A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (PTR-01-001)

March 8, 2021 updated by: Phoenix Tissue Repair, Inc.

A Phase 1/2 Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Protocol PTR-01-001 is a Phase 1/2 study of PTR-01.

The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an 8-week Follow-up Period.

Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio. Patients in Group 1 will receive three doses of active drug followed by 3 doses of saline control. Patients in Group 2 will receive three doses of saline control followed by 3 doses of active drug.

Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1 patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Protocol PTR-01-001 is a saline-controlled, single and repeat dose, dose-escalation, crossover study designed to determine the safety, tolerability, tissue kinetics, pharmacodynamics and preliminary efficacy of PTR 01.

The study is divided into three periods: an up to 4-week Screening Period, a 10-week Treatment Period and an 8-week Follow-up Period. During the Screening Period and Follow-up Period there will be no study drug treatment.

During the Treatment Period a total of 3 doses of PTR-01 and 3 doses of saline control will be administered to patients for a total of 6 doses over a 10-week period in three cohorts dosed at 0.1, 0.3, 1.0 and 3.0 mg/kg (active drug). Twelve patients with a diagnosis of RDEB and a history of at least one chronic wound will be enrolled. Those patients who do not have documentation of genetic analysis and IF staining will have blood for genetic analysis and a biopsy for IF staining prior to enrollment (both required).

Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio. Patients will receive doses 2 weeks apart. Patients in Group 1 will receive three doses of active drug followed by 3 doses of saline control. Patients in Group 2 will receive three doses of saline control followed by 3 doses of active drug. This cross-over design will yield a total of 14 patients all of whom will receive active drug and saline control.

Prior to randomization, patients will complete a Screening Period to assess the extent and impact of skin disease involvement and the chronicity of at least one wound. Only patients who meet all of the eligibility criteria will be randomized for treatment.

Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1 patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses. After the last patient in Cohort 1 has received their third dose and safety labs for all patients have been reviewed by the Data Safety Monitoring Board (DSMB), the next cohort may be enrolled. This same schedule and safety review process will be followed for all subsequent dosing cohorts, with Cohort 2, Cohort 3 and Cohort 4 receiving 0.3, 1.0 and 3.0 mg/kg respectively.

Efficacy assessments will be performed prior to first dose of therapy (at the end of the Screening Period), after the last dose of study drug in Period 1, after the last dose of study drug in Period 2 of the Treatment Period and 2 weeks (Day 85) after the last dose of study drug (at the end of the Follow-up Period).

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
12

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Redwood City, California, United States, 94063
        • Stanford University
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19107
        • Thomas Jefferson Univeristiy

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Be at least 16 years of age.
  2. Has signed the current approved informed consent form.
  3. Has a diagnosis of RDEB based on genetic analysis and consistent with a recessive inheritance pattern.
  4. Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.
  5. Has at least 1 unhealed wound 10-200 cm2 for at least 6 weeks at the Screening Visit.

  6. 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 (see Section 7.4.1.2 for details on the definition of non-childbearing potential).
    • 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.
  7. 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. Is pregnant or nursing.
  3. Has received in the last six months any investigational gene therapy product or in the last three months any non-gene therapy investigational products.
  4. Is anticipated to receive new regimens of antibiotics or other anti-infectives during the trial.
  5. 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 successful completion of the clinical study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: PTR-01 0.1 mg/kg
Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart.
Drug: PTR-01
Recombinant human collagen 7 (rC7)
Other Names:
  • Recombinant human collagen 7 (rC7)
Experimental: PTR-01 0.3 mg/kg
Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart.
Drug: PTR-01
Recombinant human collagen 7 (rC7)
Other Names:
  • Recombinant human collagen 7 (rC7)
Experimental: PTR-01 1.0 mg/kg
Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart.
Drug: PTR-01
Recombinant human collagen 7 (rC7)
Other Names:
  • Recombinant human collagen 7 (rC7)
Placebo Comparator: Normal Saline
Saline control to mimic PTR-01.
Drug: Normal saline
Saline control
Experimental: PTR-01 3.0 mg/kg
Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart.
Drug: PTR-01
Recombinant human collagen 7 (rC7)
Other Names:
  • Recombinant human collagen 7 (rC7)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incidence of treatment-emergent adverse events
Time Frame: Up to Day 127
The primary endpoint of this study is safety and tolerability, as assessed by treatment-emergent adverse events, infusion-associated reactions (IAR) and immunogenicity
Up to Day 127

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
To measure the peak serum concentration (Cmax) of PTR-01
Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
Pharmacokinetic parameter estimates of Cmax
Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the time to peak concentration (Tmax) of PTR-01
Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
Pharmacokinetic parameter estimates of Tmax
Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the area under the curve (AUC) of PTR-01
Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
Pharmacokinetic parameter estimates of AUC
Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the clearance of PTR-01
Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
Pharmacokinetic parameter estimates of clearance
Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
To measure the half-life (t1/2) of PTR-01
Time Frame: Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
Pharmacokinetic parameter estimates of t1/2
Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose
Change from Baseline in rC7
Time Frame: Screening and Day 127
Change in rC7 on skin biopsy by immunofluorescence (IF)
Screening and Day 127
Change from Baseline in anchoring fibrils
Time Frame: Screening and Day 127
Change in anchoring fibrils on skin biopsy by electron microscopy (EM)
Screening and Day 127
Duration of rC7 residence in tissue
Time Frame: Screening and Day 127
Duration of rC7 residence in tissue by skin biopsy
Screening and Day 127

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change from Baseline in suction blister time
Time Frame: Baseline and Day 127
Change from Baseline in suction blister time (as compared to placebo and historical controls)
Baseline and Day 127
Change from Baseline in target wound size
Time Frame: Baseline and Day 127
Change from Baseline in target wound size (percent healing from Baseline)
Baseline and Day 127
Change from Baseline in healing of up to 5 chronic wounds
Time Frame: Baseline and Day 127
Change in healing of up to 5 wounds that chronically heal and reopen
Baseline and Day 127
Change from Baseline in wound surface area
Time Frame: Screening and Day 127
Change from Baseline in wound surface area
Screening and Day 127
Change from Baseline in patient reported outcomes as assessed by the Leuven Itch Scale (LIS)
Time Frame: Baseline and Day 127
Change from Baseline in patient reported outcomes
Baseline and Day 127
Change from Baseline in patient reported outcomes as assessed by the pruritus-specific quality-of-life instrument ("ItchyQoL")
Time Frame: Baseline and Day 127
Change from Baseline in patient reported outcomes
Baseline and Day 127
Change from Baseline in patient reported outcomes as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) Questionnaire
Time Frame: Baseline and Day 127
Change from Baseline in patient reported outcomes
Baseline and Day 127
Change from Baseline in patient reported outcomes as assessed by the full Health Assessment Questionnaire (HAQ)
Time Frame: Baseline and Day 127
Change from Baseline in patient reported outcomes
Baseline and Day 127
Change from Baseline in the Investigator Global Assessment
Time Frame: Screening and Day 127
Change from Baseline in the Investigator Global Assessment (IGA)
Screening and Day 127
Change from Baseline in the biochemical marker albumin
Time Frame: Screening and Day 127
Change from Baseline in biochemical markers of disease (albumin)
Screening and Day 127
Change from Baseline in the biochemical marker iron
Time Frame: Screening and Day 127
Change from Baseline in biochemical markers of disease (iron)
Screening and Day 127
Change from Baseline in the biochemical marker total iron binding capacity
Time Frame: Screening and Day 127
Change from Baseline in biochemical markers of disease (total iron binding capacity)
Screening and Day 127
Change from Baseline in the biochemical marker hemoglobin
Time Frame: Screening and Day 127
Change from Baseline in biochemical markers of disease (hemoglobin)
Screening and Day 127
Change from Baseline in the biochemical marker hematocrit
Time Frame: Screening and Day 127
Change from Baseline in biochemical markers of disease (hematocrit)
Screening and Day 127
Change from Baseline in the biochemical marker total protein
Time Frame: Screening and Day 127
Change from Baseline in biochemical markers of disease (total protein)
Screening and Day 127

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Phoenix Tissue Repair, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Theresa Podrebarac, MD, Phoenix Tissue Repair

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 9, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 30, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 30, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 19, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 21, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 26, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 9, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 8, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • PTR-01-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

IPD Plan Description

There are currently no plans to share individual participant data with other researchers.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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