Phoenix Tissue Repair’s Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (PTR-01-001)

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December 2019 Update:
Phoenix Tissue Repair is looking to recruit more people into its Phase 1/2 Clinical Trial for PTR-01, a collagen VII replacement therapy for those with RDEB. If you’d like to learn more or are interested in participating, please visit:

About the Study:
Phoenix Tissue Repair is currently recruiting for the second cohort in its ongoing Phase ½ trial of PTR-01 (PTR-01-001 trial), a protein replacement therapy which uses a recombinant collagen type VII (rC7) for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). PTR-01 is designed to be systemically available through intravenous delivery.

The PTR-01-001 trial is a randomized saline-controlled, single-blind, multiple ascending dose, dose-escalation and multi-center study. Patients will be enrolled into one of three cohorts. Cohorts 1, 2 and 3 will consist of 2, 4 and 8 patients respectively. During the treatment period, a total of 3 doses of PTR-01 and 3 doses of saline control will be administered to all patients for a total of 6 doses in a cross-over design over a 10-week period. The primary objective of the trial is to evaluate the safety and tolerability of PTR-01 in RDEB patients. Additionally, the trial will assess biologic activity through skin biopsy evaluation of C7 and the presence of anchoring fibrils to potentially provide proof of concept. Wound healing and clinically meaningful patient reported outcomes will also be evaluated.

For more information about the study please click here.