Overview

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 Path

Status:
Terminated
Trial end date:
2018-12-17
Target enrollment:
Participant gender:
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Phoenicis Therapeutics
ProQR Therapeutics