Phoenix Tissue Repair's ongoing Phase I/II trial is investigating 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. Cohorts 1 – 3 have completed treatment at escalating dose levels, and an additional fourth cohort to evaluate higher dosing of PTR-01 has begun enrolling patients.
The ongoing clinical trial is a randomized, saline-controlled, double-blind, repeat dose, dose-escalation, multi-center study. Patients enrolled in the fourth cohort will each be dosed over a 10-week period, with three 3.0 mg/kg doses of PTR-01 and three doses of saline control. The primary objective of the trial is to evaluate the safety and tolerability of PTR-01 in adults with RDEB. Additional endpoints include assessment of biologic activity through skin biopsy evaluation of collagen 7 deposition and clinical assessments including wound healing, pain, itch and quality of life.
After completion of this trial, the company is planning to follow it up with a six-month open-label clinical trial to inform the design of a pivotal clinical trial.
- 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 126.96.36.199 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.