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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-003660-31 | EudraCT Number |
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Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PF-06741086 | Experimental | Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PF-06741086 | Drug | 300 milligrams(mg) subcutaneous (sc) loading dose followed by 150 mg sq once weekly (qw). 300 mg sc qw is prescribed for participants who meet dose escalation criteria. |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized bleeding rate (ABR) of treated bleeding events | Derived for each subject for each treatment period by using the following formula: ABR = number of bleeds requiring treatments/ (days on treatment period / 365.25) | Through Observational Phase (6months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Incidence and severity of thrombotic events | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months | |
| Incidence of anti drug antibody [ADA] against PF-06741086 | Throughout Active Treatment Phase (12 months) | |
| Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086 | Throughout Active Treatment Phase (12 months) | |
| Incidence and severity of injection site reaction | Throughout Active Treatment Phase (12 months) | |
| Number of participants with clinically significant changes from baseline in physical exam | From Baseline through Observation and Active Treatment (approximately 18 months) | |
| Incidence of clinically significant laboratory value abnormalities | From Screening through Observation and Active Treatment (approximately 18 months) | |
| Incidence of severe hypersensitivity and anaphylactic reactions | From Screening through Observational and Active Treatment (approximately 18 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of joint bleeds | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months | |
| Incidence of spontaneous bleeds | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
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Inclusion Criteria
Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:
Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:
Exclusion Criteria
Previous or current treatment for and/or history of coronary artery diseases, venous or arterial thrombosis or ischemic disease
Known planned surgical procedure during the planned study period.
Known hemostatic defect other than hemophilia A or B.
Abnormal renal or hepatic function
Current unstable liver or biliary disease
Abnormal hematologic parameters
Other acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator,
Current routine prophylaxis with bypassing agent or non-coagulation non-factor- replacement therapy, or any previous treatment with a gene therapy product for treatment of hemophilia (participants treated with prophylaxis using bypassing agents or who had prior treatment with non-factor products may be considered on a case-by-case basis).
Regular, concomitant therapy with immunomodulatory drugs
- Ongoing or planned use of immune tolerance induction during the Observational Phase or Active Treatment Phase, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention during the Active Treatment Phase
Previous exposure to PF 06741086 during participation in studies B7841002 and B7841003.
Participation in other studies involving investigational drug(s) or investigational vaccines within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry and/or during study participation.
CD4 cell count ≤200/uL if human immunodeficiency virus (HIV)-positive
Screening ECG that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.
Individuals with hypersensitivity or an allergic reaction to hamster protein or other components of the study intervention.
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.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| USF Health Morsani Center For Advanced Healthcare | Tampa | Florida | 33612 | United States | ||
| University of Iowa |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41351884 | Derived | Matino D, Acharya SS, Taylor CT, Sun P, Agathon D, Raje S, Gould T, Palladino A, Mahlangu J, Lissitchkov T, Todorova M, Chan A, Carcao M, Sun J, Yang R, Wu R, Jin C, Zeng X, Boban A, Bilic E, Frenzel L, Fung Chan GC, Kong Li C, Apte S, Choraria N, Chistolini A, Marchesini E, Peyvandi F, Fujii T, Matsushita T, Kaneda M, Lyu CJ, Park YS, Kim S, Martinez LV, Adrian JM, Wali Y, Al Khabori M, Kurtov I, Alzahrani H, Zaher G, Micic D, Miljic P, Kostic G, Vucic M, Djurdjevic P, Hidalgo OB, Porras JG, Jimenez-Yuste V, Calvo Villas JM, Lopez Fernandez MF, Albayrak C, Okan V, Balkan C, Sahin F, Antmen A, Unal E, Visweshwar N, Sharathkumar A, Kruse-Jarres R. Efficacy and safety of marstacimab prophylaxis in hemophilia A/B with inhibitors: results from the phase 3 BASIS trial. Blood. 2026 Feb 26;147(9):920-931. doi: 10.1182/blood.2025031065. | |
| 40608864 |
| 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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This is a one way Cross-Over Prevention study with 1 Arm that has No masking.
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| Incidence of adverse events and serious adverse events | From screening through Observation and Active treatment (approximately 18 months) |
| Number of participants with clinically significant changes from baseline in vital signs | From Baseline through Observation and Active Treatment (approximately 18 months) |
| Incidence and severity of thromboticangiopathy | Throughout Active Treatment Phase (12 months) |
| Incidence of intravascular coagulation/consumption coagulopathy | Throughout Active Treatment Phase (12 months) |
| Incidence of target joint bleeds | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Incidence of total bleeds (treated and untreated) | Through Observational and Active Treatment Phases (18 Months) |
| Change from baseline in the Hemophilia Joint Health Score (HJHS) | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Change from baseline in (Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Change from baseline in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Change from baseline in Hemophilia Adult Activities List (HAL) | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Change from baseline in Hemophilia Pediatric Activities List (PedHAL) | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Patient Global Impression of Change - Hemophilia (PGIC-H) | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L) | Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months |
| Iowa City |
| Iowa |
| 52242 |
| United States |
| Northwell Health HTC | New Hyde Park | New York | 11040 | United States |
| Washington Institute for Coagulation | Seattle | Washington | 98101 | United States |
| National Specialized Hospital for the Active Treatment of Hematological Diseases - EAD, Sofia | Sofia | 1756 | Bulgaria |
| UMHAT "Prof.Dr. Stoyan Kirkovich" | Stara Zagora | 6003 | Bulgaria |
| McMaster University Medical Centre - Hamilton Health Sciences | Hamilton | Ontario | L8N 3Z5 | Canada |
| McMaster Children's Hospital | Hamilton | Ontario | L8S 4K1 | Canada |
| McMaster University | Hamilton | Ontario | L8S 4K1 | Canada |
| The Hospital For Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| Nanfang Hospital, Southern Medical University | Guangzhou | Guangdong | 510515 | China |
| The Affiliated Hospital of Guizhou Medical University | Guiyang | Guizhou | 550004 | China |
| Jiangxi Provincial People's Hospital | Nanchang | Jiangxi | 306113 | China |
| Institute of Hematology, Chinese Academy of Medical Sciences | Tianjin | Tianjin Municipality | 300020 | China |
| Beijing Children's Hospital, Capital Medical University | Beijing | 100045 | China |
| Klinicki bolnicki centar Zagreb | Zagreb | 10000 | Croatia |
| Hôpital Necker Enfants Malades | Paris | 75015 | France |
| Prince of Wales Hospital | Hong Kong | Hong Kong |
| Queen Mary Hospital | Hong Kong | Hong Kong |
| Nirmal Hospital Pvt, Ltd | Surat | Gujarat | 395002 | India |
| Sahyadri Clinical Research and Development Center | Pune | Maharashtra | 411004 | India |
| Sahyadri Super Speciality Hospital | Pune | Maharashtra | 411004 | India |
| Christian Medical College | Vellore | Tamil Nadu | 632004 | India |
| Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico | Milan | Milan | 20122 | Italy |
| Università degli Studi di Perugia, Azienda Ospedaliera di Perugia, Ospedale Santa Maria della | Perugia | PERUGIA | 06156 | Italy |
| Università degli Studi di Roma "Sapienza"-Policlinico Umberto I | Roma | RM | 00161 | Italy |
| Nagoya University Hospital - Transfusion Medicine | Nagoya | Aichi-ken | 466-8560 | Japan |
| Hiroshima University Hospital | Hiroshima | Hiroshima | 734-8551 | Japan |
| Sapporo Tokushukai Hospital | Sapporo | Hokkaido | 004-0041 | Japan |
| Saitama Medical University Hospital | Iruma-gun | Saitama | 350-0495 | Japan |
| Hospital Universitario "Dr Jose Eleuterio Gonzalez" | Monterrey | Nuevo León | 64460 | Mexico |
| Centro Multidisciplinario para el Desarrollo Especializado de la Inv. Clínica en Yucatán, S.C.P. | Mérida | Yucatán | 97130 | Mexico |
| Sultan Qaboos University Hospital | Muscat | 123 | Oman |
| FGBOU VO "Samara State Medical University" of MoH of Russia | Samara | 443079 | Russia |
| King Abdulaziz University Hospital | Jeddah | 21589 | Saudi Arabia |
| King Faisal Specialist Hospital & Research Center | Riyadh | Saudi Arabia |
| Clinical Center of Serbia | Belgrade | 11000 | Serbia |
| Institute for Mother and Child healthcare "Dr Vukan Cupic" | Belgrade | 11000 | Serbia |
| Clinical Center Kragujevac | Kragujevac | 34000 | Serbia |
| Clinical Center Nis | Niš | 18000 | Serbia |
| Charlotte Maxeke Johannesburg Academic Hospital | Johannesburg | Gauteng | 2193 | South Africa |
| Kyungpook National University Hospital | Daegu | 41944 | South Korea |
| Severance Hospital, Yonsei University Health System | Seoul | 03722 | South Korea |
| Kyung Hee University Hospital At Gangdong | Seoul | 05278 | South Korea |
| Hospital Universitario A Coruna | A Coruña | A CORUNA | 15006 | Spain |
| Hospital Universitario Vall d´Hebron | Barcelona | BARCELONA | 08035 | Spain |
| Hospital Universitario La Paz | Madrid | Madrid | 28046 | Spain |
| Hospital Universitario de Salamanca | Salamanca | SALAMANCA | 37007 | Spain |
| Hospital Universitario Miguel Servet | Zaragoza | ZARAGOZA | 50009 | Spain |
| ChangHua Christian Hospital | Changhua | CHANGHUA COUNTY | 500 | Taiwan |
| Taichung Veterans General Hospital | Taichung | 40705 | Taiwan |
| Acibadem Adana Hospital | Adana | 01130 | Turkey (Türkiye) |
| Hacettepe University Medical Faculty | Ankara | 06230 | Turkey (Türkiye) |
| Gazi University Health Research and Practice Center Gazi Hospital | Ankara | 06500 | Turkey (Türkiye) |
| Akdeniz University Medical Faculty | Antalya | 07060 | Turkey (Türkiye) |
| Gaziantep University Sahinbey Research and Training Hospital | Gaziantep | 27310 | Turkey (Türkiye) |
| Istanbul University Oncology Institute | Istanbul | 34093 | Turkey (Türkiye) |
| Ege University Medical Faculty | Izmir | 35040 | Turkey (Türkiye) |
| Dr. Behcet Uz Child Diseases Surgery Education and Research Hospital | Izmir | 35210 | Turkey (Türkiye) |
| Erciyes University Medical Faculty | Kayseri | 38039 | Turkey (Türkiye) |
| Ondokuz Mayıs University Medical Faculty | Samsun | 55280 | Turkey (Türkiye) |
| Karadeniz Technical University Medical Faculty | Trabzon | 61080 | Turkey (Türkiye) |
| Derived |
| Matino D, Palladino A, Taylor CT, Hwang E, Raje S, Nayak S, McDonald R, Acharya SS, Mahlangu J, Jimenez-Yuste V, Choraria N, Yang R, Li CK, Al-Khabori M, Wali Y, Morales Adrian J, Park YS, Zulfikar OB, Teeter J. Marstacimab prophylaxis in hemophilia A/B without inhibitors: results from the phase 3 BASIS trial. Blood. 2025 Oct 2;146(14):1654-1663. doi: 10.1182/blood.2024027468. |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Apr 8, 2026 | Apr 28, 2026 | 69 | ||
| Jun 4, 2026 | Jun 29, 2026 | 70 |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D002836 | Hemophilia B |
| D006470 | Hemorrhage |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| 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 |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000656192 | marstacimab |
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