ProQR Therapeutics is currently recruiting for WINGS, a Phase 1b/2 research study of QR-313 in patients 6 years and older with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in a specific part of the COL7A1 gene called exon 73. QR-313 gel will be applied directly onto wounded skin. QR-313 is an experimental drug designed to exclude exon 73 (exon skipping) from the COL7A1 mRNA (the ‘blueprint’ for making proteins) with the aim to restore functionality of collagen type VII protein. This in turn may potentially lead to the formation of anchoring fibrils that bind the dermal (inner) and epidermal (outer) skin layers together, improved wound healing and skin strength.
The main objectives of WINGS are to evaluate safety after administration of QR-313 to wounded skin and to evaluate whether QR-313 is able to exclude (skip) exon 73 from the COL7A1 mRNA. The effects of QR-313 on wound healing, on skin strength and on the presence of collagen type VII protein and anchoring fibrils following treatment will also be assessed. During the study, QR-313 or a matching placebo gel, will be applied to wounded skin two to three times a week (at the time of changing of wound dressings) for four weeks with an eight week follow-up period. WINGS is planned to be conducted at specialized EB centers in the US and selected European countries.
- Male or female, ≥ 6 years of age at Screening with a clinical diagnosis of RDEB and at least one pathogenic mutation in exon 73 of the COL7A1 gene.
- Have at least one TWA, ie, a skin area of 7 x 7 cm that ishows no signs of local infection, and contains a target wound that is either new or shows dynamic wound healing and complies to the following additional criteria:
- surface area of the target wound ranging from 5 to 30 cm2, located centrally in the selected 7 x 7 cm TWA.
- exposed sub-epidermal tissue to allow absorption of the IMP.
- no suspicion of current squamous cell carcinoma (SCC) upon visual inspection.
- Please click the link below for exclusion criteria.