400 ml), receive blood transfusions or use blood products within 3 months before or during screening; Or plan to donate blood during the trial or within 1 month after the trial ends; 20. The investigator believes that the subjects are not suitable for participating in this clinical study because of other serious systemic diseases or other reasons.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase I: The incidence of TEAE during treatment by week 5;Phase I: The changes of safety data by week 5;Phase II: After 3 months of treatment (8, 10, 12 weeks), the proportion of complete healing wounds in class A; | — |
Secondary
| Measure | Time frame |
|---|---|
| Phase I: The incidence of TEAE during treatment by week6~13, week14~26 and entire study;Phase I: The changes of safety data during treatment by week6~13, week14~26 and entire study;Phase I: The proportion of complete healing wounds in class A by week 9,11,13;Phase I: The proportion of complete healing wounds in class A by week 1,3,5,16,20,22,24,26;Phase I: The proportion of 75%,50% healing wounds in class A by week 1,3,5,16,20,22,24,26;Phase I & II: The duration of complete healing in class A;Phase I: The changes of EQ-5D by week 3,5,9,11,13,16,20,22,24,26;Phase I: The changes of Pain Scale by week 3,5,9,11,13,16,20,22,24,26;Phase I & II: Pharmacokinetic end points: DNA copy number of WG1025 in blood, urine, nasal secretions, wound/bandage swab samples and stool and WG1025 activity in swab samples;Phase I & II: Immunogenic end points: (1) Detection of anti-COL7 antibody and anti-HSV-1 binding antibody; (2) Detection of anti-HSV-1 neutralizing antibody;Phase II: After 6 months of treatment (22, 24, 26 weeks), the proportion of complete healing wounds in class A;Phase II: The proportion of 100%,75%,50% healing wounds in class A by week 2,4,8,10,12,16,20,22,24,26;Phase II: The changes of EQ-5D by week 2,4,8,10,12,16,20,22,24,26;Phase II: The changes of Pain Scale by week 2,4,8,10,12,16,20,22,2426; | — |
Countries
China
Contacts
Public ContactZhimiao LIN
Dermatology Hospital of Southern Medical University
Outcome results
None listed