A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

• Status: Recruiting
• Trial end date: 0000-00-00
• Target enrollment: 12
• Participant gender: Both

Summary

The purpose of this study is to evaluate the safety of FCX-007, evaluate C7 expression and the presence of anchoring fibrils resulting from FCX-007 and to analyze wound healing as a result of FCX-007 administration in subjects with RDEB.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Fibrocell Technologies, Inc.
• Collaborator: Intrexon Corporation
• Last Updated: 2016-08-23

Criteria

Inclusion Criteria:

1. Age

1. Phase I (subjects 1 through 6): Eighteen (18) years or older.

2. Phase II (subjects 7 through 12): Seven (7) years or older.

2. Diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)

3. NC1/NC2 Status (to be tested for if unknown)

4. Subjects, who are, in the opinion of the Investigator, able to understand the study, co-operate with the study procedures and are willing to return to the clinic for all the required follow-up visits

Exclusion Criteria:

1. Medical instability limiting ability to travel to the investigative center.

2. Active infection with HIV, hepatitis B or hepatitis C

3. A positive study specific immunofluorescence result

4. Evidence of systemic infection

5. Current evidence of metastatic squamous cell carcinoma at the site to be injected

6. Known allergy to any of the constituents of the product

7. Active drug or alcohol addiction

8. Hypersensitivity to local anesthesia

9. Receipt of a chemical or biological study product for the specific treatment of RDEB in the past six months

10. Women who are pregnant or breast-feeding

11. Abnormal clinically significant laboratory result

12. Clinically significant abnormalities on NCI toxicity scale

13. Wounds greater than or equal to 40^2 cm that are located on a mucous membrane, face or have an active infection