A Phase 1 / 2 Trial of PTR-01 in Adult Patients with Recessive Dystrophic Epidermolysis Bullosa (PTR-01-001)

Status: Open / Recruiting


  • •• Diagnosis of RDEB based on genetic analysis showing 2 confirmed RDEB type VII collagen mutations consistent with a recessive inheritance pattern
  • •• 18 years or older
  • •• Presence of some but deficient type VII collagen (determined in the Characteristics study)
  • •• At least 1 unhealed wound ≥ 20 cm2 (about the size of 4 grapes) for at least 6 weeks at the screening visit
  • •• Agrees to use contraception if participant is of child bearing potential
  • •• CANNOT have:
  •             o   Known hypersensitivity to any of the inactive ingredients in PTR-01
  •             o   Be pregnant or nursing
  •             o   Have received any investigational product in the past 6 months
  •             o   Anticipate receiving new regimens of antibiotics or other anti-infectives during the trial

About the clinical trial:

This study will investigate the safety, tolerability, and efficacy of an investigational treatment called PTR-01 (type VII collagen protein). PTR-01 is an investigational product given by intravenous infusion over 1 hour. 

Patients enrolled in the study are given 3 infusions of PTR-01 (or placebo), followed by 3 doses of placebo (or PTR-01). All subjects will receive both PTR-01 and placebo.

This study involves blood and urine samples and does require skin biopsies.

Study visits occur every 2 weeks and require coming to Stanford. There are a total of 6 infusions. The treatment period is 10 weeks.


Study Coordinator: Melissa Barriga 650-850-1674 mbarriga@stanford.edu

Link in clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03752905