Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)

Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)

Safety and tolerability, as assessed by treatment-emergent adverse events

Amount of recombinant collagen 7 (PTR-01) incorporation to skin as compared to normal human skin assessed by immunofluorescence using NC1 & NC2 antibody staining

Formation of new anchoring fibrils as measured by electron microscopy

Wound surface area of lesions as assessed by medical photography using the Canfield RUBI 3D imaging system

Change in skin integrity, as assessed by suction blister time

Change in skin integrity, as assessed by time to re-blistering

Severity of itch, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains, maximum score of 5 (worst)

Change in the impact of itch on quality of life, as assessed by the Pruritus-Specific Quality of Life Instrument (ItchyQoL), maximum score of 110 (worst)

Change in pain severity, as assessed by Patient-Reported Outcome Measurement Information System (PROMIS) pain domains, maximum score of 5 (worst)

Change in pain severity, as assessed by the Instrument for Scoring Clinical, maximum score of 234 (worst)

Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire, maximum score is 40 (worst)

Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument, maximum score of 40 (worst)

Change of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI), maximum score of 100 (worst)

Stabilization of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI), maximum score of 100 (worst)

Change of nutritional markers, as assessed by hemoglobin

Change of nutritional markers, as assessed by hematocrit

Change of nutritional markers, as assessed by total protein/albumin

Change of nutritional markers, as assessed by total Fe/TIBC

Change of nutritional markers, as assessed by total C-reactive protein

Global impressions of change, as assessed through IGIC (1-7), 7 being worst

Global impressions of change, as assessed through PGIC (1-7), 7 being worst

Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire maximum score is 67 (worst)

Change in overall disability, as assessed by the Health Assessment Questionnaire or Children's Health Assessment Questionnaire (HAQ/CHAQ) maximum score is 3 (worst)

Change in mental health, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) mental health domains, maximum score is 5 (worst)

Change in social functioning, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) social function domains, maximum score is 4 (worst)

Change in amount of wound care, as assessed by patient interviews

Change in time for wound care, as assessed by patient interviews

Change in cost of wound care, as assessed by patient interviews

Change in overall anecdotal quality of life, as assessed by one-on-one patient interviews

Change in overall anecdotal disability, as assessed by one-on-one patient interviews

Correlate ADA with Cmax

Correlate ADA with Tmax

Correlate ADA with Area Under the Curve (AUC)

Correlate ADA with clearance of PTR-01

Correlate ADA with PTR-01 half-life