Clinical trial NCT04599881: A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
Official title: A Phase 2 Open-Label Study of PTR-01 in Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Recessive Dystrophic Epidermolysis Bullosa
Keywords: RDEB
Lead sponsor: Phoenix Tissue Repair, Inc.
Sponsor class: INDUSTRY
First posted: 2020-10-23
Enrollment: 6
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: Open-label study
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: Wound healing
timeFrame: Up to 162 days
measure: Incidence of treatment-emergent adverse events
timeFrame: Up to 162 days
measure: Incidence of infusion-associated reactions
timeFrame: Up to 162 days
measure: Incidence of anti-drug antibodies (ADA)
timeFrame: Up to 162 days
Secondary outcomes:
measure: Delivery of PTR-01 to skin
timeFrame: Up to 162 days
measure: Formation of anchoring fibrils
timeFrame: Up to 162 days
measure: Change in wound surface area, as assessed by wound imaging
timeFrame: Up to 162 days
measure: Change in wound surface area, as assessed by Investigator Global Impression of Change (IGIC)
timeFrame: Up to 162 days
measure: Change in total body wound surface area
timeFrame: Up to 162 days
measure: Change in skin integrity, as assessed by suction blister time
timeFrame: Up to 162 days
measure: Change in skin integrity, as assessed by time to re-blistering
timeFrame: Up to 162 days
measure: Change in itch severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) itch domains
timeFrame: Up to 162 days
measure: Change in itch severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)
timeFrame: Up to 162 days
measure: Change in the impact of itch on quality of life
timeFrame: Up to 162 days
measure: Change in pain severity, as assessed by modified Patient-Reported Outcome Measurement Information System (PROMIS) pain domains
timeFrame: Up to 162 days
measure: Change in pain severity, as assessed by the Instrument for Scoring Clinical Outcomes for Research of Epidermolysis Bullosa (iscorEB)
timeFrame: Up to 162 days
measure: Change in the impact of pain on quality of life
timeFrame: Up to 162 days
measure: Change of dysphagia, as assessed using the Brief Esophageal Dysphagia Questionnaire
timeFrame: Up to 162 days
measure: Change in dysphagia, as assessed by volume of oral nutritional intake
timeFrame: Up to 162 days
measure: Stabilization of dysphagia, as assessed using the Brief Esophageal Dysphagia Scale
timeFrame: Up to 162 days
measure: Stabilization of dysphagia, as assessed by volume oral nutritional intake
timeFrame: Up to 162 days
measure: Change in corneal symptoms
timeFrame: Up to 162 days
measure: Stabilization of corneal symptoms
timeFrame: Up to 162 days
measure: Rate of change in nutritional markers (hemoglobin/hematocrit)
timeFrame: Up to 162 days
measure: Rate of change in nutritional markers (total protein/albumin)
timeFrame: Up to 162 days
measure: Rate of change in nutritional markers (iron/TIBC)
timeFrame: Up to 162 days
measure: Rate of change in nutritional markers (C-reactive protein)
timeFrame: Up to 162 days
measure: Rate of stabilization of nutritional markers (hemoglobin/hematocrit)
timeFrame: Up to 162 days
measure: Rate of stabilization of nutritional markers (total protein/albumin)
timeFrame: Up to 162 days
measure: Rate of stabilization of nutritional markers (iron/TIBC)
timeFrame: Up to 162 days
measure: Rate of stabilization of nutritional markers (C-reactive protein)
timeFrame: Up to 162 days
measure: Change in Investigator Global Impressions of Change (IGIC)
timeFrame: Up to 162 days
measure: Change in Investigator Patient Impressions of Change (PGIC)
timeFrame: Up to 162 days
measure: Change in disease activity and scarring
timeFrame: Up to 162 days
measure: Change in overall quality of life, as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire
timeFrame: Up to 162 days
measure: Change in overall health
timeFrame: Up to 162 days
measure: Change in mental health
timeFrame: Up to 162 days
measure: Change in social function
timeFrame: Up to 162 days
measure: Change in amount of wound care
timeFrame: Up to 162 days
measure: Change in time for wound care
timeFrame: Up to 162 daysUp to 162 days
measure: Change in cost of wound care
timeFrame: Up to 162 days
measure: Change in overall patient impression of quality of life
timeFrame: Up to 162 days
measure: Change in overall patient impression of disability
timeFrame: Up to 162 days
Other outcomes:
measure: Genotype/phenotype relationships
timeFrame: Up to 162 days
measure: Impact of pharmacokinetics on safety outcomes
timeFrame: Up to 162 days
measure: Impact of pharmacokinetics on efficacy outcomes
timeFrame: Up to 162 days
measure: Impact of pharmacokinetics on pharmacodynamic outcomes
timeFrame: Up to 162 days
Locations:
city: Redwood City
country: United States
facility: Stanford University
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
Study officials:
References: