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

Phoenix Tissue Repair, Inc.2 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Interventions

PTR-01DRUG

Intravenous recombinant collagen 7

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Willing to provide informed consent form, or if 12 to \<18 years of age, legal guardian has provided informed consent form and the minor has signed an assent form acknowledging that they understand and agree to study procedures. 2. Has satisfactorily completed participation in PTR-01-002. 3. 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. * 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. 4. Be willing and able to comply with this protocol. Exclusion Criteria: 1. Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01. 2. Has previously had an anaphylactic reaction to PTR-01. 3. Is pregnant or nursing. 4. Has received in the last six months any investigational gene therapy product or in the last three months any non-gene therapy investigational products (other than PTR-01). 5. Is anticipated to receive new regimens of antibiotics or other anti-infectives during the trial. 6. Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the

Locations (2)

Stanford University

Redwood City, California, United States

Columbia University Irving Medical Center

New York, New York, United States

Outcomes

Primary Outcomes

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

Secondary Outcomes

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

Time frame: Up to 246 days

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

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

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