NCT05529134

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

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: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Phoenicis Therapeutics · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	PTW-002 10 mg/g gel	poloxamer hydrogel for topical administration
DRUG	Placebo	placebo poloxamer hydrogel for topical administration

Timeline

• Start date: 2023-04-30
• Primary completion: 2024-04-30
• Completion: 2024-07-31
• First posted: 2022-09-06
• Last updated: 2023-03-31

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study