Hemophilia APhase 3NCT03938792
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
A Phase 3 study testing PF-06741086 in people with Hemophilia A and Hemophilia B, run by Pfizer at 62 sites in 20 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 12 to 74 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- PF-06741086
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 188 participants
- Where
- 62 sitesin 20 countries
- Study dates
- 9 Mar 2020to 29 Apr 2025
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 74 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B with a minimum weight of 35 kg at screening.
- Participant or legally authorized representative, or participant's caregiver capable of giving signed informed consent (or minor assent, when applicable).
- Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:
- No detectable or documented history of inhibitors
- Participants on FVIII/FIX routine prophylaxis who have demonstrated at least 80% compliance with scheduled prophylaxis regimen during 6 months prior to enrollment and are willing to continue to receive routine prophylaxis treatment with FVIII/FIX replacement during the Observational Phase.
- Participants with on-demand treatment regimen with ≥6 acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months period prior to enrollment and willing to continue to receive on demand treatment during the Observational Phase.
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- Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:
- Documentation of current high titer inhibitor (≥5 BU/mL) or current low titer inhibitor (<5 BU/mL) refractory to FVIII or FIX replacement and with FVIII or FIX recovery <60% of expected within previous 6 months prior to enrolment into the Observational Phase
- Hemophilia A participants with on-demand treatment regimen with ≥6 bleeding episodes or hemophilia B participants with ≥4 bleeding episodes (spontaneous or traumatic) necessitating treatment with bypass factor during the 6 months prior to Enrollment into Observational Phase and willing to continue to receive on-demand treatment during the Observational Phase.
- Participants who have documented inhibitors while on factor-replacement therapy but who do not meet the quantitative inhibitor criteria described in the prior bullet at the time of Screening (eg, participant with a previously documented high-titer inhibitor (≥5 BU/mL) and whose condition precludes re-challenge with FVIII or FIX replacement) may be considered for eligibility on a case-by-case basis with prior agreement from the Pfizer Medical Monitor
- Participants who meet the bleeding criteria noted above and who are on routine prophylaxis (defined as treatment by IV injection of bypass factor to prevent bleeding) and have demonstrated at least 80% compliance with scheduled prophylaxis regimen during the 6 months prior to enrollment, may be considered for eligibility on a case-by-case basis with discussion and agreement from the Pfizer medical monitor.
Who cannot join
- 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
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- 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.
Trial locations and contacts
62 sites in 20 countries.
62 sites in 20 countries
Turkey (Türkiye)11 sites
Acibadem Adana Hospital
Adana, 01130
Hacettepe University Medical Faculty
Ankara, 06230
Gazi University Health Research and Practice Center Gazi Hospital
Ankara, 06500
Akdeniz University Medical Faculty
Antalya, 07060
Gaziantep University Sahinbey Research and Training Hospital
Gaziantep, 27310
Istanbul University Oncology Institute
Istanbul, 34093
Ege University Medical Faculty
Izmir, 35040
Dr. Behcet Uz Child Diseases Surgery Education and Research Hospital
Izmir, 35210
Erciyes University Medical Faculty
Kayseri, 38039
Ondokuz Mayıs University Medical Faculty
Samsun, 55280
Karadeniz Technical University Medical Faculty
Trabzon, 61080
China5 sites
Beijing Children's Hospital, Capital Medical University
Beijing, 100045
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550004
Jiangxi Provincial People's Hospital
Nanchang, Jiangxi, 306113
Institute of Hematology, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 300020
Spain5 sites
Hospital Universitario A Coruna
A Coruña, 15006
Hospital Universitario Vall d´Hebron
Barcelona, 08035
Hospital Universitario La Paz
Madrid, 28046
Hospital Universitario de Salamanca
Salamanca, 37007
Hospital Universitario Miguel Servet
Zaragoza, 50009
United States4 sites
University of Iowa
Iowa City, Iowa, 52242
Northwell Health HTC
New Hyde Park, New York, 11040
Washington Institute for Coagulation
Seattle, Washington, 98101
USF Health Morsani Center For Advanced Healthcare
Tampa, Florida, 33612
Canada4 sites
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5
McMaster Children's Hospital
Hamilton, Ontario, L8S 4K1
McMaster University
Hamilton, Ontario, L8S 4K1
The Hospital For Sick Children
Toronto, Ontario, M5G 1X8
India4 sites
Sahyadri Clinical Research and Development Center
Pune, Maharashtra, 411004
Sahyadri Super Speciality Hospital
Pune, Maharashtra, 411004
Nirmal Hospital Pvt, Ltd
Surat, Gujarat, 395002
Christian Medical College
Vellore, Tamil Nadu, 632004
Japan4 sites
Hiroshima University Hospital
Hiroshima, 734-8551
Saitama Medical University Hospital
Iruma-gun, Saitama, 350-0495
Nagoya University Hospital - Transfusion Medicine
Nagoya, Aichi-ken, 466-8560
Sapporo Tokushukai Hospital
Sapporo, Hokkaido, 004-0041
Serbia4 sites
Clinical Center of Serbia
Belgrade, 11000
Institute for Mother and Child healthcare "Dr Vukan Cupic"
Belgrade, 11000
Clinical Center Kragujevac
Kragujevac, 34000
Clinical Center Nis
Niš, 18000
Italy3 sites
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Milan, 20122
Università degli Studi di Perugia, Azienda Ospedaliera di Perugia, Ospedale Santa Maria della
Perugia, 06156
Università degli Studi di Roma "Sapienza"-Policlinico Umberto I
Roma, RM, 00161
South Korea3 sites
Kyungpook National University Hospital
Daegu, 41944
Severance Hospital, Yonsei University Health System
Seoul, 03722
Kyung Hee University Hospital At Gangdong
Seoul, 05278
Bulgaria2 sites
National Specialized Hospital for the Active Treatment of Hematological Diseases - EAD, Sofia
Sofia, 1756
UMHAT "Prof.Dr. Stoyan Kirkovich"
Stara Zagora, 6003
Hong Kong2 sites
Prince of Wales Hospital
Hong Kong
Queen Mary Hospital
Hong Kong
Mexico2 sites
Centro Multidisciplinario para el Desarrollo Especializado de la Inv. Clínica en Yucatán, S.C.P.
Mérida, Yucatán, 97130
Hospital Universitario "Dr Jose Eleuterio Gonzalez"
Monterrey, Nuevo León, 64460
Saudi Arabia2 sites
King Abdulaziz University Hospital
Jeddah, 21589
King Faisal Specialist Hospital & Research Center
Riyadh
Taiwan2 sites
ChangHua Christian Hospital
Changhua, Changhua County, 500
Taichung Veterans General Hospital
Taichung, 40705
Russia1 site
FGBOU VO "Samara State Medical University" of MoH of Russia
Samara, 443079
South Africa1 site
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, Gauteng, 2193
About this study
Official title: An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A Participants With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity ≤2%) With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis
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.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- PF-06741086
- Keywords
- Factor VIII, Factor IX, Inhibitors, Anti-Tissue Factor Pathway Inhibitor (TFPI), Subcutaneous (sc), Prophylaxis, On-Demand, BASIS, Inhibitor, SC, Subcutaneous, Injection, On demand, aTFPI, Anti-TFPI, Severe hemophilia, Severe bleeding, Hemophilia, PF-06741086, PF 06741086
- Medical subject headings
- Hemophilia A, Hemophilia B, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PF-06741086Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| PF-06741086 | Experimental | Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen. Receives: Drug: PF-06741086 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Model-Based Annualized Bleeding Rate (ABR) of Treated Bleeding Events: Inhibitor Cohort (Participants With Prior OD Therapy at OP)
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With Prior OD Therapy at OP)
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With RP at OP)
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Number of Participants With Adverse Events (AEs): Inhibitor and Non-Inhibitor Cohort
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs included both serious AEs and all other AEs.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Serious Adverse Events (SAEs): Inhibitor and Non-Inhibitor Cohort
AE: any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE: any untoward medical occurrence at any dose that resulted in any of the following outcomes: death; life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect; or that was considered as an important medical event.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Thrombotic Events: Inhibitor and Non-Inhibitor Cohort
Thrombotic events are when a blood clot (thrombus) forms in a blood vessel in the arm, leg, lung, or head and can be life-threatening. A thrombus can occur in veins (venous thrombosis) or arteries (arterial thrombosis).
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Thrombotic Microangiopathy: Inhibitor and Non-Inhibitor Cohort
Thrombotic microangiopathy: pathological state where micro-vessels are occluded by platelet rich thrombi leading to thrombocytopenia (low platelets) and microangiopathic haemolytic anaemia (red blood cell destruction) and potential end organ damage.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Disseminated Intravascular Coagulation/ Consumption Coagulopathy: Inhibitor and Non-Inhibitor Cohort
Disseminated intravascular coagulation is characterized by widespread clotting in small blood vessels, and consumption of platelets and clotting factors by the clots, leading to bleeding.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Anti-drug Antibodies (ADA) and Neutralizing Antibodies (NAb) Against PF- 06741086: Inhibitor and Non-Inhibitor Cohort
ADA positive: A participant with >=1 treatment induced or treatment-boosted ADA response. NAb positive: An ADA positive participant with >=1 treatment induced or treatment boosted NAb response.
Measured: During prophylaxis treatment in ATP (12 months)
Number of Participants With Transient and Persistent ADA and NAb Against PF-06741086: Inhibitor and Non-Inhibitor Cohort
Persistent ADA: participant with treatment-induced or treatment-boosted ADA detected at 2 or more times during treatment including any follow-up, where first and last ADA positive samples separated by >=16 weeks. Transient ADA: treatment-induced or treatment-boosted ADA detected at only 1 time during treatment or follow-up. Persistent NAb: NAb-positive participant with first and last positive NAb samples detected >=16 weeks posttreatment, irrespective of any negative samples in between. Transient NAb: NAb-positive participant with (1) treatment-induced/treatment-boosted NAb sample detected at only 1 time posttreatment or (2) treatment-induced/treatment-boosted NAb samples detected at 2 or more times where first and last positive samples separated by <16 weeks, and participant's last sample was NAb or ADA negative.
Measured: During prophylaxis treatment in ATP (12 months)
Number of Participants With Injection Site Reactions (ISRs): Inhibitor and Non-Inhibitor Cohort
ISR included: injection site haematoma, injection site pain, injection site bruising, injection site erythema, injection site induration, injection site oedema, Injection site pruritus and Injection site swelling. ISR graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 where grade 1: tenderness with or without associated symptoms (warmth, erythema, itching), grade 2: pain, lipodystrophy, edema, phlebitis, grade 3: ulceration or necrosis, severe tissue damage, operative intervention indicated, grade 4: life-threatening consequences, urgent intervention indicated and grade 5: death. In this outcome measure only categories with non-zero values reported.
Measured: During prophylaxis treatment in ATP (12 months)
Number of Participants With Clinically Significant Changes in Physical Examinations: Inhibitor and Non-Inhibitor Cohort
Physical examination included assessments of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. Height and weight were also measured and recorded. Clinical significance in physical examinations was determined by investigator.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Clinically Significant Changes in Vital Signs: Inhibitor and Non-Inhibitor Cohort
Vital signs included temperature, pulse rate, respiratory rate, and blood pressure. Blood pressure and pulse rate were measured in a supine position using completely automated device after at least 5 minutes of rest for the participant in a quiet setting without distractions. Respiratory rate was measured by observing and counting the respirations of the participant for 30 seconds and multiplied by 2. Clinical significance of vital signs was determined by investigator.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters: Inhibitor and Non-Inhibitor Cohort
Hematology included: hemoglobin; leukocytes; neutrophils; and platelets. Chemistry included: potassium; aspartate aminotransferase; creatinine; alkaline phosphatase; total bilirubin; albumin; calcium corrected, estimated decreased; sodium; and calcium. Clinical significance of laboratory parameters was determined by investigator.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Severe/ Systematic Hypersensitivity and Anaphylactic Reactions: Inhibitor and Non-Inhibitor Cohort
Systemic hypersensitivity is a complex immune response where the immune system reacts excessively to an antigen, often leading to severe allergic reactions, including widespread symptoms (which may affect multiple organs), such as rash, swelling of your face, lips, mouth, or tongue, trouble breathing, wheezing, dizziness, fainting, fast heartbeat, pounding in your chest or sweating. Anaphylaxis is a severe, potentially fatal, systemic allergic reaction that occurs suddenly after contact with an allergy causing substance.
Measured: During prophylaxis treatment in ATP (12 months)
Secondary outcomes (18)
Model-Based ABR of Joint Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Joint bleed: 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. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Spontaneous Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as the number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, the days on treatment ended at the last dosing date + 6 days. Spontaneous bleed: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Target Joint Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Target joint: a major joint into which repeated bleeds occurred. Joint bleed: bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of following: pain or unusual sensation in joint, palpable swelling, and warmth of the skin over joint. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Total Bleeds: Inhibitor and Non-Inhibitor Cohort
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication). Untreated bleed: If a bleed was untreated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication), it was considered as an untreated bleed. Total bleeds: Treated bleeds + untreated bleeds. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
Measured: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Change From Baseline in Hemophilia Joint Health Score (HJHS) at Month 6: Non-Inhibitor Cohort
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling [0-3], duration of swelling [0-1], muscle atrophy [0-2], crepitus on motion [0-2], flexion loss [0-3], extension loss [0-3], joint pain [0-2], and strength [0-4]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in HJHS at Month 6: Inhibitor Cohort
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling [0-3], duration of swelling [0-1], muscle atrophy [0-2], crepitus on motion [0-2], flexion loss [0-3], extension loss [0-3], joint pain [0-2], and strength [0-4]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) Total Score and Physical Health Domain at Month 6: Non-Inhibitor Cohort, Participants >=17 Years
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (>=17 years of age) with hemophilia. It contained 46 items with 10 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; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Haem-A-QoL Total Score and Physical Health Domain at Month 6: Inhibitor Cohort, Participants >=17 Years
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (>=17 years of age) with hemophilia. It contained 46 items with 10 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; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Quality of Life Questionnaire for Children (Haemo-QoL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years
Haemo-QoL assessed health-related QoL in adolescent participants (12 to <17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Haemo-QoL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years
Haemo-QoL assessed health-related QoL in adolescent participants (12 to <17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Activities List (HAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants >=17 Years
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (>=17 years of age). 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. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in HAL Total Score at Month 6: Inhibitor Cohort, Participants >=17 Years
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (>=17 years of age). 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. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Pediatric Hemophilia Activities List (pedHAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to <17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in pedHAL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to <17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Patient Global Impression of Change-Hemophilia (PGIC-H) at Month 6: Non-Inhibitor Cohort
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
Measured: Month 6
PGIC-H at Month 6: Inhibitor Cohort
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
Measured: Month 6
Change From Baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L) Index and VAS Scores at Month 6: Non-Inhibitor Cohort
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in EQ-5D-5L Index and VAS Scores at Month 6: Inhibitor Cohort
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
Measured: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2019
- Study start9 Mar 2020
- Primary completion29 Apr 2025
- Study completion29 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Crossover
- Model description
- This is a one way Cross-Over Prevention study with 1 Arm that has No masking.
- Masking
- None (open label)
- Enrolment
- 188 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03938792
- Sponsor's protocol ID
- B7841005
- Other IDs
- 2018-003660-31, 2018
- Status last verified
- Jul 2026
- First submitted
- 2 May 2019
- Last updated
- 14 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Publications
Nayak S, Suzuki A, Ravva P, Raje S. Clinical Pharmacokinetics and Pharmacodynamics of Marstacimab, an Anti-tissue Factor Pathway Inhibitor Monoclonal Antibody, in Adolescent and Adult Participants with Hemophilia. Clin Pharmacokinet. 2026 Sep 4. doi: 10.1007/s40262-026-01695-5. Online ahead of print.
PubMed 42698050Matino 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.
PubMed 41351884Matino 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.
PubMed 40608864