A study to evaluate QR-313 exposure in subjects with RDEB
Research type
Research Study
Full title
A FIRST IN HUMAN, DOUBLE-BLIND, RANDOMIZED, INTRA-SUBJECT PLACEBO-CONTROLLED, MULTIPLE DOSE STUDY OF QR-313 EVALUATING SAFETY, PROOF OF MECHANISM, PRELIMINARY EFFICACY AND SYSTEMIC EXPOSURE IN SUBJECTS WITH RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA (RDEB) DUE TO MUTATION(S) IN EXON 73 OF THE COL7A1 GENE
IRAS ID
241823
Contact name
Jemima Mellerio
Contact email
Sponsor organisation
ProQR Therapeutics
Eudract number
2017-004806-17
Duration of Study in the UK
1 years, 1 months, 1 days
Research summary
This study will look at the safety of an experimental drug called QR-313 in patients with Dystrophic Epidermolysis Bullosa (DEB), and whether this can provide a treatment to overcome the genetic defect in patients with one or more genetic mutations in exon 73 and therefore potentially improve the abnormal skin. This is a Phase I study and will be a first time in humans. QR-313 gel will be used in conjunction with a placebo gel. Study participation will be up to 22 weeks and involve 7 visits, either in hospital or at home. It is anticipated that between 8 to 14 participants will be included in the study aged 2 years and above. The study drug/placebo gel will be applied two or three times per week on the selected wound area(s) over a period of 4 weeks.\n\nThere are two possible scenarios when selecting the wound areas:\n\nScenario 1: The study doctor will select two wound areas (of 10 x 10 cm each / 4 inches x 4 inches). One wound area will be treated with QR-313 for 4 weeks and the other wound area will be treated with placebo for 4 weeks.\n\nScenario 2: The study doctor will select one wound area (of 10 x 10 cm / 4 inches x 4 inches). This wound area will be treated with either QR-313 or placebo for 4 weeks. At Visit 4 the subject will be asked to return to the study site and the study doctor will check if a second wound area can be selected for treatment. If a second wound area is selected, this wound area will be treated for 4 weeks with the opposite treatment from the first wound area.\n
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
18/YH/0101
Date of REC Opinion
18 May 2018
REC opinion
Further Information Favourable Opinion