ProQR Therapeutics has started patient enrolment in the WINGS Phase l/ll clinical trial designed to investigate the safety and efficacy of QR-313 to treat patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disease.
The first-in-human, double-blind, randomised, intra-subject placebo controlled trial expects to enrol about eight subjects who are at least six years of age and have RDEB due to mutation(s) in exon 73 of the COL7A1 gene.
As part of the trial, a gel formulation of QR-313 or placebo will be topically applied to the patient’s wounds around every other day for up to four weeks with a subsequent eight-week observation period.
The subjects are expected to receive either active gel or placebo on two separate wounds.
The investigators of the trial will conduct up to four small skin biopsies and will analyse tissue for molecular endpoints.
The trial’s primary objectives are to examine the safety and tolerability of topically applied QR-313 and assess proof of mechanism (exclusion, or skipping, of exon 73 from COL7A1 mRNA assessed by polymerase chain reaction).
Its secondary objectives are to quantify blood levels of QR-313, evaluate effects on wound healing, skin strength, as well as the presence of collagen type 7 protein and anchoring fibrils in the skin.
Set to be conducted at specialised centres in the US and selected European countries, the trial aims to release interim proof of mechanism results within this year.
Based on the interim analysis, the trial may be adapted to adjust the frequency and/or method of topical delivery to further improve uptake and activity.
In addition, a clinical proof of concept will be performed to compare the rate, strength and stability of wounds treated with active gel to those treated with placebo.