PF-06741086 Multiple Dose Study in Severe Hemophilia

• PF-06741086 (Biological): PF-06741086 subcutaneous (SC) injection
• PF-06741086 (Biological): PF-06741086 SC injection
• PF-06741086 (Biological): PF-06741086 SC injection
• PF-06741086 (Biological): PF-06741086 SC injection

Primary outcomes

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) [Study Day 1 to Day 113 Visit]: An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and non-serious AEs. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Severe TEAEs were TEAEs that interfered significantly with participants' usual function. Treatment-related TEAEs were determined by the investigator.
• Number of Participants Discontinued From Study Due to TEAEs [Study Day 1 to Day 113 Visit]: An AE was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator.
• Number of Participants With Abnormal Laboratory Findings-Hematology [Baseline to Study Day 113 Visit]: Hematology evaluation included: hemoglobin, hematocrit, erythrocytes, platelets, leukocytes, lymphocytes, neutrophils, basophils, eosinophils and monocytes. Predefined criteria for hemoglobin and hematocrit: <0.8*lower limit of normal (LLN) or <0.8*Baseline(Baseline <1.0*LLN); for platelets: <100,000*10\^3/mm\^3 or <= 0.77*Baseline (Baseline <1.0*LLN).
• Number of Participants With Abnormal Laboratory Findings-Clinical Chemistry [Baseline to Study Day 113]: Clinical chemistry evaluation included bilirubin, direct and indirect bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase, alkaline phosphatase, protein, albumin, urea nitrogen, creatinine, urate, triglycerides, sodium, potassium, chloride, calcium, bicarbonate, glucose, creatine kinase, troponin I, cholesterol and fibrinogen.
• Number of Participants With Abnormal Laboratory Findings-Urinalysis [Baseline to Study Day 113 Visit]: Urinalysis included: pH, urine glucose, ketones, urine protein, urine hemoglobin, urobilinogen, urine bilirubin, nitrite, leukocyte esterase, urine erythrocytes, urine leukocytes and bacteria.
• Change From Baseline for Globulin by Dose Cohort [Baseline, Study Day 8, 15, 22, 29, 57, 85 and 113.]: Blood samples were obtained to determine globulin level in serum, total globulin was derived as total protein other than albumin.
• Change From Baseline for Prothrombin International Normalized Ratio (PT/INR) by Dose Cohort [Baseline, Study Day 8, 15, 22, 29, 57, 85 and 113.]: Blood samples were obtained to evaluate this ratio. The prothrombin time (PT) is a test that helps evaluate your ability to appropriately form blood clots. The international normalized ratio (INR) is a calculation based on results of a PT that is used to monitor individuals who are being treated with the blood-thinning medication (anticoagulant) warfarin (Coumadin®).
• Change From Baseline for Activated Partial Thromboplastin Time (aPTT) by Dose Cohort [Baseline, Study Day 8, 15, 22, 29, 57, 85 and 113.]: The activated partial thromboplastin time (aPTT) is a screening test that helps evaluate a person's ability to appropriately form blood clots. It measures the number of seconds it takes for a clot to form in a sample of blood after substances (reagents) are added. Blood sample were obtained to evaluate aPTT.
• Change From Baseline for Fibrinogen by Dose Cohort [Baseline, Study Day 8, 15, 22, 29, 57, 85 and 113.]: Fibrinogen is a protein, specifically a clotting factor (factor I), that is essential for proper blood clot formation. Blood samples were obtained to evaluate the amount of fibrinogen.
• Change From Baseline for Antithrombin III by Dose Cohort [Baseline, Study Day 8, 15, 22 and 29.]: Antithrombin (AT) is a protein produced by the liver that helps regulate blood clot formation (i.e., a naturally-occurring mild blood thinner). Blood samples were collected to measure the activity (function) and the amount (quantity) of antithrombin in an individual's blood is used to evaluate the person for excessive blood clotting.
• Change From Baseline for Troponin I by Dose Cohort [Baseline, Study Day 8, 15, 22, 29, 57 and 85.]: Blood samples were collected to measure the level of cardiac-specific troponin I in the blood to help detect heart injury.
• Number of Participants With Vital Signs Data Meeting Pre-specified Criteria [Baseline to Study Day 113 Visit]: Criteria for potentially clinically important findings in vital signs data were defined as: 1) supine systolic blood pressure (BP): value <90 mm Hg or change >=30 mm Hg increase; 2) Supine diastolic BP: value <50 mm Hg or change >=20 mm Hg increase; 3) Supine pulse rate: value <40 beats/min or >120 beats/min.
• Number of Participants With Electrocardiogram (ECG) Change Meeting Pre-specified Criteria [Baseline to Study Day 29 Visit.]: Criteria for potentially clinically important changes in ECG were defined as: PR interval baseline >200 msec and increase of >=25%; PR interval baseline <=200 msec and increase of >=50%; QRS interval increase of >=50%. Only the number of participants meeting pre-defined criteria was reported below.
• Number of Participants With Clinically Significant Changes in Physical Examination Findings [Baseline to Study Day 113 Visit]: Physical examination included head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. Clinical significance was judged by the investigator.
• Number of Participants With Infusion and Injection Site Reactions [Baseline to Study Day 113 Visit]: Infusion and injection site reactions included: injection site bruising, injection site erythema, injection site haemorrhage, injection site induration, injection site pain, injection site pruritus, injection site swelling and injection site warmth. Grade of severity was defined as follows: Mild: Transient or mild discomfort (< 48 hours); no medical intervention/therapy required. Moderate: Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required. Severe: Marked limitation in activity, some assistance usually required; medical intervention/therapy required, hospitalizations possible.

Secondary outcomes

• Annualized Bleeding Rate (ABR) [Pre-treatment: within 6 months prior to study enrollment; On-study: Day 1 to 9 days after the last dose (Day 78)]: Pre-treatment ABR = number of bleeding episodes within 6 months prior to study enrollment (total number of bleeding episodes in hemophilia history CRF) × 2; On-study ABR = number of bleeding episodes occurred within 9 days after the last dose / (\[last dose date + 9 - first dose date + 1\] / 365.25) The historical On Demand group was constructed using the following internal Pfizer studies: ReFacto AF 3082B2-4432 (B1831004), BeneFIX B1821010, and BeneFIX 3090A1-400 (B1821004). Participants who were on On Demand treatment in B1831004, as well as data from the On Demand period in B1821004 and B1821010 were used to construct the historical On Demand group. The resulting dataset were further filtered to match the key inclusion/exclusion criteria of Study B7841002 based on age and factor activity (18 <=age <=65 and factor activity <=1%).
• Plasma PF-06741086 Concentrations [pre-dose on Study Day 1, 24hours (h), 72h post Study Day 1 dosing, pre-dose on Study Day 8, 15 and 22, pre-dose on Study Day 29, 24h, 96h post Study Day 29 dosing, pre-dose on Study Day 57, 168h, 840h post Study Day 57 dosing]: Plasma PF-06741086 concentrations were analyzed using a validated, sensitive and specific electrochemiluminescence (ECL) method.
• Area Under the Concentration-time Profile From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) of PF-06741086 [Pre-dose on Day 1, 24 and 96 hours post Day 1 dosing]: AUClast was calculated by linear/Log trapezoidal method.
• Maximum Plasma Concentration (Cmax) of PF-06741086 [Pre-dose on Day 1, 24 and 96 hours post Day 1 dosing, pre-dose on Day 29, 24 and 96 hours post Day 29 dosing]: Cmax was observed directly from data.
• Lowest Concentration Observed During the Dosing Interval (Cmin) of PF-06741086 [Pre-dose on Day 1, 24 and 96 hours post Day 1 dosing, pre-dose on Day 29, 24 and 96 hours post Day 29 dosing]: Cmin was observed directly from data.
• Time to Reach Maximum Plasma Concentration (Tmax) of PF-06741086 [Pre-dose on Day 1, 24 and 96 hours post Day 1 dosing, pre-dose on Day 29, 24 and 96 hours post Day 29 dosing]: Tmax was observed directly from data as time of first occurrence.
• Area Under the Serum Concentration-time Curve Over the Dosing Interval Tau (AUCtau) of PF-06741086 [Pre-dose on Day 29, 24 and 96 hours post Day 29 dosing]: The dosing interval tau was 1 week. AUCtau was obtained by linear/log trapezoidal method.
• Apparent Clearance After Oral Dose (CL/F) of PF-06741086 [Pre-dose on Day 29, 24 and 96 hours post Day 29 dosing]: CL/F was calculated by dose/AUCtau.
• Change From Baseline in Total Tissue Factor Pathway Inhibitor (TFPI) [Baseline, Study Day 2, 4, 8, 15, 22, 29, 30, 33, 57, 85 and 113]: Total amount of tissue factor pathway inhibitor (TFPI) (bound and unbound) in plasma. TFPI is a protease inhibitor which acts as an antagonist of the extrinsic coagulation pathway via inhibition of tissue factor activated coagulation factor VII (FVIIa) and activated factor X (FXa). Human plasma samples were analyzed for total TFPI concentrations using a validated, sensitive and specific high-performance liquid chromatography tandem mass spectrometric method (LC-MS/MS). Mixed model repeated measures (MMRM) was used to analyze the change from baseline on TFPI.
• Change From Baseline in Thrombin Generation (TGA) Lag Time [Baseline, Study Day 2, 4, 8, 15, 22, 29, 30, 33, 57, 85 and 113]: An ex vivo pharmacodynamic measure of thrombin generation (initiation of thrombin generation), the lag time is the time needed to form the first traces of thrombin.
• Change From Baseline in Thrombin Generation (TGA) Peak [Baseline, Study Day 2, 4, 8, 15, 22, 29, 30, 33, 57, 85 and 113]: An ex vivo pharmacodynamic measure of thrombin generation (initiation of thrombin generation). The peak represents the highest thrombin concentration that can be generated. There may be patients who reach the peak faster or slower than others and this may represent hyper- or hypocoagulability, respectively.
• Change From Baseline in Endogenous Thrombin Generation (TGA) Potential [Baseline, Study Day 2, 4, 8, 15, 22, 29, 30, 33, 57, 85 and 113]: An ex vivo pharmacodynamic measure of thrombin generation. The endogenous TGA potential represents the total amount of active thrombin formed during thrombin generation and the peak height the maximal amount of thrombin formed.
• Change From Baseline in Prothrombin Fragments 1 + 2 [Baseline, Study Day 2, 4, 8, 15, 22, 29, 30, 33, 57, 85 and 113]: An in vivo pharmacodynamic measure of thrombin generation (prothrombin cleavage). Prothrombin fragment 1+2 (F 1+2) is the amino terminus fragment of the prothrombin molecule. It is a polypeptide with a half-life of approximately 90 minutes. F 1+2 is released from prothrombin when prothrombin is converted to thrombin by the prothrombinase complex.
• Change From Baseline in D-Dimer [Baseline, Study Day 2, 4, 8, 15, 22, 29, 30, 33, 57, 85 and 113]: An in vivo pharmacodynamic measure of thrombin generation (fibrin degradation). D-dimer is a fibrin degradation product, a small protein fragment present in the blood after a blood clot is degraded by fibrinolysis. D-dimer is one of the protein fragments produced when a blood clot gets dissolved in the body. It is normally undetectable or detectable at a very low level unless the body is forming and breaking down blood clots. Then, its level in the blood can significantly rise.
• Change From Baseline in Dilute Prothrombin Time [Baseline, Study Day 2, 4, 8, 15, 22, 29, 30, 33, 57, 85 and 113]: An ex vivo pharmacodynamic measure of thrombin generation (via extrinsic pathway). Clotting time is measured using a dilute prothrombin time reagent consisting of a unique formulation of relipidated recombinant tissue factor and calcium.
• Number of Participants Who Tested Positive for Anti-PF-06741086 Antibody (ADA) [Baseline up to Study Day 113]: Human plasma ADA samples were analyzed for the detection of anti PF-06741086 antibodies by using semi-quantitative electrochemiluminescence (ECL) method. The criterion for positive result of ADA samples was ADA titer >=1.53. Treatment induced are negative prior to dosing and become positive during/after dosing. Treatment boosted are positive prior to dosing but titer increases during/after dosing.
• Number of Participants Who Tested Positive for Neutralizing Antibody (NAb) [Baseline up to Study Day 113]: Human plasma NAb samples were analyzed for the presence or absence of NAb to PF-06741086 using semi-quantitative electrochemiluminescence (ECL) method. Treatment induced are negative prior to dosing and become positive during/after dosing. Treatment boosted are positive prior to dosing but titer increases during/after dosing.

Sex: Male, ages: 18 Years to 64 Years

Inclusion Criteria:

* Severe hemophilia A or B (Factor VIII or Factor IX activity ≤ 1%), including patients with inhibitors to Factor VIII or Factor IX
* Episodic (on-demand) treatment regimen prior to screening
* At least 6 acute bleeding episodes during the 6-month period prior to screening

Exclusion Criteria:

* Known coronary artery, thrombotic, or ischemic disease
* Currently receiving treatment for acute bleeding episodes with APCC and cannot substitute treatment with rFVIIa

Participant flow

Outcome results

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 7; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 4; PF-06741086 450 mg SC QW Non-Inhibitor 6; PF-06741086 300 mg SC QW Inhibitor 4; Overall PF-06741086 300 mg SC 11; Total 21
• Number of Participants Discontinued From Study Due to TEAEs (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 1; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 1; Overall PF-06741086 300 mg SC 1; Total 2
• Number of Participants With Abnormal Laboratory Findings-Hematology (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 0; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 0; Overall PF-06741086 300 mg SC 0; Total 0
• Number of Participants With Abnormal Laboratory Findings-Clinical Chemistry (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 0; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 0; Overall PF-06741086 300 mg SC 0; Total 0
• Number of Participants With Abnormal Laboratory Findings-Urinalysis (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 0; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 0; Overall PF-06741086 300 mg SC 0; Total 0
• Change From Baseline for Globulin by Dose Cohort (Primary), Mean, gram/liter: PF-06741086 300 mg SC QW Non-Inhibitor -0.3 +/- 3.04; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor -1.7 +/- 3.93; PF-06741086 450 mg SC QW Non-Inhibitor -1.3 +/- 2.73; PF-06741086 300 mg SC QW Inhibitor 0.4 +/- 2.51; Overall PF-06741086 300 mg SC 0.1 +/- 2.70; Total -0.7 +/- 3.01
• Change From Baseline for Prothrombin International Normalized Ratio (PT/INR) by Dose Cohort (Primary), Mean, Ratio: PF-06741086 300 mg SC QW Non-Inhibitor -0.03 +/- 0.049; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor -0.02 +/- 0.041; PF-06741086 450 mg SC QW Non-Inhibitor 0.05 +/- 0.138; PF-06741086 300 mg SC QW Inhibitor 0.03 +/- 0.138; Overall PF-06741086 300 mg SC 0.00 +/- 0.104; Total 0.01 +/- 0.102
• Change From Baseline for Activated Partial Thromboplastin Time (aPTT) by Dose Cohort (Primary), Mean, seconds: PF-06741086 300 mg SC QW Non-Inhibitor 8.49 +/- 17.017; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 10.63 +/- 11.634; PF-06741086 450 mg SC QW Non-Inhibitor 11.03 +/- 15.467; PF-06741086 300 mg SC QW Inhibitor -12.26 +/- 7.082; Overall PF-06741086 300 mg SC -1.89 +/- 16.512; Total 3.98 +/- 16.079
• Change From Baseline for Fibrinogen by Dose Cohort (Primary), Mean, milligram/deciliter: PF-06741086 300 mg SC QW Non-Inhibitor -49.7 +/- 28.72; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor -59.7 +/- 38.20; PF-06741086 450 mg SC QW Non-Inhibitor -3.5 +/- 24.16; PF-06741086 300 mg SC QW Inhibitor -54.4 +/- 59.58; Overall PF-06741086 300 mg SC -52.1 +/- 45.00; Total -42.6 +/- 44.14
• Change From Baseline for Antithrombin III by Dose Cohort (Primary), Mean, Percentage of activity of AT in plasma: PF-06741086 300 mg SC QW Non-Inhibitor 2.1 +/- 5.18; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor -1.8 +/- 7.05; PF-06741086 450 mg SC QW Non-Inhibitor 3.7 +/- 7.58; PF-06741086 300 mg SC QW Inhibitor -7.3 +/- 9.32; Overall PF-06741086 300 mg SC -2.6 +/- 8.74; Total -1.0 +/- 8.24
• Change From Baseline for Troponin I by Dose Cohort (Primary), Mean, nanogram/milliliter: PF-06741086 300 mg SC QW Non-Inhibitor 0 +/- 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 0 +/- 0; PF-06741086 450 mg SC QW Non-Inhibitor 0 +/- 0; PF-06741086 300 mg SC QW Inhibitor 0 +/- 0; Overall PF-06741086 300 mg SC 0 +/- 0; Total 0 +/- 0
• Number of Participants With Vital Signs Data Meeting Pre-specified Criteria (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 0; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 0; Overall PF-06741086 300 mg SC 0; Total 0
• Number of Participants With Electrocardiogram (ECG) Change Meeting Pre-specified Criteria (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 0; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 0; Overall PF-06741086 300 mg SC 0; Total 0
• Number of Participants With Clinically Significant Changes in Physical Examination Findings (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 0; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 0; Overall PF-06741086 300 mg SC 0; Total 0
• Number of Participants With Infusion and Injection Site Reactions (Primary), Count of participants, Participants: PF-06741086 300 mg SC QW Non-Inhibitor 3; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 1; PF-06741086 450 mg SC QW Non-Inhibitor 0; PF-06741086 300 mg SC QW Inhibitor 1; Overall PF-06741086 300 mg SC 4; Total 5
• Annualized Bleeding Rate (ABR) (Secondary), Mean, Bleeding episodes per year: PF-06741086 300 mg SC QW Non-Inhibitor 23.00 +/- 7.457; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 14.67 +/- 1.633; PF-06741086 450 mg SC QW Non-Inhibitor 20.33 +/- 10.838; PF-06741086 300 mg SC QW Inhibitor 17.33 +/- 3.011; Overall PF-06741086 300 mg SC 20.17 +/- 6.177; Total 18.83 +/- 7.100 (p 0.0002)
• Plasma PF-06741086 Concentrations (Secondary), Mean, nanogram/milliliter
• Area Under the Concentration-time Profile From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) of PF-06741086 (Secondary), Geometric mean, nanogram*hour/milliliter: PF-06741086 300 mg SC QW Non-Inhibitor 1818000 +/- 79; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 2675000 +/- 41; PF-06741086 450 mg SC QW Non-Inhibitor 2806000 +/- 37; PF-06741086 300 mg SC QW Inhibitor 2495000 +/- 40; Overall PF-06741086 300 mg SC 2130000 +/- 61
• Maximum Plasma Concentration (Cmax) of PF-06741086 (Secondary), Geometric mean, nanogram/milliliter: PF-06741086 300 mg SC QW Non-Inhibitor 14880 +/- 70; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 19480 +/- 42; PF-06741086 450 mg SC QW Non-Inhibitor 23070 +/- 37; PF-06741086 300 mg SC QW Inhibitor 19680 +/- 51; Overall PF-06741086 300 mg SC 17110 +/- 61
• Lowest Concentration Observed During the Dosing Interval (Cmin) of PF-06741086 (Secondary), Geometric mean, nanogram/milliliter: PF-06741086 300 mg SC QW Non-Inhibitor 7980 +/- 112; PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor 13040 +/- 43; PF-06741086 450 mg SC QW Non-Inhibitor 15660 +/- 44; PF-06741086 300 mg SC QW Inhibitor 11140 +/- 41; Overall PF-06741086 300 mg SC 9429 +/- 78
• 12 more outcome measures not shown.

Adverse events

Time frame: 113 days

Serious adverse events by term

Other adverse events by term

• Results publication: 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. PMID 42698050
• Results publication: Mahlangu JN, Lamas JL, Morales JC, Malan DR, Salek SZ, Wang M, Boggio LN, Hegemann I, Mital A, Cardinal M, Zhu T, Sun P, Arkin S. A phase 1b/2 clinical study of marstacimab, targeting human tissue factor pathway inhibitor, in haemophilia. Br J Haematol. 2023 Jan;200(2):229-239. doi: 10.1111/bjh.18420. Epub 2022 Aug 23. PMID 35999026

• Study Protocol (PDF, 2017-10-02)
• Statistical Analysis Plan (PDF, 2017-10-20)

• Pfizer CT.gov Call Center, Study director, Pfizer

NIH grants: 2016