Clinical trial NCT02579369: Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa
Official title: A Phase 1/2 Clinical Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Dystrophic Epidermolysis Bullosa
Keywords: Epidermolysis Bullosa
stem cell
Lead sponsor: Anterogen Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2015-10-19
Enrollment: 2
Sex: ALL
Minimum age (years): 10
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a phase I/II open-label study to evaluate the efficacy and safety of ALLO-ASC-DFU in patients with Dystrophic Epidermolysis Bullosa.

Detailed description: ALLO-ASC-DFU is a hydrogel sheet containing allogenic adipose-derived mesenchymal stem cells. Adipose-derived stem cells have anti-inflammatory effect and release growth factors such as vascular endothelial growth factor (VEGF) and hepatocyte growth factor (HGF), which can enhance wound healing and regeneration of new tissue, finally may provide an new option in treating a Dystrophic Epidermolysis Bullosa.

Eligibility criteria: Inclusion Criteria:

1. Age : 10\~60
2. Diagnosed with Dystrophic Epidermolysis Bullosa based on immunofluorescence test.
3. Bullous skin lesion sized over 10 cm\^2
4. Test negative for Serum β-HCG pregnancy test on screening, if the subject is fertile
5. A subject who is willing to follow the protocol and provide a informed consent on screening, given that the information with respect to the clinical trial is provided.

Exclusion Criteria:

1. A subject with history of epidermoid carcinoma within a year from screening.
2. A subject who requires antibiotics due to bacterial infection on skin.
3. A subject who was dosed with oral steroid, over 0.5mg/kg a day for subjects under 18, or over 20mg in 2 weeks for subjects over 18, within 30 days prior to screening.
4. A subject treated with radiotherapy or immunosuppressants, within 30 days prior to screening.
5. A subject treated with steroids locally, within 30 days prior to screening.
6. A subject with 2-times the maximum-standardized value of ALT, AST, ALP, bilirubin, total protein
7. A subject with 2-times the maximum-standardized value of BUN, Creatinine
8. A subject with Albumin below 2.0 g/dL.
9. A subject with Hemoglobin below 6 g/dL (anemic).
10. A subject with allergic response to bovine derived protein and fibrin glue.
11. A subject administered with biologic agents or cell therapy, within 30 days prior to screening.
12. A subject administered with stem cell treatment by IV or subcutaneously to the target wound, prior to the trial
13. A subject who enrolled into another clinical trial, within 30 days prior to screening
14. A subject with serious disease that can affect on clinical trial.
15. A pregnant or breast-feeding subject.
16. A subject with history of drug abuse within 1 year of clinical significance
17. A subject who cannot proceed according to the protocol.

Primary outcomes:
measure: Area of re-epithelization
timeFrame: Follow up to 8 weeks
Secondary outcomes:
measure: Percentage of a target wound's re-epithelialization
timeFrame: Every time of visit for follow up to 8 weeks
measure: Time taken to re-epithelization
timeFrame: Over 8 weeks
measure: Safety assessed by clinically measured abnormality of laboratory tests and adverse events
timeFrame: Over 8 weeks
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Gangnam Severence Hospital
Study officials:
affiliation: Gangnam Severence Hospital
name: Su Chan Kim, PhD.
role: PRINCIPAL_INVESTIGATOR
References: