A Phase 2 interventional study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa, sponsored by Phoenix Tissue Repair, Inc.. Completed at 2 sites in United States. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2022-11-21.
Sponsored by Phoenix Tissue Repair, Inc. (part of BridgeBio) · Phase 2, Interventional, and Treatment
A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.
Protocol PTR-01-003 is a 4-part Phase 2, open-label study of PTR-01 in patients who satisfactorily completed study PTR-01-002 and meet current enrollment criteria.
In Part 1, patients will be monitored monthly for up to 2 months with patient-reported and Investigator assessments. In Part 2, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 3 in which patients will receive a dose of 3.0 mg/kg monthly for a total of 5 additional doses. At the end of each dosing period, efficacy assessments will be performed. During Part 4, patients will be evaluated at Months 1 and 3 after completion of dosing to assess the durability of wound healing and other efficacy parameters. Safety will be assessed continuously throughout the study.
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 20 are open to participants now.
This study's enrollment of 2 is below the median of 9 across 55 interventional studies indexed under Epidermolysis Bullosa Dystrophica.
Browse Epidermolysis Bullosa Dystrophica studies →Phoenix Tissue Repair, Inc. is the lead sponsor of 3 studies on the registry; none are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Agrees to use contraception as follows:
Exclusion Criteria:
All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg once monthly for a total of 5 doses.
Drug: PTR-01
Intravenous recombinant collagen 7
Also known as: Recombinant collagen 7 (rC7)
Sustained wound healing
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)
Time frame: Up to 246 days
Incidence of treatment-emergent adverse events
Safety and tolerability, as assessed by treatment-emergent adverse events
Time frame: Up to 246 days
Delivery of recombinant collagen 7 (PTR-01) to skin
Amount of recombinant collagen 7 (PTR-01) incorporation to skin as compared to normal human skin assessed by immunofluorescence using NC1 \& NC2 antibody staining
Time frame: Up to 246 days
Formation of anchoring fibrils
Formation of new anchoring fibrils as measured by electron microscopy
Time frame: Up to 246 days
Change in wound surface area
Wound surface area of lesions as assessed by medical photography using the Canfield RUBI 3D imaging system
Time frame: Up to 246 days
Change in skin integrity, as assessed by suction blister time
Change in skin integrity, as assessed by suction blister time
Time frame: Up to 246 days
Change in skin integrity, as assessed by time to re-blistering
Change in skin integrity, as assessed by time to re-blistering
Time frame: Up to 246 days
Change in itch severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains
Severity of itch, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains, maximum score of 5 (worst)
Time frame: Up to 246 days
Change in the impact of itch on quality of life
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)
Time frame: Up to 246 days
Change in pain severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) pain domains
Change in pain severity, as assessed by Patient-Reported Outcome Measurement Information System (PROMIS) pain domains, maximum score of 5 (worst)
Time frame: Up to 246 days
Change in pain severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)
Change in pain severity, as assessed by the Instrument for Scoring Clinical, maximum score of 234 (worst)
Time frame: Up to 246 days
Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire
Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire, maximum score is 40 (worst)
Time frame: Up to 246 days
Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument
Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument, maximum score of 40 (worst)
Time frame: Up to 246 days
Change in corneal symptoms
Change of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI), maximum score of 100 (worst)
Time frame: Up to 246 days
Stabilization of corneal symptoms
Stabilization of corneal symptoms (eye symptoms), as assessed by the Epidermolysis Bullosa Eye Disease Index (EB-EDI), maximum score of 100 (worst)
Time frame: Up to 246 days
Rate of change in nutritional markers (hemoglobin)
Change of nutritional markers, as assessed by hemoglobin
Time frame: Up to 246 days
Rate of change in nutritional markers (hematocrit)
Change of nutritional markers, as assessed by hematocrit
Time frame: Up to 246 days
Rate of change in nutritional markers (total protein/albumin)
Change of nutritional markers, as assessed by total protein/albumin
Time frame: Up to 246 days
Rate of change in nutritional markers (Fe/TIBC)
Change of nutritional markers, as assessed by total Fe/TIBC
Time frame: Up to 246 days
Rate of change in nutritional markers (C-reactive protein)
Change of nutritional markers, as assessed by total C-reactive protein
Time frame: Up to 246 days
Change in Investigator Global Impressions of Change (IGIC)
Global impressions of change, as assessed through IGIC (1-7), 7 being worst
Time frame: Up to 246 days
Change in Investigator Patient Impressions of Change (PGIC)
Global impressions of change, as assessed through PGIC (1-7), 7 being worst
Time frame: Up to 246 days
Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire maximum score is 67 (worst)
Time frame: Up to 246 days
Change in overall health
Change in overall disability, as assessed by the Health Assessment Questionnaire or Children's Health Assessment Questionnaire (HAQ/CHAQ) maximum score is 3 (worst)
Time frame: Up to 246 days
Change in mental health
Change in mental health, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) mental health domains, maximum score is 5 (worst)
Time frame: Up to 246 days
Change in social function
Change in social functioning, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) social function domains, maximum score is 4 (worst)
Time frame: Up to 246 days
Change in amount of wound care
Change in amount of wound care, as assessed by patient interviews
Time frame: Up to 246 days
Change in time for wound care
Change in time for wound care, as assessed by patient interviews
Time frame: Up to 246 days
Change in cost of wound care
Change in cost of wound care, as assessed by patient interviews
Time frame: Up to 246 days
Change in overall patient impression of quality of life
Change in overall anecdotal quality of life, as assessed by one-on-one patient interviews
Time frame: Up to 246 days
Change in overall patient impression of disability
Change in overall anecdotal disability, as assessed by one-on-one patient interviews
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Cmax
Correlate ADA with Cmax
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Tmax
Correlate ADA with Tmax
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Area Under the Curve (AUC)
Correlate ADA with Area Under the Curve (AUC)
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of clearance of PTR-01
Correlate ADA with clearance of PTR-01
Time frame: Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of PTR-01 half-life
Correlate ADA with PTR-01 half-life
Time frame: Up to 246 days
Plan to share: No
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Studies of the drug tested here: PTR-01
Epidermolysis Bullosa Dystrophica →