A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE PROSPECTIVE EFFICACY AND SAFETY DATA OF FACTOR IX OR FACTOR VIII PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERATELY SEVERE TO SEVERE ADULT HEMOPHILIA B SUBJECTS (FIX:C≤2%) WHO ARE NEGATIVE FOR NEUTRALIZING nAb TO AAV VECTOR SPARK100 AND MODERATELY SEVERE TO SEVERE HEMOPHILIA A ADULT SUBJECTS (FVIII:C≤1%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR SB-525 CAPSID (AAV6), PRIOR TO THE RESPECTIVE THERAPEUTIC PH 3 GENE THERAPY STUDIES (See Detailed Description Section for Official Protocol Title)

To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study.

To establish baseline prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to AAV6, prior to the Phase 3 gene therapy study.

The enrollment for hemophilia A participants is completed. At this time participants are only being enrolled for hemophilia B cohort.

Study Overview

• Status: Active, not recruiting
• Conditions: Hemophilia A; Hemophilia B
• Intervention / Treatment: Drug: Standard of Care FIX Replacement therapy; Drug: Standard of Care FVIII Replacement therapy

Detailed Description

AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE PROSPECTIVE EFFICACY AND SELECTED SAFETY DATA OF CURRENT FACTOR IX (FIX) OR FACTOR VIII (FVIII) PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERATELY SEVERE TO SEVERE ADULT HEMOPHILIA B SUBJECTS (FIX:C≤2%) WHO ARE NEGATIVE FOR NEUTRALIZING ANTIBODIES TO ADENO-ASSOCIATED VIRUS VECTOR-SPARK100 (BENEGENE-1) AND MODERATELY SEVERE TO SEVERE HEMOPHILIA A ADULT SUBJECTS (FVIII:C≤1%) WHO ARE NEGATIVE FOR NEUTRALIZING ANTIBODIES TO ADENO-ASSOCIATED VIRUS VECTOR 6 (AAV6), PRIOR TO THE RESPECTIVE THERAPEUTIC PHASE 3 GENE THERAPY STUDIES

• Study Type: Interventional
• Enrollment (Actual): 213
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Pfizer CT.gov Call Center; Phone Number: 1-800-718-1021; Email: ClinicalTrials.gov_Inquiries@pfizer.com

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Royal Prince Alfred Hospital
  • Queensland
    • Herston, Queensland, Australia, 4029
      • Royal Brisbane and Women'S Hospital
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Royal Adelaide Hospital
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • The Alfred Hospital
  • Western Australia
    • Murdoch, Western Australia, Australia, 6150
      • Fiona Stanley Hospital
• Belgium
  • Brussels, Belgium, 1200
    • Cliniques universitaires Saint-Luc/Unité d'Hématology et Hémostase
• Brazil
  • Rio de Janeiro, Brazil, 20211-030
    • lnstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO
  • São Paulo, Brazil, 05403-000
    • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
  • Espirito Santo
    • Vitoria, Espirito Santo, Brazil, 29047-105
      • Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes
  • Para
    • Belem, Para, Brazil, 66033-000
      • Centro de Hemoterapia e Hematologia do Para - Fundação HEMOPA
  • SAO Paulo
    • Campinas, SAO Paulo, Brazil, 13083-878
      • Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP
  • SÃO Paulo
    • Ribeirão Preto, SÃO Paulo, Brazil, 14051-140
      • Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto da Universidade de Sao Paulo
• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8N 3Z5
      • McMaster University Medical Centre - Hamilton Health Sciences
    • Toronto, Ontario, Canada, M5B 1W8
      • St. Michael's Hospital
• France
  • Brest, France, 29200
    • CHRU de Brest - Hôpital Morvan
  • Bron, France, 69677
    • Hôpital Cardiologique Louis Pradel
  • Nantes Cedex 1, France, 44093
    • CHU Hôtel-Dieu
  • Paris, France, 75015
    • Hopital Necker, Hematologie Adultes
• Germany
  • Berlin, Germany, 10249
    • Vivantes Klinikum Friedrichshain
  • Bonn, Germany, 53127
    • Universitatsklinikum Bonn. Anstalt des oeffentlichen Rechts
  • Frankfurt/Main, Germany, 60590
    • Universitaetsklinikum Frankfurt, Goethe-Universitaet
  • Giessen, Germany, 35392
    • Universitaetsklinikum Giessen
  • Homburg/Saar, Germany, 66421
    • Universität und Universitätsklinikum des Saarlandes
• Greece
  • Attica
    • Athens, Attica, Greece, 11527
      • General Hospital of Athens "Hippokration"
    • Athens, Attica, Greece, 11527
      • General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center
• Israel
  • Tel-Hashomer, Israel, 5262000
    • The Chaim Sheba Medical Center, The National Hemophilia Center
• Italy
  • Firenze, Italy, 50134
    • SODc. Malattie Emorragiche e della Coagulazione Centro di Riferimento Regionale per le
  • Napoli, Italy, 80131
    • Azienda Ospedaliera Universitaria Federico II
  • Roma, Italy, 00161
    • Università degli studi di Roma "La Sapienza"- Policlinico Umberto I
  • BO
    • Bologna, BO, Italy, 40138
      • IRCCS - AOU di Bologna, Policlinico di Sant'Orsola
• Japan
  • Aichi
    • Nagoya, Aichi, Japan, 466-8560
      • Nagoya University Hospital - Transfusion Medicine
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 004-0041
      • Sapporo Tokushukai Hospital
  • Nara
    • Kashihara, Nara, Japan, 634-8522
      • Nara Medical University Hospital
  • Saitama
    • Iruma-gun, Saitama, Japan, 350-0495
      • Saitama Medical University Hospital
  • Tokyo
    • Setagaya-ku, Tokyo, Japan, 157-8535
      • National Center for Child Health and Development
    • Suginami-ku, Tokyo, Japan, 167-0035
      • Ogikubo Hospital
• Korea, Republic of
  • Daegu, Korea, Republic of, 41944
    • Kyungpook National University Hospital
  • Seoul, Korea, Republic of, 03722
    • Severance Hospital, Yonsei University Health System
  • Seoul, Korea, Republic of, 05278
    • Kyung Hee University Hospital at Gangdong
• Saudi Arabia
  • Riyadh, Saudi Arabia, 11426
    • King Abdulaziz Medical City
  • Riyadh, Saudi Arabia
    • King Faisal Specialist Hospital & Research Center
• Spain
  • Barcelona, Spain, 08025
    • Hospital de la Santa Creu i Sant Pau
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d´Hebron
  • Madrid, Spain, 28046
    • H.U. La Paz.
  • Valladolid, Spain, 47012
    • H.U. Rio Hortega
  • Murcia
    • El Palmar, Murcia, Spain, 30120
      • Hospital Universitario Virgen de la Arrixaca
• Sweden
  • Malmö, Sweden, 205 02
    • Skåne University Hospital
• Taiwan
  • Changhua, Taiwan, 500
    • Changhua Christian Hospital
  • Kaohsiung, Taiwan, 807
    • Kaohsiung Medical University Chung-Ho Memorial Hospital
  • Taichung, Taiwan, 40705
    • Taichung Veterans General Hospital
  • Taichung City, Taiwan, 40201
    • Chung Shan Medical University Hospital
  • Taipei, Taiwan, 10002
    • National Taiwan University Hospital
  • Taipei City, Taiwan, 110
    • Taipei Medical University Hospital
• Turkey
  • Adana, Turkey, 01130
    • Acibadem Adana Hospital, Department of Pediatric Hematology
  • Antalya, Turkey, 07070
    • Akdeniz University Medical Faculty Hospital
  • Gaziantep, Turkey, 27310
    • Gaziantep University Sahinbey Training and Research Hospital
  • Istanbul, Turkey, 34093
    • Istanbul University Oncology Institute
  • İ̇zmir
    • Bornova, İ̇zmir, Turkey, 35040
      • Ege Universitesi Tip Fakultesi Cocuk Sagligi Ve Hastaliklari Anabilim Dali Pediatric Hematoloji
    • Bornova, İ̇zmir, Turkey, 35040
      • Ege Universitesi Tip Fakultesi Hematoloji BD
• United Kingdom
  • Glasgow, United Kingdom, G31 2ER
    • Clinical Research Facility
  • Glasgow, United Kingdom, G4 0SF
    • Department of Haematology
  • London, United Kingdom, SE1 7EH
    • Guy's and St Thomas' NHS Foundation Trust
  • Tyne & Wear
    • Newcastle upon Tyne, Tyne & Wear, United Kingdom, NE1 4LP
      • Newcastle Upon Tyne Hospitals NHS Foundation Trust
    • Newcastle upon Tyne, Tyne & Wear, United Kingdom, NE1 4LP
      • Non Malignant Haematology Research Unit
• United States
  • California
    • Palo Alto, California, United States, 94304
      • Clinical and Translational Research Unit (CTRU)
    • Palo Alto, California, United States, 94304
      • Lucile Packard Childrens Hospital
    • San Francisco, California, United States, 94143
      • University of California, San Francisco - Outpatient Hematology Clinic
    • Stanford, California, United States, 94305
      • Stanford Health Care
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University Hospital
    • Atlanta, Georgia, United States, 30308
      • Emory University Hospital Midtown
    • Atlanta, Georgia, United States, 30322
      • Investigational Drug Service
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Indiana Hemophilia & Thrombosis Center, Inc.
  • Mississippi
    • Madison, Mississippi, United States, 39110
      • Mississippi Center for Advanced Medicine
  • Nevada
    • Las Vegas, Nevada, United States, 89135
      • Alliance for Childhood Diseases
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Penn Blood Disorder Center
    • Philadelphia, Pennsylvania, United States, 19104
      • Penn Comprehensive Hemophilia & Thrombosis Program, Hospital of the University of Pennsylvania
  • Washington
    • Seattle, Washington, United States, 98104
      • Bloodworks NW

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 64 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Hemophilia B Population:

1. Evidence of a signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study.
2. Willing and able to comply with scheduled visits, FIX prophylaxis treatment plan, laboratory tests and other study procedures.
3. Males ≥ 18 and <65 years of age with moderately severe to severe hemophilia B and documented FIX activity (≤2%) prior to baseline visit.
4. Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product).
5. Participants on FIX prophylaxis replacement therapy (recombinant, plasma-derived or extended half-life FIX product) must have the intention to remain on FIX prophylaxis replacement therapy for the duration of the study.
6. No known hypersensitivity to FIX replacement product.

7. No history of FIX inhibitor (clinical or laboratory-based assessment) defined as a titer

   • 0.6 BU/mL, regardless of the laboratory normal range, or any measured Bethesda inhibitor titer greater than the upper limit of normal for the laboratory performing the assay. Clinically, no signs or symptoms of decreased response to FIX administration. Participants will not be required to undergo diagnostic evaluation of inhibitor status to participate in the study.

Hemophilia A Population:

1. Evidence of a signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study.
2. Willing and able to comply with scheduled visits, FVIII prophylaxis treatment plan, laboratory tests and other study procedures.
3. Males ≥18 and <65 years of age with moderately severe to severe hemophilia A and documented FVIII activity (≤1%) prior to baseline visit.
4. Previous experience with FVIII therapy (≥150 documented exposure days to a FVIII protein product such as recombinant, plasma-derived or extended half-life FVIII product).
5. Participants must be on a stable FVIII prophylaxis replacement therapy (recombinant, plasma-derived or extended half-life FVIII product) at study entry and must have the intention to remain on FVIII prophylaxis replacement therapy for the duration of the study. This does not include nonfactor treatments, which are prohibited.
6. No known hypersensitivity to FVIII replacement product.
7. No history of FVIII inhibitor (clinical or laboratory-based assessment) defined as a titer ≥0.6 BU/mL, regardless of the laboratory normal range, or any measured Bethesda inhibitor titer greater than the upper limit of normal for the laboratory performing the assay. Clinically, no signs or symptoms of decreased response to FVIII administration. Participants will not be required to undergo diagnostic evaluation of inhibitor status to participate in the study.

Exclusion Criteria:

1. Anti-AAV-Spark100 neutralizing antibody titer above the established threshold performed by a central laboratory during screening in hemophilia B subjects or Anti-AAV6 neutralizing antibody titer above the established threshold performed by a central laboratory during screening in hemophilia A subjects.
2. Lack of participant compliance with documentation of bleeds and/or prophylaxis replacement therapy administration.

3. If there is no documentation regarding hepatitis status, as defined below, within the last 12 months prior to screening for hepatitis B and 6 months prior to screening for hepatitis C, then participants will be required to have the following hepatitis testing performed at screening:

   1. Hepatitis B screening (acute and chronic):

      HBsAg (also referred to as Hepatitis B surface antigen), HBV-DNA viral assay (also referred to as a nucleic acid test for Hepatitis B virus DNA), and Anti-HBc (also referred to as Total Hepatitis B core antibody).

      • A participant is not eligible if either HbsAg is positive or HBV-DNA is positive/detectable.
      • Anti-HBc must be obtained in all participants for determination of whether the participant had prior hepatitis B. If the anti-HBc is positive and both HBsAg and HBV DNA are negative this would be consistent with a prior infection and the subject would be eligible for the study. Anti-HBc must be obtained in all subjects to discriminate between those with no prior hepatitis B and those with prior infection in the event of reactivation. FDA has noted reactivation of hepatitis B virus exists.
      • One documented negative HBV-DNA viral load is sufficient to assess eligibility. A participant who is currently undergoing anti-viral therapy for hepatitis B is not eligible.

   2. Hepatitis C (acute or chronic):

      • A participant who is currently undergoing anti-viral therapy for chronic hepatitis C is not eligible.
      • Participants treated with anti-viral therapy for chronic hepatitis C, must have completed anti-viral therapy at least 6 months prior to screening and have a negative HCV-RNA at least 6 months prior to screening.
      • All participants (who are not currently undergoing anti-viral therapy for chronic hepatitis C) must have a single HCV-RNA load assay (also referred to as a nucleic acid test [NAT] for HCV RNA) obtained during the 6 months preceding screening. This includes participants with prior known chronic hepatitis C who have completed treatment with anti-viral therapy.
      • A participant is not eligible if his HCV-RNA load assay result is positive/detectable.
4. Currently on antiviral therapy for hepatitis B or C.

5. Significant liver disease, as defined by pre-existing diagnosis of portal hypertension, splenomegaly, or hepatic encephalopathy.

   All participants who do not have the listed pre existing diagnoses above must have the following assessments performed within the last 12 months prior to screening and if not will need to be tested for liver fibrosis status at screening: a serum albumin level below normal limits and/or significant liver fibrosis by any of the following diagnostic modalities: FibroScan median stiffness score >8 kPa units OR FibroTest/FibroSURE >0.48*.

   * NOTE: If there is concern regarding the validity of any of the liver fibrosis test results please contact the medical monitor to discuss whether any additional testing needs to be performed (ie, either repeating any test or performing another fibrosis test). Also, note, if a participant has a known history of Gilbert's syndrome, a FibroTest cannot be used for fibrosis testing.

6. Documented serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ≤200 mm3 within the last 12 months prior to screening. Participants who are HIV positive and stable, have an adequate CD4 count (>200/mm3) and undetectable viral load (<50 gc/mL) documented within the preceding 12 months, and are on an antiretroviral drug regimen are eligible to enroll. Participants who have not been tested within the prior 12 months of screening will need to be tested for HIV status at screening.
7. History of chronic infection or other chronic disease that the investigator deems an unacceptable risk. In addition, any participant with conditions associated with increased thromboembolic risk such as known inherited or acquired thrombophilia, or a history of thrombotic events including but not limited to stroke, myocardial infarction, and/or venous thromboembolism, is excluded.
8. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation or other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study. In addition, any participant with a history of a neoplasm (including hepatic malignancy) that required treatment (eg, chemotherapy, radiotherapy, immunotherapy), is excluded, except for adequately treated basal or squamous cell carcinoma of the skin or a surgically removed benign neoplasm not requiring chemotherapy, radiotherapy and/or immunotherapy. Any other neoplasm that has been cured by resection should be discussed between the investigator and sponsor.

9. Participation in other studies if involving administration of investigational product(s) within the last 3 months prior to study entry and/or during study participation or in a previous gene therapy clinical study within the last 12 months prior to screening.

   • Participants already enrolled in this lead-in study (C0371004) may be allowed to participate in the screening and baseline periods of either C0371002 or C3731003 protocols prior to their completion of the end of study visit in this lead-in study.

10. Any participant who previously received fidanacogene elaparvovec (hemophilia B) or giroctocogene fitelparvovec (hemophilia A) or any AAV gene-based therapy.
11. Participants using restricted therapies.
12. Any participant with a planned surgical procedure requiring FIX (hemophilia B) or FVIII (hemophilia A) surgical prophylactic factor treatment in the next 24 months.
13. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.

NOTE: The sponsor's medical team should be contacted if there are any questions regarding any of the inclusion or exclusion criteria (Sections: 4.1 and 4.2).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Standard of Care FIX replacement therapy	Drug: Standard of Care FIX Replacement therapy; There is no investigational product being administered. Subjects will be administering their own standard of care FIX replacement therapy.
Other: Standard of Care FVIII replacement therapy	Drug: Standard of Care FVIII Replacement therapy; There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized bleeding rate (ABR)	The annualized bleeding rate (ABR) per subject will be calculated as the number of bleeds over number of days subject received FIX prophylaxis replacement therapy from baseline visit (Day 1) to end of study x 365.25 days. ABR will be summarized using descriptive statistics (n, mean, standard deviation, median, Q1, Q3, minimum, maximum). The annualized bleeding rate (ABR) per subject will be calculated as the number of bleeds over number of days subject received FVIII prophylaxis replacement therapy from baseline visit (Day 1) to end of study x 365.25 days. ABR will be summarized using descriptive statistics (n, mean, standard deviation, median, Q1, Q3, minimum, maximum).	6 months
Incidence of serious adverse events	The primary safety analysis will be performed on all subjects that sign the informed consent document and are subsequently identified as nAb negative and are enrolled (complete baseline visit) into the study.	6 months
Events of special interest (ESI):inhibitor against FIX or FVIII, thrombotic events, and FIX or FVIII hypersensitivity reactions	Frequency and percentage of these ESI events will be summarized by event. In addition any events leading to discontinuation from the study will be described.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized infusion rate (AIR)	The annualized infusion rate (AIR) per subject will be calculated as the number of infusions received over number of days subject received FIX prophylaxis replacement therapy from baseline visit (Day 1) to end of study x 365.25 days. AIR will be summarized using descriptive statistics (n, mean, standard deviation, median, Q1, Q3, minimum, maximum). The annualized infusion rate (AIR) per subject will be calculated as the number of infusions received over number of days subject received FVIII prophylaxis replacement therapy from baseline visit (Day 1) to end of study x 365.25 days. AIR will be summarized using descriptive statistics (n, mean, standard deviation, median, Q1, Q3, minimum, maximum).	6 months
Dose and total factor consumption	The total factor IX replacement therapy consumption and the corresponding dose will be descriptively summarized by the categories of the replacement therapy, where appropriate. Infusion diary (electronic infusion diary) of the factor IX replacement therapy will be listed. The total factor VIII replacement therapy consumption and the corresponding dose will be descriptively summarized by the categories of the replacement therapy, where appropriate. Infusion diary (electronic infusion diary) of the factor VIII replacement therapy will be listed.	6 months
Number of bleeding events (spontaneous and/or traumatic)	The number of bleeding episodes will be summed up by spontaneous, traumatic and overall as defined as any bleed occurring >72 hours after stopping treatment from the original bleed for which treatment was initiated or a bleed occurring at a different site from the original bleed regardless of the time from last injection.	6 months

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): July 26, 2018
• Primary Completion (Estimated): May 24, 2024
• Study Completion (Estimated): May 24, 2024

Study Registration Dates

• First Submitted: June 29, 2018
• First Submitted That Met QC Criteria: July 2, 2018
• First Posted (Actual): July 16, 2018

Study Record Updates

• Last Update Posted (Actual): March 21, 2024
• Last Update Submitted That Met QC Criteria: March 19, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: C0371004; 2017-001271-23 (EudraCT Number); NAB PROTOCOL (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No