<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/NCT03307980</url>
  </required_header>
  <id_info>
    <org_study_id>C0371003</org_study_id>
    <secondary_id>SPK-9001-LTFU-101</secondary_id>
    <nct_id>NCT03307980</nct_id>
  </id_info>
  <brief_title>Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B</brief_title>
  <official_title>A FACTOR IX (FIX) GENE TRANSFER, MULTI CENTER EVALUATION OF THE LONG TERM SAFETY AND EFFICACY STUDY OF PF 06838435 AND A DOSE ESCALATION SUBSTUDY IN INDIVIDUALS WITH HEMOPHILIA B</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Pfizer</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Pfizer</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>
      Long-term safety and efficacy follow-up for participants with Hemophilia B who were&#xD;
      previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation&#xD;
      sub-study evaluating safety, tolerability, and kinetics of a higher dose with long-term&#xD;
      safety and efficacy follow-up. Participants in the substudy do not need to have participated&#xD;
      in C0371005.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Evaluation of the long-term level of persistence and potential late or delayed adverse events&#xD;
      associated with PF-06838435 (formerly SPK-9001), assessment of the durability of the&#xD;
      transgene expression, and determination of the effects of PF-06838435 on clinical outcomes in&#xD;
      individuals who have previously received a single administration of PF-06838435 in the&#xD;
      C0371005 study. Amendment 2 of this study incorporates a dose-escalation substudy to evaluate&#xD;
      the safety, tolerability, and kinetics of a single IV infusion of PF-06838435 at a higher&#xD;
      dose than that used in the C0371005 study. The dose-escalation participants will also be&#xD;
      followed for long-term safety and efficacy.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">June 22, 2017</start_date>
  <completion_date type="Anticipated">May 29, 2029</completion_date>
  <primary_completion_date type="Anticipated">May 29, 2029</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Sequential Assignment</intervention_model>
    <intervention_model_description>This study was originally designed as a long-term follow up study for individuals dosed in Study C0371005 to evaluate the overall long-term safety, durability of transgene expression, and effect on clinical outcomes of PF-06838435 mediated gene transfer. For these individuals this study will last for 5 years providing a minimum of 6 years of follow up post vector administration.&#xD;
Amendment 2 of this study introduces a dose-escalation substudy to evaluate the safety, tolerability, and kinetics of a single IV infusion of PF-06838435 at a higher dose(s) than that used in the C0371005 study. For these participants this study will last for a total of 6 years post vector administration.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of PF-06838435 related adverse events</measure>
    <time_frame>Baseline up to Year 6</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Incidence of clinically significant changes from baseline</measure>
    <time_frame>Baseline up to 52 weeks</time_frame>
    <description>Clinically significant changes in physical examination, vital signs, laboratory values. (to be reported as AEs, regardless of causality)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of protocol-defined medically important events</measure>
    <time_frame>Baseline up to 52 weeks</time_frame>
    <description>Clinical thrombotic events, FIX inhibitor development as assessed by Nijmegen Bethesda assay, Hypersensitivity reaction (eg, bronchospasm and anaphylaxis), Hepatic malignancy, Study intervention-related elevated hepatic transaminases that fail to improve or resolve, Malignancy assessed as having reasonable possibility of being related to study intervention (to be reported as SAEs).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Immune response against AAV capsid protein and hFIX transgene</measure>
    <time_frame>Baseline up to 52 weeks</time_frame>
    <description>Positive immune response based on peripheral blood mononuclear cell (PBMC) results by interferon gamma enzyme-linked immunospot assay (ELISPOT).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Coagulation Clotting Assay for FIX activity levels</measure>
    <time_frame>Baseline up to Year 6</time_frame>
    <description>Coagulation Clotting assays to assess FIX activity levels (percent of normal)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean and standard deviation of vector-derived FIX Activity levels</measure>
    <time_frame>Baseline up to 52 weeks</time_frame>
    <description>Mean and standard deviation of peak and steady-state FIX Activity</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean and standard deviation of FIX Antigen levels</measure>
    <time_frame>Baseline up to 52 weeks</time_frame>
    <description>Mean and standard deviation of FIX Antigen levels</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Annualized bleeding rate (ABR)</measure>
    <time_frame>Baseline up to Year 6</time_frame>
    <description>ABR (not including those for surgery)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Annualized (factor FIX) infusion rate</measure>
    <time_frame>Baseline up to Year 6</time_frame>
    <description>AIR (not including those for surgery)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Total factor consumption (IU)</measure>
    <time_frame>Baseline up to Year 6</time_frame>
    <description>total quantity of factor infused annually (not including those for surgery) as recorded on the infusion log</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Total number of bleeding events</measure>
    <time_frame>Baseline up to Year 6</time_frame>
    <description>spontaneous and traumatic</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Haem-A-QoL</measure>
    <time_frame>Baseline up to Year 6</time_frame>
    <description>Quality-of-life (QoL) assessment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>EQ-5D-5L</measure>
    <time_frame>Baseline up to Year 6</time_frame>
    <description>Quality-of-life (QoL) assessment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Brief Pain Inventory</measure>
    <time_frame>Year 2 up to Year 6</time_frame>
    <description>Quality-of-life (QoL) assessment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>McGill Pain Questionnaire</measure>
    <time_frame>Baseline up to 52 weeks</time_frame>
    <description>Quality-of-life (QoL) assessment</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">21</enrollment>
  <condition>Hemophilia B</condition>
  <arm_group>
    <arm_group_label>PF-06838435 Dose-Escalation</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Single intravaneous infusion of PF-06838435. After 2 participants receive initial dose, data will be evaluated and a decision will be made to escalate or reduce the dose being evaluated, increase the number of participants receiving the dose, or stop dosing. Multiple iterations may be undertaken.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>PF-06838435 (formerly SPK-9001)</intervention_name>
    <description>Gene Therapy: A novel, bioengineered adeno-associated viral vector carrying human factor IX variant</description>
    <arm_group_label>PF-06838435 Dose-Escalation</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        This study is currently only enrolling into the dose-escalation substudy with subsequent&#xD;
        long-term follow-up. The Eligibility Criteria for entry into the dose-escalation substudy&#xD;
        is presented below:&#xD;
&#xD;
        Inclusion Criteria:&#xD;
&#xD;
          1. Able to provide informed consent and comply with requirements of the study&#xD;
&#xD;
          2. Males age 18 to 65 years with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2%&#xD;
             endogenous factor IX)&#xD;
&#xD;
          3. Received ≥50 exposure days to factor IX products&#xD;
&#xD;
          4. No measurable factor IX inhibitor as assessed by the central laboratory and have no&#xD;
             prior history of inhibitors to factor IX protein&#xD;
&#xD;
          5. Agree to refrain from donating sperm and either abstain from intercourse or use&#xD;
             reliable barrier contraception until 3 consecutive semen samples are negative for&#xD;
             vector sequences&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Evidence of active hepatitis B or C&#xD;
&#xD;
          2. Currently on antiviral therapy for hepatitis B or C&#xD;
&#xD;
          3. Have significant underlying liver disease&#xD;
&#xD;
          4. Serological evidence* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (* participants who&#xD;
             are HIV+ and stable with CD4 count &gt;200/mm3 and undetectable viral load are eligible&#xD;
             to enroll)&#xD;
&#xD;
          5. Neutralizing antibody titers to the capsid portion of PF-06838435 above the&#xD;
             established threshold&#xD;
&#xD;
          6. Sensitivity to heparin or heparin induced thrombocytopenia; sensitivity to any of the&#xD;
             study interventions, or components thereof, or drug or other allergy&#xD;
&#xD;
          7. Previously dosed in a gene therapy research trial at any time or in an interventional&#xD;
             clinical study within 3 months of screening visit&#xD;
&#xD;
          8. Any concurrent clinically significant major disease or condition&#xD;
&#xD;
          9. Unable or unwilling to comply with the study procedures&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <gender_based>Yes</gender_based>
    <gender_description>Genetic males</gender_description>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Pfizer CT.gov Call Center</last_name>
    <role>Study Director</role>
    <affiliation>Pfizer</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>UC Davis Comprehensive Cancer Center</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UC Davis Ellison Ambulatory Care Clinic</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UC Davis Medical Center department of Radiology</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UC Davis Medical Center</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UC Davis Midtown Cancer Center</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UC DavisHealth Main Hospital</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>LA Center for Bleeding and Clotting Disorders - Metairie</name>
      <address>
        <city>Metairie</city>
        <state>Louisiana</state>
        <zip>70001</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tulane Lakeside Hospital</name>
      <address>
        <city>Metairie</city>
        <state>Louisiana</state>
        <zip>70001</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>LA Center for Bleeding and Clotting Disorders</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tulane University Clinical Translational Unit</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tulane University Hospitals and Clinics</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tulane University Hospitals and Clinic</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tulane University School of Medicine</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Medical Center New Orleans</name>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Louisiana Center for Advanced Medicine</name>
      <address>
        <city>Slidell</city>
        <state>Louisiana</state>
        <zip>70461</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mississippi Center for Advanced Medicine</name>
      <address>
        <city>Madison</city>
        <state>Mississippi</state>
        <zip>39110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Weill Cornell Medicine - New York Presbyterian Hospital</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10065</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Prince Alfred Hospital</name>
      <address>
        <city>Camperdown</city>
        <state>New South Wales</state>
        <zip>2050</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>McMaster University Medical Centre - Hamilton Health Sciences</name>
      <address>
        <city>Hamilton</city>
        <state>Ontario</state>
        <zip>L8N 3Z5</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>McMaster University Medical Centre</name>
      <address>
        <city>Hamilton</city>
        <state>Ontario</state>
        <zip>L8S 4K1</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Juravinski Hospital - Hamilton Health Sciences</name>
      <address>
        <city>Hamilton</city>
        <state>Ontario</state>
        <zip>L8V 1C3</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St. Michael's Hospital</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5B 1W8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St. Michaels Hospital</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5B 1W8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>McGill University Health Center - Research Institute</name>
      <address>
        <city>Montreal</city>
        <state>Quebec</state>
        <zip>H4A 3J1</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal University Hospital</name>
      <address>
        <city>Saskatoon</city>
        <state>Saskatchewan</state>
        <zip>S7N 0W8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Istanbul Universitesi Onkoloji Enstitusu Çocuk Hematoloji Onkoloji Bilim Dali</name>
      <address>
        <city>Istanbul</city>
        <zip>34093</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ege Universitesi Tip Fakultesi Cocuk Sagligi ve Hastaliklari Anabilim Dali</name>
      <address>
        <city>Izmir</city>
        <zip>35100</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>Turkey</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://pmiform.com/clinical-trial-info-request?StudyID=C0371003</url>
    <description>To obtain contact information for a study center near you, click here.</description>
  </link>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>September 29, 2017</study_first_submitted>
  <study_first_submitted_qc>October 6, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">October 12, 2017</study_first_posted>
  <last_update_submitted>September 15, 2023</last_update_submitted>
  <last_update_submitted_qc>September 15, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 18, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>gene therapy</keyword>
  <keyword>BeneGene LTE</keyword>
  <keyword>hemophilia</keyword>
  <keyword>SPK-9001</keyword>
  <keyword>Factor IX</keyword>
  <keyword>FIX</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hemophilia A</mesh_term>
    <mesh_term>Hemophilia B</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.</ipd_description>
    <ipd_url>https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests</ipd_url>
  </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/NCT03307980
org study id: C0371003
secondary id: SPK-9001-LTFU-101
nct id: NCT03307980
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
Long-term safety and efficacy follow-up for participants with Hemophilia B who were
      previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation
      sub-study evaluating safety, tolerability, and kinetics of a higher dose with long-term
      safety and efficacy follow-up. Participants in the substudy do not need to have participated
      in C0371005.
Evaluation of the long-term level of persistence and potential late or delayed adverse events
      associated with PF-06838435 (formerly SPK-9001), assessment of the durability of the
      transgene expression, and determination of the effects of PF-06838435 on clinical outcomes in
      individuals who have previously received a single administration of PF-06838435 in the
      C0371005 study. Amendment 2 of this study incorporates a dose-escalation substudy to evaluate
      the safety, tolerability, and kinetics of a single IV infusion of PF-06838435 at a higher
      dose than that used in the C0371005 study. The dose-escalation participants will also be
      followed for long-term safety and efficacy.
allocation: N/A
intervention model: Sequential Assignment
intervention model description: This study was originally designed as a long-term follow up study for individuals dosed in Study C0371005 to evaluate the overall long-term safety, durability of transgene expression, and effect on clinical outcomes of PF-06838435 mediated gene transfer. For these individuals this study will last for 5 years providing a minimum of 6 years of follow up post vector administration.
Amendment 2 of this study introduces a dose-escalation substudy to evaluate the safety, tolerability, and kinetics of a single IV infusion of PF-06838435 at a higher dose(s) than that used in the C0371005 study. For these participants this study will last for a total of 6 years post vector administration.
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of PF-06838435 related adverse events
time frame: Baseline up to Year 6
measure: Incidence of clinically significant changes from baseline
time frame: Baseline up to 52 weeks
description: Clinically significant changes in physical examination, vital signs, laboratory values. (to be reported as AEs, regardless of causality)
measure: Incidence of protocol-defined medically important events
time frame: Baseline up to 52 weeks
description: Clinical thrombotic events, FIX inhibitor development as assessed by Nijmegen Bethesda assay, Hypersensitivity reaction (eg, bronchospasm and anaphylaxis), Hepatic malignancy, Study intervention-related elevated hepatic transaminases that fail to improve or resolve, Malignancy assessed as having reasonable possibility of being related to study intervention (to be reported as SAEs).
measure: Immune response against AAV capsid protein and hFIX transgene
time frame: Baseline up to 52 weeks
description: Positive immune response based on peripheral blood mononuclear cell (PBMC) results by interferon gamma enzyme-linked immunospot assay (ELISPOT).
measure: Coagulation Clotting Assay for FIX activity levels
time frame: Baseline up to Year 6
description: Coagulation Clotting assays to assess FIX activity levels (percent of normal)
measure: Mean and standard deviation of vector-derived FIX Activity levels
time frame: Baseline up to 52 weeks
description: Mean and standard deviation of peak and steady-state FIX Activity
measure: Mean and standard deviation of FIX Antigen levels
time frame: Baseline up to 52 weeks
description: Mean and standard deviation of FIX Antigen levels
measure: Annualized bleeding rate (ABR)
time frame: Baseline up to Year 6
description: ABR (not including those for surgery)
measure: Annualized (factor FIX) infusion rate
time frame: Baseline up to Year 6
description: AIR (not including those for surgery)
measure: Total factor consumption (IU)
time frame: Baseline up to Year 6
description: total quantity of factor infused annually (not including those for surgery) as recorded on the infusion log
measure: Total number of bleeding events
time frame: Baseline up to Year 6
description: spontaneous and traumatic
measure: Haem-A-QoL
time frame: Baseline up to Year 6
description: Quality-of-life (QoL) assessment
measure: EQ-5D-5L
time frame: Baseline up to Year 6
description: Quality-of-life (QoL) assessment
measure: Brief Pain Inventory
time frame: Year 2 up to Year 6
description: Quality-of-life (QoL) assessment
measure: McGill Pain Questionnaire
time frame: Baseline up to 52 weeks
description: Quality-of-life (QoL) assessment
arm group label: PF-06838435 Dose-Escalation
arm group type: Experimental
description: Single intravaneous infusion of PF-06838435. After 2 participants receive initial dose, data will be evaluated and a decision will be made to escalate or reduce the dose being evaluated, increase the number of participants receiving the dose, or stop dosing. Multiple iterations may be undertaken.
intervention type: Biological
intervention name: PF-06838435 (formerly SPK-9001)
description: Gene Therapy: A novel, bioengineered adeno-associated viral vector carrying human factor IX variant
arm group label: PF-06838435 Dose-Escalation
criteria: 
gender: Male
gender based: Yes
gender description: Genetic males
minimum age: 18 Years
maximum age: 65 Years
healthy volunteers: No
last name: Pfizer CT.gov Call Center
role: Study Director
affiliation: Pfizer
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country: Australia
country: Canada
country: Turkey
country: United States
url: https://pmiform.com/clinical-trial-info-request?StudyID=C0371003
description: To obtain contact information for a study center near you, click here.
responsible party type: Sponsor
mesh term: Hemophilia A
mesh term: Hemophilia B
sharing ipd: Yes
ipd description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
ipd url: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests