Phoenix Tissue Repair is currently recruiting for its ongoing Phase I/II 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.
- Be at least 18 years of age.
- Has signed the current approved informed consent form.
- Has a diagnosis of RDEB based on genetic analysis showing two confirmed RDEB type VII collagen mutations consistent with a recessive inheritance pattern.
- Has the presence of some but deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.
- Has at least 1 unhealed wound ≥ 20 cm2 for at least 6 weeks at the Screening Visit.
- Agrees to use contraception as follows:
- For women of childbearing potential (WOCBP) agrees to use highly effective contraceptive (including abstinence) methods from Screening, through the study, and for at least 10 weeks after the last dose of study drug. Non-childbearing potential is defined as a female who meets either of the following criteria: age ≥50 years and no menses for at least 1 year or documented hysterectomy, bilateral tubal ligation, or bilateral oophorectomy (see Section 220.127.116.11 for details on the definition of non-childbearing potential).
- For males, agrees to use a condom with any WOCBP sexual partner from Day 1 of study treatment, through the study, and at least 10 weeks after the last dose of study drug.
- Be willing and able to comply with this protocol.
- Please click the link below for exclusion criteria.