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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-502844-11-00 | Registry Identifier | CTIS (EU) |
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This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate [ABR] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PF-06838435/ fidanacogene elaparvovec | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PF-06838435/ fidanacogene elaparvovec | Biological | Gene Therapy |
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| Measure | Description | Time Frame |
|---|---|---|
| Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| Measure | Description | Time Frame |
|---|---|---|
| ABR for Treated Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. |
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Inclusion Criteria
Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF IDS Pharmacy | San Francisco | California | 94143 | United States | ||
| University of California, San Francisco - Clinical Research Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40750723 | Derived | Wojciechowski J, Gaitonde P, Hughes JH, Ravva P. Population Modeling of Factor IX Activity Following Administration of Fidanacogene Elaparvovec Gene Therapy in Participants with Hemophilia B. Clin Pharmacokinet. 2025 Oct;64(10):1531-1548. doi: 10.1007/s40262-025-01535-y. Epub 2025 Aug 1. | |
| 39321362 | Derived |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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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.
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A total of 51 participants were screened in this study and 45 participants received a single dose of PF-06838435 (fidanacogene elaparvovec). Results presented are for primary outcome measures and only those secondary measures whose analysis was complete on a data cutoff date of 16 November 2022 (primary completion date [PCD]).
Participants with moderately severe to severe hemophilia B, who completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead-in study C0371004 (NCT03587116), were enrolled in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | PF-06838435 | Participants received PF-06838435 infusion on Day 1 at a dose of 5*10^11 vector genomes per kilogram (kg) of body weight. Short term follow-up: Participants were followed up for 52 weeks/Year 1 post infusion. Long term follow-up: Participants were followed up from Year 2 to 6. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Screening Phase (3 Weeks) |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 29, 2022 | Nov 1, 2023 |
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| Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| Annualized Infusion Rate (AIR) of Exogenous FIX From Week 12 to Month 15 | AIR = number of exogenous infusions (for any reason) received during given time period *365.25/ (Date of last day - date of first day +1) in that time period. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| Steady State Circulating Factor IX (FIX:C) From Week 12 to Month 15 | The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent. Data reported in this Outcome Measure is the geometric mean of all assessments from week 12 to Month 15. | Week 12 to Month 15 |
| Circulating Factor IX (FIX:C) at Week 12, Week 24, Week 65 | The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent. | Week 12, Week 24, Week 65 |
| Annualized Factor IX (FIX) Consumption From Week 12 to Month 15 | Annualized FIX consumption was reported by International Units per kilogram (IU/kg). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. Data reported in this Outcome Measure is average of all assessments from Week 12 to Month 15. | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| ABR for Spontaneous Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Spontaneous Bleeds: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| ABR for Traumatic Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Traumatic Bleeds: Bleeding event occurring for an apparent/known reason. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| ABR for Untreated Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| Number of Target Joint Bleeds From Week 12 to Month 15 | Target Joint: Defined as a major joint (e.g., hip, elbow, wrist, shoulder, knee, and ankle) into which repeated bleeds occurred (three or more spontaneous bleeds into a single joint within a consecutive 6-month period). A target joint was considered resolved when there were =<2 bleeds into the joint within a 12-month period. Joint Bleed: A bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of the following: pain or an unusual sensation in the joint, palpable swelling, and warmth of the skin over the joint. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
| Percentage of the Participants Without Bleeds From Week 12 to Month 15 | Percentage of participants without bleeds (total bleeds and treated bleeds) were summarized by type from Week 12 to Month 15. | Week 12 to Month 15 |
| Change From Baseline in Joint Health as Measured by the Hemophilia Joint Health Score (HJHS) Instrument at Month 12 | A qualified healthcare professional assessed six joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) scored from 0 to 20 based on: duration of swelling, muscle atrophy, crepitus, flexion loss, extension loss, instability, joint pain, and strength. Gait was scored (0 to 4) based on walking, stairs, running, hopping on one leg. Total score = sum of scores from all joints + gait score ranged from 0 to 124, with the higher the number equating to more severe joint damage. | Baseline, Month 12 |
| Change From Baseline in Hemophilia Quality of Life (Haem A QoL) Physical Health Domain at Month 12 | The Haem-A-QoL questionnaire contained 46 items with ten domains that assessed health in the following areas: Physical Health; Feelings; View of Self; Sports and Leisure; Work and School; Dealing with Haemophilia; Treatment; Future; Family Planning; and Partnership and Sexuality. The physical health domain was considered as the primary domain in this questionnaire, had a transformed score range from 0 to 100, with lower scores representing higher quality of life. In this Outcome Measure Physical Health domain scores of Haem A QoL are reported. | Baseline, Month 12 |
| Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Month 12 | The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults. The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Selected items from five of the domains were used to create three components: upper extremity; basic lower extremity; and complex lower extremity activities. The component score of "complex lower extremity activities" was the most important in this questionnaire, had a transformed score range from 0 to 100, higher values indicated less functional limitations in performing tasks. | Baseline, Month 12 |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| Number of Participants With Adverse Events of Special Interest | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| Number of Participants With Positive Neutralizing Antibody (nAb) to Adeno-associated Virus Vector (AAV) and Anti-Drug Antibody (ADA) | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| ABR for Total Bleeds (Treated and Untreated) Through the Study | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| ABR for Treated Bleeds Through the Study | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| AIR of Exogenous Factor IX Through the Study | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| FIX: C Level Through the Study | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| Annualized Factor IX Consumption Through the Study | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| ABR for Spontaneous and Traumatic, and Untreated Bleeds Through the Study | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| Change From Baseline in Joint Health as Measured by the HJHS Instrument Through the Study | Results would be posted at secondary completion date. | Baseline, 6 years |
| Number of Target Joint Bleeds Through the Study | Results would be posted at secondary completion date. | Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| Change From Baseline in Haem A QoL Physical Health Domain Through the Study | Results would be posted at secondary completion date. | Baseline, Maximum up to 6 years (Week 312) after PF-06838435 infusion |
| Change From Baseline in HAL Complex Lower Extremity Activities Component Score Through the Study | Results would be posted at secondary completion date. | Baseline, 6 years |
| San Francisco |
| California |
| 94143 |
| United States |
| University of California, San Francisco - Outpatient Hematology Clinic | San Francisco | California | 94143 | United States |
| The Regents of the University of California, San Francisco campus | San Francisco | California | 94158 | United States |
| Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | United States |
| Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts | Aurora | Colorado | 80045 | United States |
| Indiana Hemophilia & Thrombosis Center, Inc. | Indianapolis | Indiana | 46260 | United States |
| Indiana Hemophilia and Thrombosis Center, Inc | Indianapolis | Indiana | 46260 | United States |
| Innovative Hematology, Inc. | Indianapolis | Indiana | 46260 | United States |
| St. Vincent Hospital & Health Care Center, Inc. | Indianapolis | Indiana | 46260 | United States |
| Madison Radiological Group | Madison | Mississippi | 39110 | United States |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | 39110 | United States |
| The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal | Hamilton | Pennsylvania | 19104 | United States |
| Center for Human Phenomic Science | Philadelphia | Pennsylvania | 19104 | United States |
| Investigational Drug Service | Philadelphia | Pennsylvania | 19104 | United States |
| Penn Blood Disorder Center | Philadelphia | Pennsylvania | 19104 | United States |
| The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal | Philadelphia | Pennsylvania | 19104 | United States |
| Royal Prince Alfred Hospital | Camperdown | New South Wales | 2050 | Australia |
| Royal Adelaide Hospital | Adelaide | South Australia | 5000 | Australia |
| The Alfred Hospital | Melbourne | Victoria | 3004 | Australia |
| Fiona Stanley Hospital | Murdoch | Western Australia | 6150 | Australia |
| Royal Brisbane and Women's Hospital | Herston | 4029 | Australia |
| Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes | Vitória | Espírito Santo | 29047-105 | Brazil |
| Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP | Campinas | São Paulo | 13083-878 | Brazil |
| Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo | São Paulo | São Paulo | 05403-010 | Brazil |
| Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO | Rio de Janeiro | 20211-030 | Brazil |
| McMaster University Medical Centre - Hamilton Health Sciences | Hamilton | Ontario | L8N 3Z5 | Canada |
| Juravinski Hospital - Hamilton Health Sciences | Hamilton | Ontario | L8V 1C3 | Canada |
| St. Michael's Hospital | Toronto | Ontario | M5B 1W8 | Canada |
| Hopital Cardiologique Louis Pradel - CRTH | Bron | 69677 | France |
| Hopital Necker | Paris | 75015 | France |
| Vivantes Klinikum Friedrichshain | Berlin | 10249 | Germany |
| Vivantes Klinikum im Friedrichshain | Berlin | 10249 | Germany |
| Universitätsklinikum Bonn Institut für Klinische Chemie und Klinische Pharmakologie -Phase 1-Einheit | Bonn | 53127 | Germany |
| Universitätsklinikum Bonn, Anstalt des öffentlichen Rechts | Bonn | 53127 | Germany |
| Universitätsklinikum Bonn | Bonn | 53127 | Germany |
| General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center | Athens | 11527 | Greece |
| Azienda Ospedaliero Universitaria Careggi | Florence | 50134 | Italy |
| Azienda Ospedaliera Universitaria Federico II | Naples | 80131 | Italy |
| Sapporo Tokushukai Hospital | Sapporo | Hokkaido | 004-0041 | Japan |
| Nara Medical University Hospital | Kashihara | Nara | 634-8522 | Japan |
| Saitama Medical University Hospital | Iruma-gun | Saitama | 350-0495 | Japan |
| National Center for Child Health and Development | Setagaya-ku | Tokyo | 157 8535 | Japan |
| King Abdulaziz Medical City-Ministry of national guard | Riyadh | Kingdom of Saudi Arabia | 14611 | Saudi Arabia |
| King Faisal Specialist Hospital and Research Centre | Riyadh | 11211 | Saudi Arabia |
| King Abdullah International Medical Research Center | Riyadh | 11481 | Saudi Arabia |
| Kyung Hee University Hospital at Gangdong | Seoul | 05278 | South Korea |
| Hospital Universitario Virgen de la Arrixaca | El Palmar | Murcia | 30120 | Spain |
| Hospital Universitari Vall d´Hebrón | Barcelona | 08035 | Spain |
| Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics | Malmö | 205 02 | Sweden |
| ApoEx AB | Malmö | 211 24 | Sweden |
| Changhua Christian Hospital | Changhua | 500 | Taiwan |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | 807 | Taiwan |
| Chung Shan Medical University Hospital | Taichung | 40201 | Taiwan |
| Chung Shan Medical University | Taichung | 40201 | Taiwan |
| Taichung Veterans General Hospital | Taichung | 40705 | Taiwan |
| National Taiwan University Hospital | Taipei | 10002 | Taiwan |
| Acibadem Adana Hospital | Adana | 01130 | Turkey (Türkiye) |
| Gaziantep University Sahinbey Training and Research Hospital | Gaziantep | 27310 | Turkey (Türkiye) |
| Istanbul University Oncology Institute | Istanbul | 34093 | Turkey (Türkiye) |
| Ege University Medical Faculty Hospital | Izmir | 35040 | Turkey (Türkiye) |
| Newcastle upon Tyne Hospitals NHS FT | Newcastle upon Tyne | TYNE & WEAR | NE1 4LP | United Kingdom |
| Newcastle upon Tyne Hospitals NHS FT | Newcastle upon Tyne | TYNE & WEAR | NE2 4HH | United Kingdom |
| Glasgow Royal Infirmary | Glasgow | G4 0SF | United Kingdom |
| Guy's & St Thomas' NHS Foundation Trust, St Thomas' Hospital | London | SE1 7EH | United Kingdom |
| Guy's and St. Thomas' NHS Foundation Trust | London | SE1 7EH | United Kingdom |
| Newcastle upon Tyne Hospitals NHS Foundation Trust | Newcastle upon Tyne | NE2 4HH | United Kingdom |
| Cuker A, Kavakli K, Frenzel L, Wang JD, Astermark J, Cerqueira MH, Iorio A, Katsarou-Fasouli O, Klamroth R, Shapiro AD, Hermans C, Ishiguro A, Leavitt AD, Oldenburg JB, Ozelo MC, Teitel J, Biondo F, Fang A, Fuiman J, McKay J, Sun P, Rasko JEJ, Rupon J; BENEGENE-2 Trial Investigators. Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B. N Engl J Med. 2024 Sep 26;391(12):1108-1118. doi: 10.1056/NEJMoa2302982. |
| COMPLETED |
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| NOT COMPLETED |
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| Treatment Phase (Day 1) |
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| Follow-up Phase (Year 1) |
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| Long-Term Follow-Up (Year 2 to Year 6) |
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Safety analysis set included all participants enrolled in the study and who received PF-06838435 infusion.
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| ID | Title | Description |
|---|---|---|
| BG000 | PF-06838435 | All participants enrolled in the study and who received PF-06838435 infusion. |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
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| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | 95% Confidence Interval | Total bleeds per year | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | ABR for Treated Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | 95% Confidence Interval | Treated bleeds per year | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | Annualized Infusion Rate (AIR) of Exogenous FIX From Week 12 to Month 15 | AIR = number of exogenous infusions (for any reason) received during given time period *365.25/ (Date of last day - date of first day +1) in that time period. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | Standard Deviation | Exogenous infusions per year | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | Steady State Circulating Factor IX (FIX:C) From Week 12 to Month 15 | The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent. Data reported in this Outcome Measure is the geometric mean of all assessments from week 12 to Month 15. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. The assessments at the visits following participant withdrawal/dropout/FIX prophylaxis resumption were imputed as 1.9%. Here, "Number Analyzed" signifies number of participants evaluable for specified rows of respective arms. | Posted | Geometric Mean | Standard Deviation | Percentage of Normal | Week 12 to Month 15 |
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| Secondary | Circulating Factor IX (FIX:C) at Week 12, Week 24, Week 65 | The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent. | Dosed analysis set: participants enrolled in the study who received PF-06838435 infusion. The assessments at the visits following participant withdrawal/dropout/FIX prophylaxis resumption were imputed as 1.9%. "Number Analyzed": at each row represents the number of participants with valid FIX:C assessments at the respective time points. All participants reported under 'Number of Participants Analyzed' contributed data to the table; however, may not have evaluable data for every row. | Posted | Mean | Standard Deviation | Percentage of Normal | Week 12, Week 24, Week 65 |
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| Secondary | Annualized Factor IX (FIX) Consumption From Week 12 to Month 15 | Annualized FIX consumption was reported by International Units per kilogram (IU/kg). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. Data reported in this Outcome Measure is average of all assessments from Week 12 to Month 15. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | Standard Deviation | IU/kg per year | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | ABR for Spontaneous Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Spontaneous Bleeds: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | 95% Confidence Interval | Spontaneous bleeds per year | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | ABR for Traumatic Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Traumatic Bleeds: Bleeding event occurring for an apparent/known reason. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | 95% Confidence Interval | Traumatic bleeds per year | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | ABR for Untreated Bleeds From Week 12 to Month 15 | ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | 95% Confidence Interval | Untreated bleeds per year | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | Number of Target Joint Bleeds From Week 12 to Month 15 | Target Joint: Defined as a major joint (e.g., hip, elbow, wrist, shoulder, knee, and ankle) into which repeated bleeds occurred (three or more spontaneous bleeds into a single joint within a consecutive 6-month period). A target joint was considered resolved when there were =<2 bleeds into the joint within a 12-month period. Joint Bleed: A bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of the following: pain or an unusual sensation in the joint, palpable swelling, and warmth of the skin over the joint. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Mean | Standard Deviation | Target joint bleeds | Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm) |
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| Secondary | Percentage of the Participants Without Bleeds From Week 12 to Month 15 | Percentage of participants without bleeds (total bleeds and treated bleeds) were summarized by type from Week 12 to Month 15. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. | Posted | Number | Percentage of participants | Week 12 to Month 15 |
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| Secondary | Change From Baseline in Joint Health as Measured by the Hemophilia Joint Health Score (HJHS) Instrument at Month 12 | A qualified healthcare professional assessed six joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) scored from 0 to 20 based on: duration of swelling, muscle atrophy, crepitus, flexion loss, extension loss, instability, joint pain, and strength. Gait was scored (0 to 4) based on walking, stairs, running, hopping on one leg. Total score = sum of scores from all joints + gait score ranged from 0 to 124, with the higher the number equating to more severe joint damage. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. Here, "Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies number of participants evaluable for specified rows of respective arms. | Posted | Mean | 95% Confidence Interval | Units on a scale | Baseline, Month 12 |
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| Secondary | Change From Baseline in Hemophilia Quality of Life (Haem A QoL) Physical Health Domain at Month 12 | The Haem-A-QoL questionnaire contained 46 items with ten domains that assessed health in the following areas: Physical Health; Feelings; View of Self; Sports and Leisure; Work and School; Dealing with Haemophilia; Treatment; Future; Family Planning; and Partnership and Sexuality. The physical health domain was considered as the primary domain in this questionnaire, had a transformed score range from 0 to 100, with lower scores representing higher quality of life. In this Outcome Measure Physical Health domain scores of Haem A QoL are reported. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. Here, "Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies number of participants evaluable for specified rows of respective arms. | Posted | Mean | 95% Confidence Interval | Units on a scale | Baseline, Month 12 |
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| Secondary | Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Month 12 | The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults. The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Selected items from five of the domains were used to create three components: upper extremity; basic lower extremity; and complex lower extremity activities. The component score of "complex lower extremity activities" was the most important in this questionnaire, had a transformed score range from 0 to 100, higher values indicated less functional limitations in performing tasks. | Dosed analysis set included all participants enrolled in the study who received PF-06838435 infusion. Here, "Number of Participants Analyzed" signifies number of participants evaluable for this outcome measure and "Number Analyzed" signifies number of participants evaluable for specified rows of respective arms. | Posted | Mean | 95% Confidence Interval | Units on a scale | Baseline, Month 12 |
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| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Adverse Events of Special Interest | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Positive Neutralizing Antibody (nAb) to Adeno-associated Virus Vector (AAV) and Anti-Drug Antibody (ADA) | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | ABR for Total Bleeds (Treated and Untreated) Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | ABR for Treated Bleeds Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | AIR of Exogenous Factor IX Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | FIX: C Level Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Annualized Factor IX Consumption Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | ABR for Spontaneous and Traumatic, and Untreated Bleeds Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Joint Health as Measured by the HJHS Instrument Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Baseline, 6 years | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Target Joint Bleeds Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Haem A QoL Physical Health Domain Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2031 | Baseline, Maximum up to 6 years (Week 312) after PF-06838435 infusion | Participants | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in HAL Complex Lower Extremity Activities Component Score Through the Study | Results would be posted at secondary completion date. | Not Posted | Mar 2030 | Baseline, 6 years | Participants |
Day 1 up to analysis data cut-off 16-Nov-2022 (approximately 3 years 3 months)
Same event may appear as both non-SAE and a serious AE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study. All participants enrolled in the study and who received PF-06838435 infusion.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PF-06838435 | All participants enrolled in the study and who received PF-06838435 infusion and have no significant interruption of efficacy measurement | 0 | 45 | 7 | 45 | 37 | 45 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Duodenal ulcer | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Duodenal ulcer haemorrhage | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Drug-induced liver injury | Hepatobiliary disorders | MedDRA v25.1 | Non-systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| COVID-19 pneumonia | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Pilonidal disease | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Alcohol poisoning | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
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| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA v25.1 | Non-systematic Assessment |
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| Coagulation factor IX level decreased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| Hypokalaemia | Metabolism and nutrition disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Hepatic function abnormal | Hepatobiliary disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Hepatic steatosis | Hepatobiliary disorders | MedDRA v25.1 | Non-systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA v25.1 | Non-systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| Hepatic enzyme increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| SARS-CoV-2 test positive | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| Transaminases increased | Investigations | MedDRA v25.1 | Non-systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA v25.1 | Non-systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA v25.1 | Non-systematic Assessment |
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C0371004 lead-in ongoing study: did not involve any investigational intervention; designed to collect bleed, infusion data while participants were on their prophylaxis FIX therapy for a minimum of 6 months; data served as within participant comparison group for C0371002; participant flow and AEs are missing as the study has not concluded yet (some participants in the C0371004 are ongoing and supporting a different study NCT04370054) and data will be provided at conclusion of the lead-in study.
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 7, 2022 | Nov 1, 2023 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| D006467 | Hemophilia A |
| D006402 | Hematologic Diseases |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
Not provided
Not provided
| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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Eligible participants enrolled in the current study C0371002 and who received PF-06838435 infusion on Day 1. This reporting arm served as experimental arm.
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Eligible participants enrolled in the current study C0371002 and who received PF-06838435 infusion on Day 1. This reporting arm served as experimental arm.
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Eligible participants enrolled in the current study C0371002 and who received PF-06838435 infusion on Day 1. This reporting arm served as experimental arm.
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| OG001 |
| PF-06838435 |
Eligible participants enrolled in the current study C0371002 and who received PF-06838435 infusion on Day 1. This reporting arm served as experimental arm. |
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| Title | Denominators | Categories | ||||
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| Without treated bleeds |
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| Without treated and untreated bleeds |
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