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A Double-blind, Randomized, Intra-patient Placebo- Controlled, Multiple Dose Study of PTW-002 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy, and Systemic Exposure in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa (DDEB / RDEB) Due to Mutation(s) in Exon 73 of the COL7A1 Gene

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Trial Profile

A Double-blind, Randomized, Intra-patient Placebo- Controlled, Multiple Dose Study of PTW-002 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy, and Systemic Exposure in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa (DDEB / RDEB) Due to Mutation(s) in Exon 73 of the COL7A1 Gene

Status: Not yet recruiting
Phase of Trial: Phase I/II

Latest Information Update: 03 Apr 2023

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At a glance

  • Drugs QR 313 (Primary)
  • Indications Epidermolysis bullosa dystrophica
  • Focus Adverse reactions; Biomarker; Pharmacodynamics; Proof of concept
  • Sponsors Phoenicis Therapeutics

Most Recent Events

  • 29 Mar 2023 Planned End Date changed from 31 Jul 2023 to 31 Jul 2024.
  • 29 Mar 2023 Planned primary completion date changed from 30 Apr 2023 to 30 Apr 2024.
  • 29 Mar 2023 Planned initiation date changed from 1 Oct 2022 to 30 Apr 2023.

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