CClinicalTrials.gg
Sign In
CompletedNCT01439971Updated May 12, 2017Results posted

Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia

A Phase 1 interventional study of PF-05280602 and PF-05280602 in Hemophilia A, sponsored by Catalyst Biosciences. Completed at 23 sites in 8 countries. Open to male participants aged 18 Years to 64 Years. Per ClinicalTrials.gov, last updated 2017-05-12.

Sponsored by Catalyst Biosciences · Phase 1, Interventional, and Basic science

Phase
Phase 1
Study type
Interventional
Enrollment
29
Allocation
Non-randomized
Ages
18 Years to 64 Years
Sex
Male
01

Study summary

This study hypothesizes that the study drug, PF-05280602 (at the selected doses) will be safe to administer to subjects with severe Hemophilia A or B with or without inhibitors and will demonstrate evidence of hemostatic activity. This is supported by the preclinical findings in hemophilic animal models.

02

Conditions studied

  • Hemophilia A

Browse trials for

Keywords

  • Phase 1
  • Safety
  • Pharmacokinetic
  • Pharmacodynamic
03

In context

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 29 is close to the median of 28 across 512 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Catalyst Biosciences is the lead sponsor of 8 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 64 Years
Sexes eligible
Male
Accepts healthy volunteers
No

Inclusion criteria

  • Male subjects 18 to \<65 years old with severe hemophilia A or B with or without inhibitors to FVIII or FIX.
  • Subjects is willing and able to comply with the mandatory washout periods prior to screening and prior to dosing and through 48 hours post dosing. At screening this includes a washout of FIX for 96 hours and FVIII for 72 houts. At dosing this includes a washout of FIX for 96 hours and FVIII and other hemostatic agents for 72 hours through 48 hours post dosing.
  • Subjects must agree and commit to using a a highly effective method of birth control from the time of screening through four weeks after study drug administration.

Exclusion criteria

Exclusion Criteria:

  • Presence of a bleeding disorder in addition to hemophilia A or B.
  • Regular, concomitant therapy with immunomodulating drugs (eg, intravenous immunoglobulin, and routine systemic corticosteroids).
  • History of coronary artery disease, thrombolic disease or diagnosis of prothrombic disorder.
05

Study design

Phase
Phase 1
Primary purpose
Basic science
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
29 participants (actual)

Study arms

  • Experimental
    1

    Biological: PF-05280602

  • Experimental
    2

    Biological: PF-05280602

  • Experimental
    3

    Biological: PF-05280602

  • Experimental
    4

    Biological: PF-05280602

  • Experimental
    5

    Biological: PF-05280602

Interventions

  • BiologicalPF-05280602

    0.5 micrograms per kilogram of PF-05280602, IV infusion, single dose

  • BiologicalPF-05280602

    4.5 micrograms per kilogram of PF-05280602, IV infusion, single dose

  • BiologicalPF-05280602

    9.0 micrograms per kilogram of PF-05280602, IV infusion, single dose

  • BiologicalPF-05280602

    18.0 micrograms per kilogram of PF-05280602, IV infusion, single dose

  • BiologicalPF-05280602

    30.0 micrograms per kilogram of PF-05280602, IV infusion, single dose

06

What researchers measure

Primary outcomes

  1. Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

    Supine blood pressure (BP) was measured with the participant's arm supported at the level of the heart, and recorded to the nearest millimeters of mercury (mmHg) after 5 minutes of rest. The same arm (preferably the dominant arm) was to be used throughout the study.

    Time frame: Baseline, Day 2, Day 3, and Day 15

  2. Change From Baseline in Body Weight

    Time frame: Baseline, Day 2, Day 3, and Day 15

  3. Change From Baseline in Body Temperature

    Body temperature was measured by mouth (oral) or ear (tympanic). A temperature greater than 38.5 degree Celsius was considered a fever.

    Time frame: Baseline, Day 2, Day 3, and Day 15

  4. Change From Baseline in Respiration Rate

    Respiration rate measured as respirations per minute (resp/min).

    Time frame: Baseline, Day 2, Day 3, and Day 15

  5. Change From Baseline in Supine Pulse Rate

    Change from baseline is the vital sign value at Day 2, Day 3, and Day 15 minus vital sign value at baseline.

    Time frame: Baseline, Day 2, Day 3, and Day 15

  6. Number of Participants With Changes Since Previous Physical Examination

    Physical examinations were conducted by a physician, trained physician's assistant, or nurse practitioner. A complete physical examination included head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, genitourinary, gastrointestinal, musculoskeletal, and neurological systems. The limited or abbreviated physical examination focused on general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms.

    Time frame: Baseline (Day 0), Day 1, Day 2, Day 3, Day 15

  7. Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Findings

    ECG findings of potential clinical concern were: PR interval greater than or equal to (\>=)300 milliseconds (msec), \>=25% increase from baseline for baseline values \>200 msec, \>=50% increase from baseline for baseline values less than or equal to (\<=)200 msec; QRS complex \>=140 msec or \>=50% increase from baseline; QTcF interval (Fridericia's correction) \>=450 msec or \>=30 msec increase from baseline.

    Time frame: Baseline through Day 15

  8. Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Withdrawals Due to AEs (Except Hemophilia AEs)

    An AE was any untoward medical occurrence in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to Day 15 that were absent before treatment or that worsened relative to pre-treatment state. AEs included both SAEs and non-SAEs.

    Time frame: Baseline through Day 60

  9. Number of Participants With Treatment-Emergent Hemophilia AEs and Withdrawals Due to Hemophilia AEs

    Hemophilia AEs included spontaneous (no known contributing factor) and traumatic (known or presumed contributing factor/reason) bleeding episodes.

    Time frame: Baseline through Day 60

  10. Number of Treatment-Emergent AEs and SAEs by Severity (Except Hemophilia AEs)

    AE severity were graded as mild, moderate, or severe. Mild severity AEs do not interfere with the participant's usual function. Moderate AEs interfere to some extent with the participant's usual function. Severe AEs interfere significantly with the participant's usual function.

    Time frame: Baseline through Day 60

  11. Number of Treatment-Emergent Hemophilia AEs by Severity

    Mild severity AEs do not interfere with the participant's usual function. Moderate AEs interfere to some extent with the participant's usual function. Severe AEs interfere significantly with the participant's usual function.

    Time frame: Baseline through Day 60

  12. Number of Participants With Treatment-Emergent Abnormal Troponin-T Levels by Magnitude

    Troponin-T is a cardiac marker for the evaluation of possible cardiovascular injury. Troponin-T levels of potential clinical concern are values \>1.5 times the upper limit of normal (1.5X ULN) or \>=2.5X ULN.

    Time frame: Baseline through Day 15

  13. Number of Participants With Treatment-Emergent Abnormal Anti-Thrombin III (ATIII) Levels by Magnitude

    ATIII is a protein in the blood that blocks abnormal blood clots from forming. Low levels of ATIII can cause abnormal blood clots. ATIII levels of potential clinical concern are values \<1X LLN and \>1X ULN.

    Time frame: Baseline through Day 3

  14. Number of Participants With Treatment-Emergent Abnormal Tissue Factor Pathway Inhibitor (TFPI) Levels by Magnitude

    TFPI is a polypeptide that can regulate blood coagulation. TFPI levels of potential clinical concern are values \<1X LLN and \>1X ULN.

    Time frame: Baseline through Day 3

  15. Number of Participants With Treatment-Emergent Laboratory Test Abnormalities (Normal Baseline)

    The following laboratory parameters were analyzed: hematology (hemoglobin, hematocrit, red blood cell \[RBC\] count, platelets, leukocytes, total neutrophils, eosinophils, basophils, lymphocytes, monocytes); chemistry (total bilirubin, direct bilirubin, indirect bilirubin, aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\], alkaline phosphatase, creatinine, blood urea nitrogen \[BUN\], glucose, uric acid, sodium, potassium, chloride, bicarbonate, calcium, albumin, total protein, creatine kinase); urinalysis (urine white blood cell \[WBC\], urine RBC); other (troponin T).

    Time frame: Baseline through Day 15

  16. Number of Participants With Clinically Significant Laboratory Abnormalities Meeting Stopping Criteria

    Clinically significant findings for stopping rules are: hemoglobin \<8 grams/deciliter (g/dL) or \>20% decrease from normal baseline; WBC \>20,000 cells/mm\^3 or \<1,500 decrease with normal baseline; platelets \<100,000/mm\^3 or \>33% decrease from baseline; total bilirubin \>1.5X ULN; AST or ALT \>2.5X ULN; alkaline phosphatase \>3X ULN; creatinine \>1.5X baseline; BUN \>31.0 mg/dL; glucose \<0.6 or \>1.5X reference range; uric acid \> ULN; sodium \>150 or \<130 mEq/L; potassium \>5.5 or \<3.0 mEq/L; calcium \>11.5 or \<8.0 mg/dL; albumin \<2.0 g/L; total protein \<5.0 g/L; positive D-dimer at Day 15; PT prolonged by 3 seconds above baseline; ATIII \< LLN and \>20% decrease from baseline; troponin-T values above the reference range; fibrinogen \<0.75X LLN or \>25% decrease from baseline.

    Time frame: Baseline through Day 15

  17. Number of Participants With Positive Immune Response (Anti-Drug Antibodies [ADA], PF-05280602 Inhibitor, Factor VIIa Inhibitor, Factor VII Inhibitor, and Depletion of Factor VII Activity)

    Assays for the determination of a positive immune response was performed. An antibody immune response was defined as a confirmed post-treatment positive ELISA result in combination with a negative baseline sample ELISA result. Positive antibody immune responses to PF-05280602 by ELISA was evaluated for cross reactivity to NovoSeven RT and to Factor VII.

    Time frame: Baseline through Day 60

Secondary outcomes

  1. Maximum Observed Plasma Concentration (Cmax)

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  2. Area Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast)

    Area under the plasma concentration time-curve from zero to the last measured concentration (AUClast)

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  3. Terminal Elimination Half-Life (t1/2)

    t1/2 is the time measured for the plasma concentration to decrease by one half.

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  4. Incremental Recovery (IncRec)

    IncRec is the maximum rise in plasma concentration per administered dose.

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  5. Area Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf)

    AUCinf is area under the plasma concentration-time curve from time 0 extrapolated to infinite time.

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  6. Mean Residence Time (MRT)

    MRT is AUMCinf/AUCinf, where AUMC is the area under the first moment curve.

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  7. Volume of Distribution at Steady State (Vss)

    Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Steady state volume of distribution (Vss) is the apparent volume of distribution at steady-state.

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  8. Clearance (CL)

    Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood (rate at which a drug is metabolized or eliminated by normal biological processes). Clearance obtained after intravenous infusion dose is influenced by the fraction of the dose absorbed.

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  9. Time to Reach Maximum Observed Plasma Concentration (Tmax)

    Time frame: Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)

  10. Maximum Mean Decrease From Baseline in Prothrombin Time (PT)

    PT measures how long it takes blood to clot. Maximum mean decrease from baseline at any time point was reported.

    Time frame: Baseline through Day 15

  11. Maximum Mean Decrease From Baseline in Activated Partial Thromboplastin Time (aPTT)

    aPTT is a blood test that characterizes blood coagulation. Maximum mean decrease from baseline at any time point was reported.

    Time frame: Baseline through Day 15

  12. Maximum Mean Increase From Baseline in Thrombin Anti-Thrombin (TAT) Complexes

    TAT complex is a parameter of coagulation and fibrinolysis. The normal reference range of values for TAT is 1 to 4.1 mcg/L. Elevated TAT concentrations may signify predisposition to thrombosis. Maximum mean increase from baseline at any time point was reported.

    Time frame: Baseline through Day 3

  13. Maximum Mean Increase From Baseline in Prothrombin Fragments 1+2

    Prothrombin fragment 1+2 is a coagulation factor, released when prothrombin is cleaved by activated Factor X. Elevated plasma levels of prothrombin fragment 1+2 indicate high risk of thrombosis. Maximum mean increase from baseline at any time point was reported.

    Time frame: Baseline through Day 3

  14. Maximum Mean Increase From Baseline in D-Dimers

    D-dimer is an indicator of fibrin formation and its subsequent lysis and is a useful biomarker representing overall activation of blood coagulation. Maximum mean increase from baseline at any time point was reported.

    Time frame: Baseline through Day 15

  15. Maximum Mean Increase From Baseline in Endogenous Thrombin Potential (ETP)

    ETP was evaluated using a Thrombin Generation Assay (TGA), a validated automated ex-vivo assay that measures the ability of plasma to generate thrombin. Thrombin generation curves are generated and calculated using dedicated software. ETP is the area under the thrombin generation curve and represents the total amount of generated thrombin. Maximum mean increase from baseline at any time point was reported.

    Time frame: Baseline through Day 3

  16. Maximum Mean Decrease From Baseline in Thrombin Generation Lag Time

    The lag time is defined as the time to reach one sixth of the peak height and is a measure of the initiation phase. It is equivalent to the clotting time. Maximum mean decrease from baseline at any time point was reported.

    Time frame: Baseline through Day 3

  17. Maximum Mean Increase From Baseline in Peak Thrombin Generation

    The peak height is defined as the maximum thrombin concentration produced. Maximum mean increase from baseline at any time point was reported.

    Time frame: Baseline through Day 3

07

Results

Posted Apr 6, 2017
Limitations and caveats
The protocol was amended to a starting dose of 4.5 mcg/kg. The positive anti-PF-05280602 antibody was cross-reactive with NovoSeven and native Factor VII and the weak positive immune response at Day 60 may represent a false-positive test result.

Participant flow

Participant flow — Overall Study
MilestonePF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Started17867
Received treatment16666
Completed16666
Not completed01201
Withdrew: Required number in cohort dosed01201

Outcome measures

PrimaryChange From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)

Supine blood pressure (BP) was measured with the participant's arm supported at the level of the heart, and recorded to the nearest millimeters of mercury (mmHg) after 5 minutes of rest. The same arm (preferably the dominant arm) was to be used throughout the study.

Time frame:
Baseline, Day 2, Day 3, and Day 15
Reported as:
Mean · mmHg
Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
mmHgPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
SBP: Baseline122.0 ± NA130.2 ± 7.14120.2 ± 25.41124.2 ± 7.68125.5 ± 8.53
SBP: Change at Day 20.0 ± NA-8.7 ± 8.24-0.2 ± 14.41-3.0 ± 6.51-1.5 ± 5.58
SBP: Change at Day 35.0 ± NA-7.0 ± 6.992.2 ± 19.30-5.5 ± 7.660.0 ± 8.58
SBP: Change at Day 15-10.0 ± NA-5.5 ± 12.184.7 ± 18.00-4.5 ± 14.921.8 ± 11.84
DBP: Baseline78.0 ± NA73.7 ± 6.3570.5 ± 10.9775.8 ± 4.6777.0 ± 6.87
DBP: Change at Day 22.0 ± NA-2.8 ± 6.432.7 ± 6.28-0.8 ± 5.12-3.3 ± 5.50
DBP: Change at Day 3-3.0 ± NA-6.5 ± 6.573.3 ± 6.56-2.2 ± 8.114.3 ± 10.41
DBP: Change at Day 15-9.0 ± NA-3.5 ± 11.416.0 ± 13.11-4.3 ± 11.15-4.5 ± 5.65
PrimaryChange From Baseline in Body Weight
Time frame:
Baseline, Day 2, Day 3, and Day 15
Reported as:
Mean · kilograms (kg)
Change From Baseline in Body Weight
kilograms (kg)PF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Baseline72.7 ± NA75.0 ± 12.6578.7 ± 10.7378.0 ± 11.3173.4 ± 6.88
Change at Day 2-0.9 ± NA-1.0 ± 0.98-0.6 ± 0.72-0.8 ± 1.31-0.4 ± 0.83
Change at Day 3-0.8 ± NA-0.6 ± 0.85-0.5 ± 0.60-0.8 ± 1.39-0.3 ± 0.56
Change at Day 15-0.5 ± NA0.4 ± 1.090.6 ± 1.550.6 ± 1.260.2 ± 0.59
PrimaryChange From Baseline in Body Temperature

Body temperature was measured by mouth (oral) or ear (tympanic). A temperature greater than 38.5 degree Celsius was considered a fever.

Time frame:
Baseline, Day 2, Day 3, and Day 15
Reported as:
Mean · degree Celcius
Change From Baseline in Body Temperature
degree CelciusPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Baseline36.1 ± NA36.5 ± 0.2936.5 ± 0.3436.6 ± 0.3036.4 ± 0.43
Change at Day 20.4 ± NA-0.4 ± 0.62-0.1 ± 0.300.1 ± 0.340.2 ± 0.29
Change at Day 30.3 ± NA-0.2 ± 0.600.1 ± 0.15-0.0 ± 0.410.2 ± 0.13
Change at Day 150.3 ± NA0.1 ± 0.390.1 ± 0.360.1 ± 0.240.2 ± 0.60
PrimaryChange From Baseline in Respiration Rate

Respiration rate measured as respirations per minute (resp/min).

Time frame:
Baseline, Day 2, Day 3, and Day 15
Reported as:
Mean · resp/min
Change From Baseline in Respiration Rate
resp/minPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Baseline12.0 ± NA15.0 ± 2.7615.2 ± 2.0415.7 ± 1.9716.0 ± 2.83
Change at Day 20.0 ± NA0.5 ± 1.760.3 ± 3.502.2 ± 2.560.5 ± 1.22
Change at Day 30.0 ± NA0.2 ± 3.370.3 ± 3.501.7 ± 1.630.2 ± 2.23
Change at Day 152.0 ± NA0.7 ± 4.32-0.2 ± 2.141.2 ± 3.49-0.3 ± 1.97
PrimaryChange From Baseline in Supine Pulse Rate

Change from baseline is the vital sign value at Day 2, Day 3, and Day 15 minus vital sign value at baseline.

Time frame:
Baseline, Day 2, Day 3, and Day 15
Reported as:
Mean · beats per minute (bpm)
Change From Baseline in Supine Pulse Rate
beats per minute (bpm)PF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Baseline62.0 ± NA64.8 ± 6.2169.3 ± 10.6560.7 ± 10.6966.5 ± 8.22
Change at Day 21.0 ± NA-1.3 ± 3.563.7 ± 6.898.8 ± 7.882.0 ± 5.69
Change at Day 30.0 ± NA1.3 ± 6.802.5 ± 7.8910.7 ± 5.653.3 ± 3.56
Change at Day 152.0 ± NA8.3 ± 12.684.5 ± 12.6810.2 ± 12.223.0 ± 5.33
PrimaryNumber of Participants With Changes Since Previous Physical Examination

Physical examinations were conducted by a physician, trained physician's assistant, or nurse practitioner. A complete physical examination included head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, genitourinary, gastrointestinal, musculoskeletal, and neurological systems. The limited or abbreviated physical examination focused on general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms.

Time frame:
Baseline (Day 0), Day 1, Day 2, Day 3, Day 15
Reported as:
Number · participants
Number of Participants With Changes Since Previous Physical Examination
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Baseline00000
Day 100000
Day 2NA0100
Day 300110
Day 1501010
PrimaryNumber of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Findings

ECG findings of potential clinical concern were: PR interval greater than or equal to (\>=)300 milliseconds (msec), \>=25% increase from baseline for baseline values \>200 msec, \>=50% increase from baseline for baseline values less than or equal to (\<=)200 msec; QRS complex \>=140 msec or \>=50% increase from baseline; QTcF interval (Fridericia's correction) \>=450 msec or \>=30 msec increase from baseline.

Time frame:
Baseline through Day 15
Reported as:
Number · participants
Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Findings
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
PR Interval >=300 msec0 ± NA0 ± 7.140 ± 25.410 ± 7.680 ± 8.53
QRS Complex >=140 msec00000
QTcF Interval >=450 msec00000
PR Interval >=25/50% Increase From Baseline00000
QRS Complex >=50% Increase From Baseline00000
QTcF Interval 30-<60 msec Increase From Baseline01000
QTcF Interval >=60 msec Increase From Baseline00000
PrimaryNumber of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Withdrawals Due to AEs (Except Hemophilia AEs)

An AE was any untoward medical occurrence in a participant who received study drug. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to Day 15 that were absent before treatment or that worsened relative to pre-treatment state. AEs included both SAEs and non-SAEs.

Time frame:
Baseline through Day 60
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Withdrawals Due to AEs (Except Hemophilia AEs)
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
AEs14432
SAEs00000
Withdrawals Due to AEs00000
PrimaryNumber of Participants With Treatment-Emergent Hemophilia AEs and Withdrawals Due to Hemophilia AEs

Hemophilia AEs included spontaneous (no known contributing factor) and traumatic (known or presumed contributing factor/reason) bleeding episodes.

Time frame:
Baseline through Day 60
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Hemophilia AEs and Withdrawals Due to Hemophilia AEs
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Hemophilia AEs11112
Hemophilia SAEs00000
Withdrawals Due to Hemophilia AEs00000
PrimaryNumber of Treatment-Emergent AEs and SAEs by Severity (Except Hemophilia AEs)

AE severity were graded as mild, moderate, or severe. Mild severity AEs do not interfere with the participant's usual function. Moderate AEs interfere to some extent with the participant's usual function. Severe AEs interfere significantly with the participant's usual function.

Time frame:
Baseline through Day 60
Reported as:
Number · adverse events
Number of Treatment-Emergent AEs and SAEs by Severity (Except Hemophilia AEs)
adverse eventsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Mild AEs26571
Moderate AEs11521
Severe AEs00000
PrimaryNumber of Treatment-Emergent Hemophilia AEs by Severity

Mild severity AEs do not interfere with the participant's usual function. Moderate AEs interfere to some extent with the participant's usual function. Severe AEs interfere significantly with the participant's usual function.

Time frame:
Baseline through Day 60
Reported as:
Number · adverse events
Number of Treatment-Emergent Hemophilia AEs by Severity
adverse eventsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Mild Hemophilia AEs10000
Moderate Hemophilia AEs11212
Severe Hemophilia AEs00000
PrimaryNumber of Participants With Treatment-Emergent Abnormal Troponin-T Levels by Magnitude

Troponin-T is a cardiac marker for the evaluation of possible cardiovascular injury. Troponin-T levels of potential clinical concern are values \>1.5 times the upper limit of normal (1.5X ULN) or \>=2.5X ULN.

Time frame:
Baseline through Day 15
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Abnormal Troponin-T Levels by Magnitude
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
<1X lower limit of normal (LLN)00000
>1X ULN00000
>1.5X ULN00000
>=2.5X ULN00000
PrimaryNumber of Participants With Treatment-Emergent Abnormal Anti-Thrombin III (ATIII) Levels by Magnitude

ATIII is a protein in the blood that blocks abnormal blood clots from forming. Low levels of ATIII can cause abnormal blood clots. ATIII levels of potential clinical concern are values \<1X LLN and \>1X ULN.

Time frame:
Baseline through Day 3
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Abnormal Anti-Thrombin III (ATIII) Levels by Magnitude
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
<1X LLN00002
>1X ULN00010
PrimaryNumber of Participants With Treatment-Emergent Abnormal Tissue Factor Pathway Inhibitor (TFPI) Levels by Magnitude

TFPI is a polypeptide that can regulate blood coagulation. TFPI levels of potential clinical concern are values \<1X LLN and \>1X ULN.

Time frame:
Baseline through Day 3
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Abnormal Tissue Factor Pathway Inhibitor (TFPI) Levels by Magnitude
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
<1X LLN01200
>1X ULN10001
PrimaryNumber of Participants With Treatment-Emergent Laboratory Test Abnormalities (Normal Baseline)

The following laboratory parameters were analyzed: hematology (hemoglobin, hematocrit, red blood cell \[RBC\] count, platelets, leukocytes, total neutrophils, eosinophils, basophils, lymphocytes, monocytes); chemistry (total bilirubin, direct bilirubin, indirect bilirubin, aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\], alkaline phosphatase, creatinine, blood urea nitrogen \[BUN\], glucose, uric acid, sodium, potassium, chloride, bicarbonate, calcium, albumin, total protein, creatine kinase); urinalysis (urine white blood cell \[WBC\], urine RBC); other (troponin T).

Time frame:
Baseline through Day 15
Reported as:
Number · participants
Number of Participants With Treatment-Emergent Laboratory Test Abnormalities (Normal Baseline)
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Number of Participants With Treatment-Emergent Laboratory Test Abnormalities (Normal Baseline)00212
PrimaryNumber of Participants With Clinically Significant Laboratory Abnormalities Meeting Stopping Criteria

Clinically significant findings for stopping rules are: hemoglobin \<8 grams/deciliter (g/dL) or \>20% decrease from normal baseline; WBC \>20,000 cells/mm\^3 or \<1,500 decrease with normal baseline; platelets \<100,000/mm\^3 or \>33% decrease from baseline; total bilirubin \>1.5X ULN; AST or ALT \>2.5X ULN; alkaline phosphatase \>3X ULN; creatinine \>1.5X baseline; BUN \>31.0 mg/dL; glucose \<0.6 or \>1.5X reference range; uric acid \> ULN; sodium \>150 or \<130 mEq/L; potassium \>5.5 or \<3.0 mEq/L; calcium \>11.5 or \<8.0 mg/dL; albumin \<2.0 g/L; total protein \<5.0 g/L; positive D-dimer at Day 15; PT prolonged by 3 seconds above baseline; ATIII \< LLN and \>20% decrease from baseline; troponin-T values above the reference range; fibrinogen \<0.75X LLN or \>25% decrease from baseline.

Time frame:
Baseline through Day 15
Reported as:
Number · participants
Number of Participants With Clinically Significant Laboratory Abnormalities Meeting Stopping Criteria
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Hemoglobin00000
Platelets00000
WBC00000
Prothrombin Time00000
Total Bilirubin00000
Total Protein00000
Albumin00000
AST00000
ALT00000
Alkaline Phosphatase00000
BUN00000
Creatinine01000
Uric Acid00000
Sodium00000
Potassium00100
Calcium00000
Glucose00110
Fibrinogen00001
Troponin-T00000
Anti-Thrombin III00000
D-Dimer00001
PrimaryNumber of Participants With Positive Immune Response (Anti-Drug Antibodies [ADA], PF-05280602 Inhibitor, Factor VIIa Inhibitor, Factor VII Inhibitor, and Depletion of Factor VII Activity)

Assays for the determination of a positive immune response was performed. An antibody immune response was defined as a confirmed post-treatment positive ELISA result in combination with a negative baseline sample ELISA result. Positive antibody immune responses to PF-05280602 by ELISA was evaluated for cross reactivity to NovoSeven RT and to Factor VII.

Time frame:
Baseline through Day 60
Reported as:
Number · participants
Number of Participants With Positive Immune Response (Anti-Drug Antibodies [ADA], PF-05280602 Inhibitor, Factor VIIa Inhibitor, Factor VII Inhibitor, and Depletion of Factor VII Activity)
participantsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
ADA00010
PF-05280602 Inhibitor00000
Factor VIIa Inhibitor00000
Factor VII Inhibitor00000
Depletion of Factor VII Activity00000
SecondaryMaximum Observed Plasma Concentration (Cmax)
Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Geometric mean · ng/mL
Maximum Observed Plasma Concentration (Cmax)
ng/mLPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Observed Plasma Concentration (Cmax)1.68 ± NA84.78 ± 20183.1 ± 28496.2 ± 14814.8 ± 23
SecondaryArea Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast)

Area under the plasma concentration time-curve from zero to the last measured concentration (AUClast)

Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Geometric mean · ng*hr/mL
Area Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast)
ng*hr/mLPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Area Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast)3.82 ± NA300.7 ± 15638.9 ± 211557 ± 192788 ± 31
SecondaryTerminal Elimination Half-Life (t1/2)

t1/2 is the time measured for the plasma concentration to decrease by one half.

Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Mean · hour
Terminal Elimination Half-Life (t1/2)
hourPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Terminal Elimination Half-Life (t1/2)NA ± NA3.533 ± 0.5603.637 ± 0.8033.303 ± 0.6383.580 ± 0.564
SecondaryIncremental Recovery (IncRec)

IncRec is the maximum rise in plasma concentration per administered dose.

Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Geometric mean · ng/mL/mcg/kg
Incremental Recovery (IncRec)
ng/mL/mcg/kgPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Incremental Recovery (IncRec)3.48 ± NA18.49 ± 2020.06 ± 2827.67 ± 1626.87 ± 24
SecondaryArea Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf)

AUCinf is area under the plasma concentration-time curve from time 0 extrapolated to infinite time.

Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Geometric mean · ng*hr/mL
Area Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf)
ng*hr/mLPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Area Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf)NA ± NA300.7 ± 15638.9 ± 211557 ± 192788 ± 31
SecondaryMean Residence Time (MRT)

MRT is AUMCinf/AUCinf, where AUMC is the area under the first moment curve.

Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Mean · hours
Mean Residence Time (MRT)
hoursPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Mean Residence Time (MRT)NA ± NA7.948 ± 1.7865.697 ± 0.7274.963 ± 0.7415.087 ± 0.649
SecondaryVolume of Distribution at Steady State (Vss)

Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. Steady state volume of distribution (Vss) is the apparent volume of distribution at steady-state.

Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Geometric mean · L/kg
Volume of Distribution at Steady State (Vss)
L/kgPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Volume of Distribution at Steady State (Vss)NA ± NA0.1168 ± 350.08060 ± 240.05651 ± 210.05494 ± 32
SecondaryClearance (CL)

Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood (rate at which a drug is metabolized or eliminated by normal biological processes). Clearance obtained after intravenous infusion dose is influenced by the fraction of the dose absorbed.

Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Geometric mean · L/hr/kg
Clearance (CL)
L/hr/kgPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Clearance (CL)NA ± NA0.01502 ± 150.01423 ± 210.01149 ± 200.01089 ± 31
SecondaryTime to Reach Maximum Observed Plasma Concentration (Tmax)
Time frame:
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Reported as:
Median · hours
Time to Reach Maximum Observed Plasma Concentration (Tmax)
hoursPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Time to Reach Maximum Observed Plasma Concentration (Tmax)0.167 ± NA0.167 ± 150.242 ± 210.142 ± 200.150 ± 31
SecondaryMaximum Mean Decrease From Baseline in Prothrombin Time (PT)

PT measures how long it takes blood to clot. Maximum mean decrease from baseline at any time point was reported.

Time frame:
Baseline through Day 15
Reported as:
Mean · seconds
Maximum Mean Decrease From Baseline in Prothrombin Time (PT)
secondsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Decrease From Baseline in Prothrombin Time (PT)-0.70 ± NA-3.37 ± 0.372-3.80 ± 0.629-3.85 ± 0.409-4.45 ± 0.493
SecondaryMaximum Mean Decrease From Baseline in Activated Partial Thromboplastin Time (aPTT)

aPTT is a blood test that characterizes blood coagulation. Maximum mean decrease from baseline at any time point was reported.

Time frame:
Baseline through Day 15
Reported as:
Mean · seconds
Maximum Mean Decrease From Baseline in Activated Partial Thromboplastin Time (aPTT)
secondsPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Decrease From Baseline in Activated Partial Thromboplastin Time (aPTT)-1.30 ± NA-17.93 ± 8.944-24.47 ± 12.273-43.02 ± 16.541-46.85 ± 12.281
SecondaryMaximum Mean Increase From Baseline in Thrombin Anti-Thrombin (TAT) Complexes

TAT complex is a parameter of coagulation and fibrinolysis. The normal reference range of values for TAT is 1 to 4.1 mcg/L. Elevated TAT concentrations may signify predisposition to thrombosis. Maximum mean increase from baseline at any time point was reported.

Time frame:
Baseline through Day 3
Reported as:
Mean · mcg/L
Maximum Mean Increase From Baseline in Thrombin Anti-Thrombin (TAT) Complexes
mcg/LPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Increase From Baseline in Thrombin Anti-Thrombin (TAT) Complexes3.70 ± NA23.82 ± 51.1304.72 ± 5.2825.52 ± 6.53022.47 ± 28.010
SecondaryMaximum Mean Increase From Baseline in Prothrombin Fragments 1+2

Prothrombin fragment 1+2 is a coagulation factor, released when prothrombin is cleaved by activated Factor X. Elevated plasma levels of prothrombin fragment 1+2 indicate high risk of thrombosis. Maximum mean increase from baseline at any time point was reported.

Time frame:
Baseline through Day 3
Reported as:
Mean · picomoles per liter (pmol/L)
Maximum Mean Increase From Baseline in Prothrombin Fragments 1+2
picomoles per liter (pmol/L)PF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Increase From Baseline in Prothrombin Fragments 1+238.0 ± NA31.8 ± 30.5666.4 ± 84.7376.7 ± 28.27238.3 ± 162.22
SecondaryMaximum Mean Increase From Baseline in D-Dimers

D-dimer is an indicator of fibrin formation and its subsequent lysis and is a useful biomarker representing overall activation of blood coagulation. Maximum mean increase from baseline at any time point was reported.

Time frame:
Baseline through Day 15
Reported as:
Mean · ng/mL
Maximum Mean Increase From Baseline in D-Dimers
ng/mLPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Increase From Baseline in D-Dimers240.0 ± NA75.0 ± 103.6828.3 ± 173.6088.3 ± 123.36238.3 ± 285.06
SecondaryMaximum Mean Increase From Baseline in Endogenous Thrombin Potential (ETP)

ETP was evaluated using a Thrombin Generation Assay (TGA), a validated automated ex-vivo assay that measures the ability of plasma to generate thrombin. Thrombin generation curves are generated and calculated using dedicated software. ETP is the area under the thrombin generation curve and represents the total amount of generated thrombin. Maximum mean increase from baseline at any time point was reported.

Time frame:
Baseline through Day 3
Reported as:
Mean · nanomolar*minute (nM*min)
Maximum Mean Increase From Baseline in Endogenous Thrombin Potential (ETP)
nanomolar*minute (nM*min)PF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Increase From Baseline in Endogenous Thrombin Potential (ETP)13.400 ± NA212.643 ± 77.3337186.196 ± 147.6925287.173 ± 421.1883522.758 ± 94.0122
SecondaryMaximum Mean Decrease From Baseline in Thrombin Generation Lag Time

The lag time is defined as the time to reach one sixth of the peak height and is a measure of the initiation phase. It is equivalent to the clotting time. Maximum mean decrease from baseline at any time point was reported.

Time frame:
Baseline through Day 3
Reported as:
Mean · minutes
Maximum Mean Decrease From Baseline in Thrombin Generation Lag Time
minutesPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Decrease From Baseline in Thrombin Generation Lag Time-0.540 ± NA-6.235 ± 5.7141-7.088 ± 7.9294-13.400 ± 14.6902-2.425 ± 0.9507
SecondaryMaximum Mean Increase From Baseline in Peak Thrombin Generation

The peak height is defined as the maximum thrombin concentration produced. Maximum mean increase from baseline at any time point was reported.

Time frame:
Baseline through Day 3
Reported as:
Mean · Nanomolar (nM)
Maximum Mean Increase From Baseline in Peak Thrombin Generation
Nanomolar (nM)PF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
Maximum Mean Increase From Baseline in Peak Thrombin Generation-0.620 ± NA17.982 ± 6.092416.568 ± 12.912423.362 ± 28.461849.130 ± 21.0371

Adverse events

Collected over Baseline up to 28 days after last study drug administration (Day 60). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
PF-05280602 0.5 mcg/kg—0/1 (0%)1/1 (100%)
PF-05280602 4.5 mcg/kg—0/6 (0%)5/6 (83.3%)
PF-05280602 9.0 mcg/kg—0/6 (0%)5/6 (83.3%)
PF-05280602 18.0 mcg/kg—0/6 (0%)3/6 (50%)
PF-05280602 30.0 mcg/kg—0/6 (0%)3/6 (50%)
Most frequent other events
Showing 10 of 31
Most frequent other events
EventPF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kg
DiarrhoeaGastrointestinal disorders1/10/60/60/60/6
NasopharyngitisInfections and infestations1/10/60/60/60/6
ArthralgiaMusculoskeletal and connective tissue disorders1/11/61/61/60/6
Flank painMusculoskeletal and connective tissue disorders1/10/60/60/60/6
PrurigoSkin and subcutaneous tissue disorders1/10/60/60/60/6
TinnitusEar and labyrinth disorders0/10/61/60/60/6
Visual impairmentEye disorders0/11/60/60/60/6
Abdominal painGastrointestinal disorders0/10/61/60/60/6
NauseaGastrointestinal disorders0/10/61/60/60/6
VomitingGastrointestinal disorders0/10/61/60/60/6

Baseline characteristics

The baseline analysis population included all enrolled participants who received the study drug.

Age, Continuous
Age, Continuous(years)PF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kgTotal
Mean59.0 ± NA32.7 ± 13.438.5 ± 12.132.8 ± 12.341.0 ± 15.737.2 ± 13.6
Sex: Female, Male
Sex: Female, Male(Participants)PF-05280602 0.5 mcg/kgPF-05280602 4.5 mcg/kgPF-05280602 9.0 mcg/kgPF-05280602 18.0 mcg/kgPF-05280602 30.0 mcg/kgTotal
FEMALE000000
MALE1666625
08

Study locations

23 sites
  • University of California San Diego Medical Center
    San Diego, California 92103-8651, United States
  • New Haven Clinical Research Unit
    New Haven, Connecticut 06511, United States
  • Rush University Medical Center
    Chicago, Illinois 60612, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Doernbecher Children's Hospital
    Portland, Oregon 97239, United States
  • OHSU Investigational Pharmacy
    Portland, Oregon 97239, United States
  • Oregon Health & Science University
    Portland, Oregon 97239, United States
  • CTRC
    Philadelphia, Pennsylvania 19104, United States
  • Penn Comprehensive Hemophilia Program - Center for Blood Disorders
    Philadelphia, Pennsylvania 19104, United States
  • Pfizer Clinical Research Unit
    Bruxelles, B-1070, Belgium
  • Semmelweis Egyetem Altalanos Orvostudomanyi Kar/ I.sz. Belgyogyaszati Klinika
    Budapest, 1083, Hungary
  • Dipartimento di Medicina Clinica 1-Centro Regionale per le Malattie del Sangue
    Castelfranco Veneto (TV), 31033, Italy
  • Servizio Farmacia- Ospedale Castelfranco Veneto
    Castelfranco Veneto - Treviso, 31033, Italy
  • Centro Emofilia e Trombosi "Angelo Bianchi Bonomi" U.O.S. Dipartimentale per la Diagnosi e la Terapi
    Milano, 20122, Italy
  • Servizio Farmacia Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
    Milano, 20122, Italy
  • Centro Malattie Emorragiche e Trombotiche - Ematologia
    Vicenza, 36100, Italy
  • Farmacia Ospedaliera Ospedale San Bortolo
    Vicenza, 36100, Italy
  • Christchurch Clinical Studies Trust
    Christchurch, 08011, New Zealand
  • Phoenix Pharma (Pty) Ltd
    Port Elizabeth, Eastern Cape 6001, South Africa
  • Ege University Tip Fakultesi
    Izmir, 35100, Turkey
  • Royal Free Hampstead NHS Trust, Royal Free Hospital
    London, NW3 2QG, United Kingdom
  • Haematology Department
    London, W12 0HS, United Kingdom
  • Central Manchester Universtiy Hospitals NHS Foundation Trust
    Manchester, M13 9WL, United Kingdom
09

References and documents

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 12, 2017, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT01439971
Lead sponsor
Catalyst Biosciences
Responsible party
Sponsor
First posted
Sep 23, 2011
Start date
Dec 2011
Primary completion
Oct 2015
Completion
Oct 2015
Results posted
Apr 6, 2017
Last update
May 12, 2017

Study contacts

Pfizer CT.gov Call Center
study director · Pfizer

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2017. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Hemophilia A→

Other studies from this sponsor

Catalyst Biosciences

Start the discussion