Clinical trial NCT05143190: Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Recessive Dystrophic Epidermolysis Bullosa
Keywords: Dystrophic Epidermolysis Bullosa
Epidermolysis Bullosa
RDEB
EB
Lead sponsor: Phoenix Tissue Repair, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Phoenix Tissue Repair, a BridgeBio company
First posted: 2021-12-03
Enrollment: 2
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Phase 2 open-label
Primary purpose: TREATMENT

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Sustained wound healing
timeFrame: Up to 246 days
measure: Incidence of treatment-emergent adverse events
timeFrame: Up to 246 days
Secondary outcomes:
measure: Delivery of recombinant collagen 7 (PTR-01) to skin
timeFrame: Up to 246 days
measure: Formation of anchoring fibrils
timeFrame: Up to 246 days
measure: Change in wound surface area
timeFrame: Up to 246 days
measure: Change in skin integrity, as assessed by suction blister time
timeFrame: Up to 246 days
measure: Change in skin integrity, as assessed by time to re-blistering
timeFrame: Up to 246 days
measure: Change in itch severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains
timeFrame: Up to 246 days
measure: Change in the impact of itch on quality of life
timeFrame: Up to 246 days
measure: Change in pain severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) pain domains
timeFrame: Up to 246 days
measure: Change in pain severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)
timeFrame: Up to 246 days
measure: Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire
timeFrame: Up to 246 days
measure: Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Instrument
timeFrame: Up to 246 days
measure: Change in corneal symptoms
timeFrame: Up to 246 days
measure: Stabilization of corneal symptoms
timeFrame: Up to 246 days
measure: Rate of change in nutritional markers (hemoglobin)
timeFrame: Up to 246 days
measure: Rate of change in nutritional markers (hematocrit)
timeFrame: Up to 246 days
measure: Rate of change in nutritional markers (total protein/albumin)
timeFrame: Up to 246 days
measure: Rate of change in nutritional markers (Fe/TIBC)
timeFrame: Up to 246 days
measure: Rate of change in nutritional markers (C-reactive protein)
timeFrame: Up to 246 days
measure: Change in Investigator Global Impressions of Change (IGIC)
timeFrame: Up to 246 days
measure: Change in Investigator Patient Impressions of Change (PGIC)
timeFrame: Up to 246 days
measure: Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
timeFrame: Up to 246 days
measure: Change in overall health
timeFrame: Up to 246 days
measure: Change in mental health
timeFrame: Up to 246 days
measure: Change in social function
timeFrame: Up to 246 days
measure: Change in amount of wound care
timeFrame: Up to 246 days
measure: Change in time for wound care
timeFrame: Up to 246 days
measure: Change in cost of wound care
timeFrame: Up to 246 days
measure: Change in overall patient impression of quality of life
timeFrame: Up to 246 days
measure: Change in overall patient impression of disability
timeFrame: Up to 246 days
measure: Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Cmax
timeFrame: Up to 246 days
measure: Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Tmax
timeFrame: Up to 246 days
measure: Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter Area Under the Curve (AUC)
timeFrame: Up to 246 days
measure: Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of clearance of PTR-01
timeFrame: Up to 246 days
measure: Effect of anti-drug antibodies (ADA) on pharmacokinetics parameter of PTR-01 half-life
timeFrame: Up to 246 days
Other outcomes:
Locations:
city: Redwood City
country: United States
facility: Stanford University
state: California
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
Study officials:
affiliation: Phoenix Tissue Repair
name: Dan Rudin, MD
role: STUDY_DIRECTOR
References: