A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene
Stopped: Low enrollment
United States, France2 participantsStarted 2018-07-02
Plain-language summary
A double-blind, randomized, intra-subject placebo-controlled, multicenter, multiple dose study, evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with confirmed DDEB or RDEB diagnosis with one or more pathogenic mutations in exon 73 in the COL7A1 gene.
Who can participate
Age range4 Years
SexALL
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Inclusion criteria
✓. Male or female, ≥ 4 years of age at Screening with a clinical diagnosis of DDEB or 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.
Exclusion criteria
✕. Pregnant or breast-feeding female
✕. Hemoglobin level at Screening requiring transfusion. The subject may be rescreened when the condition is considered stable.
✕. Use of aminoglycosides, by any route of administration, except eye drops, 7 days or 5 half-lives, whichever is longer, prior to Baseline visit.
What they're measuring
1
Incidence of treatment emergent adverse events/serious adverse events
Timeframe: through 8 weeks after last dose of IMP (EOS)
2
To assess the effect of QR-313 on the exclusion (skipping) of exon 73 from COL7A1 mRNA
✕. Untreated carcinoma of the TWA or history of carcinoma within 5 years prior to Screening, except adequately treated cutaneous squamous or basal cell carcinoma.
✕. Life expectancy less than 6 months, as assessed by the Investigator
✕. 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.
✕. Treatment with any systemic immunomodulators, immunosuppressants or cytotoxic chemotherapy within 2 months prior to the Baseline visit.
✕. Use of any investigational drug or device within 28 days or 5 half-lives of the Baseline visit, whichever is longer, or plans to participate in another study of a drug or device during the study period. The washout of 5 half-lives does not apply to gene and cell therapy.