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 - WINGS

Wings Therapeutics Inc.0 个研究点目标入组 8 人开始时间: 2018年2月8日最近更新: 2019年12月10日

试验速览

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•1. Male or female, = 6 years of age at Screening with a clinical diagnosis of RDEB with confirmation of at least one of the alleles of the COL7A1 gene containing one or more pathogenic mutations in exon 73.
•2. Have at least one TWA of 10 x 10 cm that shows dynamic wound healing, no signs of local infection and in which a wound can be selected with the following criteria:
•a. a surface area ranging from 5 to 60 cm2
•b. open wound that has healing potential
•c. exposed sub-epidermal tissue to allow absorption of the IMP
•d. no suspicion of current squamous cell carcinoma (SCC) upon visual inspection
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 4
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Pregnant or breast-feeding female
•2. Hemoglobin level at Screening requiring transfusion. The subject may be rescreened when the condition is considered stable
•3. Untreated carcinoma of the TWA or history of carcinoma within 5 years prior to Screening, except adequately treated cutaneous squamous or basal cell carcinoma.
•4. Life expectancy less than 6 months, as assessed by the Investigator
•5. Current or known history of clinically significant hepatic or renal disease, that in the opinion of the Investigator, could impact subject safety or study participation.
•6. Treatment with any systemic immunomodulators, immunosuppressants or cytotoxic chemotherapy within 2 months prior to the Baseline visit.
•7. Known hypersensitivity to oligonucleotide treatment or any of the excipients of the IMP (see Section 3.1 in the IB)

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