NCT05143190

Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)

A Phase 2b Open-Label Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01 in Study PTR-01-002

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Phoenix Tissue Repair, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.

Detailed description

Protocol PTR-01-003 is a 4-part Phase 2, open-label study of PTR-01 in patients who satisfactorily completed study PTR-01-002 and meet current enrollment criteria. In Part 1, patients will be monitored monthly for up to 2 months with patient-reported and Investigator assessments. In Part 2, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 3 in which patients will receive a dose of 3.0 mg/kg monthly for a total of 5 additional doses. At the end of each dosing period, efficacy assessments will be performed. During Part 4, patients will be evaluated at Months 1 and 3 after completion of dosing to assess the durability of wound healing and other efficacy parameters. Safety will be assessed continuously throughout the study.

Conditions

• Recessive Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	PTR-01	Intravenous recombinant collagen 7

Timeline

• Start date: 2021-12-02
• Primary completion: 2022-06-20
• Completion: 2022-08-09
• First posted: 2021-12-03
• Last updated: 2022-11-21

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study