<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03752905</url>
  </required_header>
  <id_info>
    <org_study_id>PTR-01-001</org_study_id>
    <nct_id>NCT03752905</nct_id>
  </id_info>
  <brief_title>A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)</brief_title>
  <acronym>PTR-01-001</acronym>
  <official_title>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)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Phoenix Tissue Repair, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Phoenix Tissue Repair, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Protocol PTR-01-001 is a Phase 1/2 study of PTR-01.&#xD;
&#xD;
      The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an&#xD;
      8-week Follow-up Period.&#xD;
&#xD;
      Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will&#xD;
      consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio.&#xD;
      Patients in Group 1 will receive three doses of active drug followed by 3 doses of saline&#xD;
      control. Patients in Group 2 will receive three doses of saline control followed by 3 doses&#xD;
      of active drug.&#xD;
&#xD;
      Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at&#xD;
      a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1&#xD;
      patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of&#xD;
      active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Protocol PTR-01-001 is a saline-controlled, single and repeat dose, dose-escalation,&#xD;
      crossover study designed to determine the safety, tolerability, tissue kinetics,&#xD;
      pharmacodynamics and preliminary efficacy of PTR 01.&#xD;
&#xD;
      The study is divided into three periods: an up to 4-week Screening Period, a 10-week&#xD;
      Treatment Period and an 8-week Follow-up Period. During the Screening Period and Follow-up&#xD;
      Period there will be no study drug treatment.&#xD;
&#xD;
      During the Treatment Period a total of 3 doses of PTR-01 and 3 doses of saline control will&#xD;
      be administered to patients for a total of 6 doses over a 10-week period in three cohorts&#xD;
      dosed at 0.1, 0.3, 1.0 and 3.0 mg/kg (active drug). Twelve patients with a diagnosis of RDEB&#xD;
      and a history of at least one chronic wound will be enrolled. Those patients who do not have&#xD;
      documentation of genetic analysis and IF staining will have blood for genetic analysis and a&#xD;
      biopsy for IF staining prior to enrollment (both required).&#xD;
&#xD;
      Cohorts 1, 2, 3 and 4 will consist of 2, 4, 3 and 3 patients respectively. Each cohort will&#xD;
      consist of patients divided into two groups (Group 1 and Group 2) randomized in a 1:1 ratio.&#xD;
      Patients will receive doses 2 weeks apart. Patients in Group 1 will receive three doses of&#xD;
      active drug followed by 3 doses of saline control. Patients in Group 2 will receive three&#xD;
      doses of saline control followed by 3 doses of active drug. This cross-over design will yield&#xD;
      a total of 14 patients all of whom will receive active drug and saline control.&#xD;
&#xD;
      Prior to randomization, patients will complete a Screening Period to assess the extent and&#xD;
      impact of skin disease involvement and the chronicity of at least one wound. Only patients&#xD;
      who meet all of the eligibility criteria will be randomized for treatment.&#xD;
&#xD;
      Cohort 1 patients randomized to Group 1 will receive 3 doses of active treatment (PTR-01) at&#xD;
      a dose of 0.1 mg/kg followed by 3 doses of saline control for a total of 6 doses. Cohort 1&#xD;
      patients randomized to Group 2 will receive 3 doses of saline control followed by 3 doses of&#xD;
      active treatment (PTR-01) at a dose of 0.1 mg/kg for a total of 6 doses. After the last&#xD;
      patient in Cohort 1 has received their third dose and safety labs for all patients have been&#xD;
      reviewed by the Data Safety Monitoring Board (DSMB), the next cohort may be enrolled. This&#xD;
      same schedule and safety review process will be followed for all subsequent dosing cohorts,&#xD;
      with Cohort 2, Cohort 3 and Cohort 4 receiving 0.3, 1.0 and 3.0 mg/kg respectively.&#xD;
&#xD;
      Efficacy assessments will be performed prior to first dose of therapy (at the end of the&#xD;
      Screening Period), after the last dose of study drug in Period 1, after the last dose of&#xD;
      study drug in Period 2 of the Treatment Period and 2 weeks (Day 85) after the last dose of&#xD;
      study drug (at the end of the Follow-up Period).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">January 9, 2019</start_date>
  <completion_date type="Actual">November 30, 2020</completion_date>
  <primary_completion_date type="Actual">November 30, 2020</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Crossover Assignment</intervention_model>
    <intervention_model_description>Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
    <masking_description>Single-blind</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of treatment-emergent adverse events</measure>
    <time_frame>Up to Day 127</time_frame>
    <description>The primary endpoint of this study is safety and tolerability, as assessed by treatment-emergent adverse events, infusion-associated reactions (IAR) and immunogenicity</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>To measure the peak serum concentration (Cmax) of PTR-01</measure>
    <time_frame>Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose</time_frame>
    <description>Pharmacokinetic parameter estimates of Cmax</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To measure the time to peak concentration (Tmax) of PTR-01</measure>
    <time_frame>Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose</time_frame>
    <description>Pharmacokinetic parameter estimates of Tmax</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To measure the area under the curve (AUC) of PTR-01</measure>
    <time_frame>Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose</time_frame>
    <description>Pharmacokinetic parameter estimates of AUC</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To measure the clearance of PTR-01</measure>
    <time_frame>Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose</time_frame>
    <description>Pharmacokinetic parameter estimates of clearance</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To measure the half-life (t1/2) of PTR-01</measure>
    <time_frame>Pre-dose, 15 minutes, 60 minutes, 2 hours, 4 hours, 8 hours and 24 hours post-dose</time_frame>
    <description>Pharmacokinetic parameter estimates of t1/2</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in rC7</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change in rC7 on skin biopsy by immunofluorescence (IF)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from Baseline in anchoring fibrils</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change in anchoring fibrils on skin biopsy by electron microscopy (EM)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Duration of rC7 residence in tissue</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Duration of rC7 residence in tissue by skin biopsy</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Change from Baseline in suction blister time</measure>
    <time_frame>Baseline and Day 127</time_frame>
    <description>Change from Baseline in suction blister time (as compared to placebo and historical controls)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in target wound size</measure>
    <time_frame>Baseline and Day 127</time_frame>
    <description>Change from Baseline in target wound size (percent healing from Baseline)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in healing of up to 5 chronic wounds</measure>
    <time_frame>Baseline and Day 127</time_frame>
    <description>Change in healing of up to 5 wounds that chronically heal and reopen</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in wound surface area</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in wound surface area</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in patient reported outcomes as assessed by the Leuven Itch Scale (LIS)</measure>
    <time_frame>Baseline and Day 127</time_frame>
    <description>Change from Baseline in patient reported outcomes</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in patient reported outcomes as assessed by the pruritus-specific quality-of-life instrument (&quot;ItchyQoL&quot;)</measure>
    <time_frame>Baseline and Day 127</time_frame>
    <description>Change from Baseline in patient reported outcomes</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in patient reported outcomes as assessed by the Quality of Life in Epidermolysis Bullosa (QOLEB) Questionnaire</measure>
    <time_frame>Baseline and Day 127</time_frame>
    <description>Change from Baseline in patient reported outcomes</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in patient reported outcomes as assessed by the full Health Assessment Questionnaire (HAQ)</measure>
    <time_frame>Baseline and Day 127</time_frame>
    <description>Change from Baseline in patient reported outcomes</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in the Investigator Global Assessment</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in the Investigator Global Assessment (IGA)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in the biochemical marker albumin</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in biochemical markers of disease (albumin)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in the biochemical marker iron</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in biochemical markers of disease (iron)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in the biochemical marker total iron binding capacity</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in biochemical markers of disease (total iron binding capacity)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in the biochemical marker hemoglobin</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in biochemical markers of disease (hemoglobin)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in the biochemical marker hematocrit</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in biochemical markers of disease (hematocrit)</description>
  </other_outcome>
  <other_outcome>
    <measure>Change from Baseline in the biochemical marker total protein</measure>
    <time_frame>Screening and Day 127</time_frame>
    <description>Change from Baseline in biochemical markers of disease (total protein)</description>
  </other_outcome>
  <number_of_arms>5</number_of_arms>
  <enrollment type="Actual">12</enrollment>
  <condition>Recessive Dystrophic Epidermolysis Bullosa</condition>
  <arm_group>
    <arm_group_label>PTR-01 0.1 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PTR-01 0.3 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PTR-01 1.0 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Normal Saline</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Saline control to mimic PTR-01.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PTR-01 3.0 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>PTR-01</intervention_name>
    <description>Recombinant human collagen 7 (rC7)</description>
    <arm_group_label>PTR-01 0.1 mg/kg</arm_group_label>
    <arm_group_label>PTR-01 0.3 mg/kg</arm_group_label>
    <arm_group_label>PTR-01 1.0 mg/kg</arm_group_label>
    <arm_group_label>PTR-01 3.0 mg/kg</arm_group_label>
    <other_name>Recombinant human collagen 7 (rC7)</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Normal saline</intervention_name>
    <description>Saline control</description>
    <arm_group_label>Normal Saline</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Be at least 16 years of age.&#xD;
&#xD;
          2. Has signed the current approved informed consent form.&#xD;
&#xD;
          3. Has a diagnosis of RDEB based on genetic analysis and consistent with a recessive&#xD;
             inheritance pattern.&#xD;
&#xD;
          4. Has deficient C7 staining at the dermal-epidermal junction (DEJ) by IF.&#xD;
&#xD;
          5. Has at least 1 unhealed wound 10-200 cm2 for at least 6 weeks at the Screening Visit.&#xD;
&#xD;
          6. Agrees to use contraception as follows:&#xD;
&#xD;
               -  For women of childbearing potential (WOCBP) agrees to use highly effective&#xD;
                  contraceptive (including abstinence) methods from Screening, through the study,&#xD;
                  and for at least 10 weeks after the last dose of study drug. Non-childbearing&#xD;
                  potential is defined as a female who meets either of the following criteria: age&#xD;
                  ≥50 years and no menses for at least 1 year or documented hysterectomy, bilateral&#xD;
                  tubal ligation, or bilateral oophorectomy (see Section 7.4.1.2 for details on the&#xD;
                  definition of non-childbearing potential).&#xD;
&#xD;
               -  For males, agrees to use a condom with any WOCBP sexual partner from Day 1 of&#xD;
                  study treatment, through the study, and at least 10 weeks after the last dose of&#xD;
                  study drug.&#xD;
&#xD;
          7. Be willing and able to comply with this protocol.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Has known systemic hypersensitivity to any of the inactive ingredients in PTR-01.&#xD;
&#xD;
          2. Is pregnant or nursing.&#xD;
&#xD;
          3. Has received in the last six months any investigational gene therapy product or in the&#xD;
             last three months any non-gene therapy investigational products.&#xD;
&#xD;
          4. Is anticipated to receive new regimens of antibiotics or other anti-infectives during&#xD;
             the trial.&#xD;
&#xD;
          5. Has any other medical or personal condition that, in the opinion of the Investigator,&#xD;
             may potentially compromise the safety or compliance of the patient, or may preclude&#xD;
             the patient's successful completion of the clinical study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>16 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Theresa Podrebarac, MD</last_name>
    <role>Study Director</role>
    <affiliation>Phoenix Tissue Repair</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Stanford University</name>
      <address>
        <city>Redwood City</city>
        <state>California</state>
        <zip>94063</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Thomas Jefferson Univeristiy</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19107</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://phoenixtissuerepair.com</url>
    <description>Sponsor website</description>
  </link>
  <verification_date>March 2021</verification_date>
  <study_first_submitted>November 19, 2018</study_first_submitted>
  <study_first_submitted_qc>November 21, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 26, 2018</study_first_posted>
  <last_update_submitted>March 8, 2021</last_update_submitted>
  <last_update_submitted_qc>March 8, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">March 9, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>RDEB</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Epidermolysis Bullosa</mesh_term>
    <mesh_term>Epidermolysis Bullosa Dystrophica</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
    <ipd_description>There are currently no plans to share individual participant data with other researchers.</ipd_description>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03752905
org study id: PTR-01-001
nct id: NCT03752905
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
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.
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).
allocation: Randomized
intervention model: Crossover Assignment
intervention model description: Randomized, Saline-Controlled, Single-Blind, Multiple Ascending Dose, Dose-Escalation, Multi-Center
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
masking description: Single-blind
measure: Incidence of treatment-emergent adverse events
time frame: Up to Day 127
description: The primary endpoint of this study is safety and tolerability, as assessed by treatment-emergent adverse events, infusion-associated reactions (IAR) and immunogenicity
measure: 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
description: Pharmacokinetic parameter estimates of Cmax
measure: 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
description: Pharmacokinetic parameter estimates of Tmax
measure: 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
description: Pharmacokinetic parameter estimates of AUC
measure: 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
description: Pharmacokinetic parameter estimates of clearance
measure: 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
description: Pharmacokinetic parameter estimates of t1/2
measure: Change from Baseline in rC7
time frame: Screening and Day 127
description: Change in rC7 on skin biopsy by immunofluorescence (IF)
measure: Change from Baseline in anchoring fibrils
time frame: Screening and Day 127
description: Change in anchoring fibrils on skin biopsy by electron microscopy (EM)
measure: Duration of rC7 residence in tissue
time frame: Screening and Day 127
description: Duration of rC7 residence in tissue by skin biopsy
measure: Change from Baseline in suction blister time
time frame: Baseline and Day 127
description: Change from Baseline in suction blister time (as compared to placebo and historical controls)
measure: Change from Baseline in target wound size
time frame: Baseline and Day 127
description: Change from Baseline in target wound size (percent healing from Baseline)
measure: Change from Baseline in healing of up to 5 chronic wounds
time frame: Baseline and Day 127
description: Change in healing of up to 5 wounds that chronically heal and reopen
measure: Change from Baseline in wound surface area
time frame: Screening and Day 127
description: Change from Baseline in wound surface area
measure: Change from Baseline in patient reported outcomes as assessed by the Leuven Itch Scale (LIS)
time frame: Baseline and Day 127
description: Change from Baseline in patient reported outcomes
measure: Change from Baseline in patient reported outcomes as assessed by the pruritus-specific quality-of-life instrument ("ItchyQoL")
time frame: Baseline and Day 127
description: Change from Baseline in patient reported outcomes
measure: 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
description: Change from Baseline in patient reported outcomes
measure: Change from Baseline in patient reported outcomes as assessed by the full Health Assessment Questionnaire (HAQ)
time frame: Baseline and Day 127
description: Change from Baseline in patient reported outcomes
measure: Change from Baseline in the Investigator Global Assessment
time frame: Screening and Day 127
description: Change from Baseline in the Investigator Global Assessment (IGA)
measure: Change from Baseline in the biochemical marker albumin
time frame: Screening and Day 127
description: Change from Baseline in biochemical markers of disease (albumin)
measure: Change from Baseline in the biochemical marker iron
time frame: Screening and Day 127
description: Change from Baseline in biochemical markers of disease (iron)
measure: Change from Baseline in the biochemical marker total iron binding capacity
time frame: Screening and Day 127
description: Change from Baseline in biochemical markers of disease (total iron binding capacity)
measure: Change from Baseline in the biochemical marker hemoglobin
time frame: Screening and Day 127
description: Change from Baseline in biochemical markers of disease (hemoglobin)
measure: Change from Baseline in the biochemical marker hematocrit
time frame: Screening and Day 127
description: Change from Baseline in biochemical markers of disease (hematocrit)
measure: Change from Baseline in the biochemical marker total protein
time frame: Screening and Day 127
description: Change from Baseline in biochemical markers of disease (total protein)
arm group label: PTR-01 0.1 mg/kg
arm group type: Experimental
description: Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart.
arm group label: PTR-01 0.3 mg/kg
arm group type: Experimental
description: Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart.
arm group label: PTR-01 1.0 mg/kg
arm group type: Experimental
description: Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart.
arm group label: Normal Saline
arm group type: Placebo Comparator
description: Saline control to mimic PTR-01.
arm group label: PTR-01 3.0 mg/kg
arm group type: Experimental
description: Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart.
intervention type: Drug
intervention name: PTR-01
description: Recombinant human collagen 7 (rC7)
arm group label: PTR-01 0.1 mg/kg
arm group label: PTR-01 0.3 mg/kg
arm group label: PTR-01 1.0 mg/kg
arm group label: PTR-01 3.0 mg/kg
other name: Recombinant human collagen 7 (rC7)
intervention type: Drug
intervention name: Normal saline
description: Saline control
arm group label: Normal Saline
criteria: 
gender: All
minimum age: 16 Years
maximum age: N/A
healthy volunteers: No
last name: Theresa Podrebarac, MD
role: Study Director
affiliation: Phoenix Tissue Repair
facility: 
facility: 
facility: 
country: United States
url: http://phoenixtissuerepair.com
description: Sponsor website
responsible party type: Sponsor
mesh term: Epidermolysis Bullosa
mesh term: Epidermolysis Bullosa Dystrophica
sharing ipd: No
ipd description: There are currently no plans to share individual participant data with other researchers.