A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa

Phoenix Tissue Repair, Inc.6 enrolled

Overview

Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.

Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001. In Part 1, patients will receive a dose of 3.0 mg/kg every week for a total of 4 doses. This will be followed by Part 2 in which patients will receive a dose of 3.0 mg/kg every other week for a total of 7 doses. In Part 3, patients will be followed for 12 weeks. No investigational therapy will be administered during this time. At the end of each dosing period, an efficacy assessment will be performed. Safety will be assessed continuously throughout the study. Following the end of Part 3, patients may be eligible for a potential long-term extension to further refine the dosing regimen, depending upon study drug availability.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Recessive Dystrophic Epidermolysis Bullosa

Interventions

PTR-01DRUG

IV recombinant collagen 7 at 3 mg/kg given weekly for 4 doses, followed by bi-weekly for 7 doses

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: Patients must meet all of the following criteria to be eligible for study participation in the three month run in period of the study: 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 a diagnosis of RDEB based on genetic analysis and consistent with a recessive inheritance pattern. 3. Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF. 4. 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. 5. Be willing and able to comply with this protocol. Exclusion Criteria: Patients with any of the following will be excluded from participation in the study: 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. 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 patient, or may preclude the patient's

Locations (2)

Stanford University

Redwood City, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Outcomes

Primary Outcomes

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 162 days

Incidence of treatment-emergent adverse events

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

Time frame: Up to 162 days

Incidence of infusion-associated reactions

Safety and tolerability, as assessed by infusion-associated reactions (IAR)

Time frame: Up to 162 days

Incidence of anti-drug antibodies (ADA)

Safety and tolerability, as assessed by immunogenicity through anti-drug antibody (ADA) testing

Time frame: Up to 162 days

Secondary Outcomes

Delivery of PTR-01 to skin

PTR-01 incorporation by immunofluorescence using NC1 \& NC2 staining, by dose frequency period

Time frame: Up to 162 days

Formation of anchoring fibrils

Formation of new anchoring fibrils as measured by electron microscopy

Time frame: Up to 162 days

Change in wound surface area, as assessed by wound imaging

Wound area of target lesions, as assessed by wound imaging

Time frame: Up to 162 days

Change in wound surface area, as assessed by Investigator Global Impression of Change (IGIC)

Data from ClinicalTrials.gov

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Wound area of target lesions, as assessed by IGIC

Time frame: Up to 162 days

Change in total body wound surface area

Change in total body wound surface area, using Rule of Nines

Time frame: Up to 162 days

Change in skin integrity, as assessed by suction blister time

Time frame: Up to 162 days

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

Time frame: Up to 162 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

Time frame: Up to 162 days

Change in itch severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)

Severity of itch, as assessed by Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB), maximum score of 234 (worst)

Time frame: Up to 162 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 162 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

Time frame: Up to 162 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 162 days

Change in the impact of pain on quality of life

Change in the impact of pain on quality of life, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB) instrument, maximum score of 234 (worst)

Time frame: Up to 162 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 162 days

Change in dysphagia, as assessed by volume of oral nutritional intake

Change of dysphagia, as assessed by volume of oral nutritional intake, using patient interview and diary, maximum score is 40 (worst)

Time frame: Up to 162 days

Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Scale

Time frame: Up to 162 days

Stabilization of dysphagia, as assessed by volume oral nutritional intake

Stabilization of dysphagia, as assessed by volume oral nutritional intake, using patient interview and diary

Time frame: Up to 162 days

Change in corneal symptoms

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

Time frame: Up to 162 days

Stabilization of corneal symptoms

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

Time frame: Up to 162 days

Rate of change in nutritional markers (hemoglobin/hematocrit)

Change of nutritional markers, as assessed by hemoglobin/hematocrit

Time frame: Up to 162 days

Rate of change in nutritional markers (total protein/albumin)

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

Time frame: Up to 162 days

Rate of change in nutritional markers (iron/TIBC)

Change of nutritional markers, as assessed by iron/TIBC

Time frame: Up to 162 days

Rate of change in nutritional markers (C-reactive protein)

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

Time frame: Up to 162 days

Rate of stabilization of nutritional markers (hemoglobin/hematocrit)

Stabilization of nutritional markers, as assessed by hemoglobin/hematocrit

Time frame: Up to 162 days

Rate of stabilization of nutritional markers (total protein/albumin)

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

Time frame: Up to 162 days

Rate of stabilization of nutritional markers (iron/TIBC)

Stabilization of nutritional markers, as assessed by iron/TIBC

Time frame: Up to 162 days

Rate of stabilization of nutritional markers (C-reactive protein)

Stabilization of nutritional markers, as assessed by C-reactive protein

Time frame: Up to 162 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 162 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 162 days

Change in disease activity and scarring

Change in disease activity and scarring, as assessed by the Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI)

Time frame: Up to 162 days

Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire

Time frame: Up to 162 days

Change in overall health

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

Time frame: Up to 162 days

Change in mental health

Change in mental health and social functioning, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) mental health domains

Time frame: Up to 162 days

Change in social function

Change in mental health and social functioning, as assessed by the Patient-Reported Outcomes Measurement Information System (PROMIS) social function domains

Time frame: Up to 162 days

Change in amount of wound care

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

Time frame: Up to 162 days

Change in time for wound care

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

Time frame: Up to 162 daysUp to 162 days

Change in cost of wound care

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

Time frame: Up to 162 days

Change in overall patient impression of quality of life

Change in overall quality of life, as assessed by patient interviews

Time frame: Up to 162 days

Change in overall patient impression of disability

Change in overall disability, as assessed by patient interviews

Time frame: Up to 162 days