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

Trial Profile

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

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 20 Jul 2018

At a glance

  • Drugs QR 313 (Primary)
  • Indications Epidermolysis bullosa
  • Focus Adverse reactions; First in man; Proof of concept
  • Acronyms WINGS
  • Sponsors ProQR Therapeutics
  • Most Recent Events

    • 28 Jun 2018 Status changed from planning to recruiting, according to a ProQR Therapeutics media release.
    • 12 Jun 2018 According to a ProQR Therapeutics media release, this trial is expected to initiate patient enrollment in the second quarter of 2018 and is expected to report interim proof of mechanism results later in 2018.
    • 28 Feb 2018 According to a ProQR media release, company expects interim data in 2018 and full data in 2019.
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