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Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene

Dystrophic Epidermolysis Bullosa Clinical Trial

Official title:
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

Clinical Trial Summary

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 Dystrophic Epidermolysis Bullosa (DDEB) or Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene. Up to two RDEB patients 4 to 17 years of age and up to 6 DDEB patients 4 years of age and older will be enrolled.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05529134
Study type Interventional
Source Phoenicis Therapeutics
Contact Ramsey Johnson
Phone 978-726-1478
Email ramsey@phoenicistx.com
Status Not yet recruiting
Phase Phase 1/Phase 2
Start date April 30, 2023
Completion date July 31, 2024
View Details
See also
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