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 email@example.com
Link in clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03752905