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%)
NCT ID: NCT03587116
Last Updated: 2025-11-06
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
212 participants
INTERVENTIONAL
2018-07-26
2024-12-13
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
OTHER
NONE
Study Groups
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Standard of Care FIX replacement therapy
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.
Standard of Care FVIII replacement therapy
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.
Interventions
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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.
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.
Eligibility Criteria
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Inclusion Criteria
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
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.
18 Years
64 Years
MALE
No
Sponsors
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Pfizer
INDUSTRY
Responsible Party
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Principal Investigators
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Pfizer CT.gov Call Center
Role: STUDY_DIRECTOR
Pfizer
Locations
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Clinical and Translational Research Unit (CTRU)
Palo Alto, California, United States
Lucile Packard Childrens Hospital
Palo Alto, California, United States
University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, United States
Stanford Health Care
Stanford, California, United States
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
Emory University Hospital Midtown
Atlanta, Georgia, United States
Emory University Hospital
Atlanta, Georgia, United States
Investigational Drug Service
Atlanta, Georgia, United States
Indiana Hemophilia & Thrombosis Center, Inc.
Indianapolis, Indiana, United States
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States
Alliance for Childhood Diseases
Las Vegas, Nevada, United States
Penn Blood Disorder Center
Philadelphia, Pennsylvania, United States
Bloodworks NW
Seattle, Washington, United States
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
The Alfred Hospital
Melbourne, Victoria, Australia
Fiona Stanley Hospital
Murdoch, Western Australia, Australia
Cliniques universitaires Saint-Luc/Unité d'Hématology et Hémostase
Brussels, , Belgium
Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes
Vitória, Espírito Santo, Brazil
Centro de Hemoterapia e Hematologia do Para - Fundação HEMOPA
Belém, Pará, Brazil
Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP
Campinas, São Paulo, Brazil
Hospital Das Clínicas Da Faculdade de Medicina de Ribeirao Preto Da Universidade de Sao Paulo
Ribeirão Preto, São Paulo, Brazil
lnstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO
Rio de Janeiro, , Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, , Brazil
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, Canada
St. Michael's Hospital
Toronto, Ontario, Canada
CHRU de Brest - La Cavale Blanche
Brest, , France
Hôpital Cardiologique Louis Pradel
Bron, , France
CHU Hôtel-Dieu
Nantes, , France
Hopital Necker, Hematologie Adultes
Paris, , France
Vivantes Klinikum Friedrichshain
Berlin, , Germany
Universitatsklinikum Bonn. Anstalt des oeffentlichen Rechts
Bonn, , Germany
Universitaetsklinikum Giessen
Giessen, , Germany
Universität und Universitätsklinikum des Saarlandes
Homburg/Saar, , Germany
General Hospital of Athens "Hippokration"
Athens, Attica, Greece
General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center
Athens, Attica, Greece
The Chaim Sheba Medical Center, The National Hemophilia Center
Tel Litwinsky, , Israel
IRCCS - AOU di Bologna, Policlinico di Sant'Orsola
Bologna, BO, Italy
UOC Medicina Interna - Malattie Emorragiche e Trombotiche
Napoli, Naples, Italy
SODc. Malattie Emorragiche e della Coagulazione Centro di Riferimento Regionale per le
Florence, , Italy
Università degli studi di Roma "La Sapienza"- Policlinico Umberto I
Roma, , Italy
Nagoya University Hospital - Transfusion Medicine
Nagoya, Aichi-ken, Japan
Sapporo Tokushukai Hospital
Sapporo, Hokkaido, Japan
Nara Medical University Hospital
Kashihara, Nara, Japan
Saitama Medical University Hospital
Iruma-gun, Saitama, Japan
National Center for Child Health and Development
Setagaya-ku, Tokyo, Japan
Ogikubo Hospital
Suginami-ku, Tokyo, Japan
King Abdulaziz Medical City
Riyadh, , Saudi Arabia
King Faisal Specialist Hospital & Research Center
Riyadh, , Saudi Arabia
Kyungpook National University Hospital
Daegu, , South Korea
Severance Hospital, Yonsei University Health System
Seoul, , South Korea
Kyung Hee University Hospital At Gangdong
Seoul, , South Korea
Hospital Universitario Virgen de la Arrixaca
El Palmar, Murcia, Spain
Hospital de la Santa Creu i Sant Pau
Barcelona, , Spain
Hospital Universitari Vall d´Hebrón
Barcelona, , Spain
H.U. La Paz.
Madrid, , Spain
H.U. Rio Hortega
Valladolid, , Spain
Skåne University Hospital
Malmo, , Sweden
Changhua Christian Hospital
Changhua, , Taiwan
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
Chung Shan Medical University Hospital
Taichung, , Taiwan
Taichung Veterans General Hospital
Taichung, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Ege Universitesi Tip Fakultesi Cocuk Sagligi Ve Hastaliklari Anabilim Dali Pediatric Hematoloji
Bornova, İ̇zmir, Turkey (Türkiye)
Ege Universitesi Tip Fakultesi Hematoloji BD
Bornova, İ̇zmir, Turkey (Türkiye)
Acibadem Adana Hospital, Department of Pediatric Hematology
Adana, , Turkey (Türkiye)
Akdeniz University Medical Faculty Hospital
Antalya, , Turkey (Türkiye)
Gaziantep University Sahinbey Training and Research Hospital
Gaziantep, , Turkey (Türkiye)
Istanbul University Oncology Institute
Istanbul, , Turkey (Türkiye)
Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, TYNE & WEAR, United Kingdom
Non Malignant Haematology Research Unit
Newcastle upon Tyne, TYNE & WEAR, United Kingdom
Clinical Research Facility
Glasgow, , United Kingdom
Department of Haematology
Glasgow, , United Kingdom
Guy's and St Thomas' NHS Foundation Trust
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain contact information for a study center near you, click here.
Other Identifiers
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2017-001271-23
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
C0371004
Identifier Type: -
Identifier Source: org_study_id
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