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

A Phase 2b Open-Label Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01 in Study PTR-01-002

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.

Study Overview

• Status: Completed
• Conditions: Recessive Dystrophic Epidermolysis Bullosa
• Intervention / Treatment: Drug: PTR-01

Detailed Description

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
• Enrollment (Actual): 2
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Redwood City, California, United States, 94063
      • Stanford University
  • New York
    • New York, New York, United States, 10032
      • Columbia University Irving Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PTR-01; 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; Other Names: Recombinant collagen 7 (rC7)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	Up to 246 days
Incidence of treatment-emergent adverse events	Safety and tolerability, as assessed by treatment-emergent adverse events	Up to 246 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Up to 246 days
Formation of anchoring fibrils	Formation of new anchoring fibrils as measured by electron microscopy	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	Up to 246 days
Change in skin integrity, as assessed by suction blister time	Change in skin integrity, as assessed by suction blister time	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	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)	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)	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)	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)	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)	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)	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)	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)	Up to 246 days
Rate of change in nutritional markers (hemoglobin)	Change of nutritional markers, as assessed by hemoglobin	Up to 246 days
Rate of change in nutritional markers (hematocrit)	Change of nutritional markers, as assessed by hematocrit	Up to 246 days
Rate of change in nutritional markers (total protein/albumin)	Change of nutritional markers, as assessed by total protein/albumin	Up to 246 days
Rate of change in nutritional markers (Fe/TIBC)	Change of nutritional markers, as assessed by total Fe/TIBC	Up to 246 days
Rate of change in nutritional markers (C-reactive protein)	Change of nutritional markers, as assessed by total C-reactive protein	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	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	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)	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)	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)	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)	Up to 246 days
Change in amount of wound care	Change in amount of wound care, as assessed by patient interviews	Up to 246 days
Change in time for wound care	Change in time for wound care, as assessed by patient interviews	Up to 246 days
Change in cost of wound care	Change in cost of wound care, as assessed by patient interviews	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	Up to 246 days
Change in overall patient impression of disability	Change in overall anecdotal disability, as assessed by one-on-one patient interviews	Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Cmax	Correlate ADA with Cmax	Up to 246 days
Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Tmax	Correlate ADA with Tmax	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)	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	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	Up to 246 days

Collaborators and Investigators

• Sponsor: Phoenix Tissue Repair, Inc.
• Collaborators: Phoenix Tissue Repair, a BridgeBio company
• Investigators: Study Director: Dan Rudin, MD, Phoenix Tissue Repair

Study record dates

Study Major Dates

• Study Start (Actual): December 2, 2021
• Primary Completion (Actual): June 20, 2022
• Study Completion (Actual): August 9, 2022

Study Registration Dates

• First Submitted: November 9, 2021
• First Submitted That Met QC Criteria: November 19, 2021
• First Posted (Actual): December 3, 2021

Study Record Updates

• Last Update Posted (Actual): November 21, 2022
• Last Update Submitted That Met QC Criteria: November 17, 2022
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Keywords: Dystrophic Epidermolysis Bullosa; RDEB; EB; Epidermolysis Bullosa
• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Collagen Diseases; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica
• Other Study ID Numbers: PTR-01-003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No