Trial Outcomes & Findings for Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia (NCT NCT04072237)
NCT ID: NCT04072237
Last Updated: 2021-09-13
Results Overview
Comparative pharmacokinetics (PK) by dose level/stage based on examination of AUC frequencies of these for each of the dose groups
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
11 participants
Primary outcome timeframe
Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.
Results posted on
2021-09-13
Participant Flow
Fourteen subjects were screened in the study and three subjects were screen failures. Two subjects failed due to having a known positive antibody at screening, and one subject failed screening due to other.
Participant milestones
| Measure |
Overall Study Population
Coagulation Factor VIIa variant: Single intravenous dose and ascending doses of subcutaneous injection of MarzAA
|
|---|---|
|
Stage I: MarzAA 18 µg/kg IV
STARTED
|
11
|
|
Stage I: MarzAA 18 µg/kg IV
COMPLETED
|
10
|
|
Stage I: MarzAA 18 µg/kg IV
NOT COMPLETED
|
1
|
|
Stage 2: MarzAA 30 µg/kg SC
STARTED
|
8
|
|
Stage 2: MarzAA 30 µg/kg SC
COMPLETED
|
8
|
|
Stage 2: MarzAA 30 µg/kg SC
NOT COMPLETED
|
0
|
|
Stage 3: MarzAA 45 µg/kg SC
STARTED
|
8
|
|
Stage 3: MarzAA 45 µg/kg SC
COMPLETED
|
8
|
|
Stage 3: MarzAA 45 µg/kg SC
NOT COMPLETED
|
0
|
|
Stage 4: MarzAA 60 µg/kg SC
STARTED
|
8
|
|
Stage 4: MarzAA 60 µg/kg SC
COMPLETED
|
8
|
|
Stage 4: MarzAA 60 µg/kg SC
NOT COMPLETED
|
0
|
|
Stage 5: MarzAA 2 x 30 ug/kg SC
STARTED
|
8
|
|
Stage 5: MarzAA 2 x 30 ug/kg SC
COMPLETED
|
8
|
|
Stage 5: MarzAA 2 x 30 ug/kg SC
NOT COMPLETED
|
0
|
|
Stage 6: MarzAA 90 µg/kg SC
STARTED
|
8
|
|
Stage 6: MarzAA 90 µg/kg SC
COMPLETED
|
8
|
|
Stage 6: MarzAA 90 µg/kg SC
NOT COMPLETED
|
0
|
|
Stage 7: MarzAA 120 µg/kg SC
STARTED
|
8
|
|
Stage 7: MarzAA 120 µg/kg SC
COMPLETED
|
8
|
|
Stage 7: MarzAA 120 µg/kg SC
NOT COMPLETED
|
0
|
|
Stage 8: MarzAA 2 x 60 µg/kg Q3H SC
STARTED
|
8
|
|
Stage 8: MarzAA 2 x 60 µg/kg Q3H SC
COMPLETED
|
8
|
|
Stage 8: MarzAA 2 x 60 µg/kg Q3H SC
NOT COMPLETED
|
0
|
|
Stage 9: MarzAA 3 x 60 µg/kg Q3H SC
STARTED
|
8
|
|
Stage 9: MarzAA 3 x 60 µg/kg Q3H SC
COMPLETED
|
8
|
|
Stage 9: MarzAA 3 x 60 µg/kg Q3H SC
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
Baseline characteristics by cohort
| Measure |
Study Population
n=11 Participants
MarzAA (Marzeptacog Alfa \[activated\], Coagulation Factor VIIa variant) 18 µg/kg intravenously (Stage 1) followed by MarzAA 30 µg/kg subcutaneously (SC) (Stage 2), MarzAA 45 µg/kg SC (Stage 3), MarzAA 60 µg/kg SC (Stage 4), MarzAA 2x30 µg/kg SC (Stage 5), MarzAA 90 µg/kg SC (Stage 6), MarzAA 120 µg/kg SC (Stage 7), MarzAA 2×60 µg/kg SC (Stage 8), MarzAA 3x60 µg/kg SC (Stage 9)
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Bulgaria
|
8 participants
n=5 Participants
|
|
Region of Enrollment
Russia
|
3 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Comparative pharmacokinetics (PK) by dose level/stage based on examination of AUC frequencies of these for each of the dose groups
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last
AUC0-∞
|
1390.0 h*ng/mL
Standard Deviation 433.99
|
516.4 h*ng/mL
Standard Deviation 170.01
|
849.4 h*ng/mL
Standard Deviation 275.00
|
1060.0 h*ng/mL
Standard Deviation 205.86
|
1025.6 h*ng/mL
Standard Deviation 328.31
|
1487.9 h*ng/mL
Standard Deviation 429.39
|
2087.6 h*ng/mL
Standard Deviation 648.85
|
2108.0 h*ng/mL
Standard Deviation 409.00
|
3235.5 h*ng/mL
Standard Deviation 735.43
|
|
Comparative MarzAA Activity by Dose Level/Stage - AUC0-∞ and AUC0-last
AUC0-last
|
1382.6 h*ng/mL
Standard Deviation 431.45
|
429.5 h*ng/mL
Standard Deviation 178.30
|
692.9 h*ng/mL
Standard Deviation 232.53
|
922.1 h*ng/mL
Standard Deviation 192.38
|
934.1 h*ng/mL
Standard Deviation 305.32
|
1322.6 h*ng/mL
Standard Deviation 440.03
|
1868.1 h*ng/mL
Standard Deviation 651.04
|
1939.1 h*ng/mL
Standard Deviation 357.25
|
3038.1 h*ng/mL
Standard Deviation 760.56
|
PRIMARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Comparative pharmacokinetics by dose level/stage based on examination of AUC frequency of these for each of the dose groups
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity by Dose Level/Stage - AUCT1-T2 Normalized by Dose = AUC0-last/Dose
|
76.81 h*kg/mL
Standard Deviation 23.97
|
14.32 h*kg/mL
Standard Deviation 5.94
|
15.40 h*kg/mL
Standard Deviation 5.17
|
15.37 h*kg/mL
Standard Deviation 3.206
|
15.57 h*kg/mL
Standard Deviation 5.09
|
14.70 h*kg/mL
Standard Deviation 4.89
|
15.57 h*kg/mL
Standard Deviation 5.43
|
16.16 h*kg/mL
Standard Deviation 2.98
|
16.88 h*kg/mL
Standard Deviation 4.23
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in Cmax at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - Cmax
|
419.49 ng/mL
Standard Deviation 185.18
|
18.96 ng/mL
Standard Deviation 10.29
|
32.91 ng/mL
Standard Deviation 18.49
|
41.88 ng/mL
Standard Deviation 15.24
|
40.75 ng/mL
Standard Deviation 15.98
|
54.29 ng/mL
Standard Deviation 24.68
|
76.70 ng/mL
Standard Deviation 34.51
|
68.04 ng/mL
Standard Deviation 26.58
|
98.33 ng/mL
Standard Deviation 32.64
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in Tmax at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - Tmax
|
0.17 hour
Standard Deviation 0.29
|
7.50 hour
Standard Deviation 1.60
|
7.38 hour
Standard Deviation 2.56
|
8.25 hour
Standard Deviation 1.39
|
6.75 hour
Standard Deviation 1.398
|
7.12 hour
Standard Deviation 1.55
|
8.25 hour
Standard Deviation 1.39
|
8.37 hour
Standard Deviation 2.67
|
12.25 hour
Standard Deviation 5.04
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in T1/2eqα at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2eqα
|
1.73 hours
Standard Deviation 0.55
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
NA hours
Standard Deviation NA
T1/2eqα was measured and derived only at IV dose.
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in T1/2λ-z at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - T1/2λ-z
|
3.3 hours
Standard Deviation 0.38
|
18.55 hours
Standard Deviation 5.88
|
19.26 hours
Standard Deviation 6.69
|
15.70 hours
Standard Deviation 3.45
|
13.05 hours
Standard Deviation 5.07
|
15.50 hours
Standard Deviation 5.65
|
15.03 hours
Standard Deviation 4.43
|
18.82 hours
Standard Deviation 5.82
|
18.07 hours
Standard Deviation 6.78
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in CL at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - CL
|
14.22 mL/h
Standard Deviation 4.66
|
63.33 mL/h
Standard Deviation 19.64
|
57.85 mL/h
Standard Deviation 18.21
|
58.88 mL/h
Standard Deviation 13.68
|
63.92 mL/h
Standard Deviation 20.36
|
65.13 mL/h
Standard Deviation 18.67
|
64.39 mL/h
Standard Deviation 26.93
|
59.05 mL/h
Standard Deviation 12.68
|
58.22 mL/h
Standard Deviation 13.28
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in Vd1 at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - Vd1
|
53.41 mL
Standard Deviation 18.02
|
1868.28 mL
Standard Deviation 962.02
|
1651.05 mL
Standard Deviation 799.32
|
1400.41 mL
Standard Deviation 478.92
|
1344.19 mL
Standard Deviation 557.26
|
1577.63 mL
Standard Deviation 791.28
|
1509.24 mL
Standard Deviation 796.50
|
1699.49 mL
Standard Deviation 474.39
|
1717.33 mL
Standard Deviation 629.45
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in BAabs at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - BAabs
|
100 Percentage
Standard Deviation 100
|
19.47 Percentage
Standard Deviation 8.07
|
21.52 Percentage
Standard Deviation 10.63
|
21.32 Percentage
Standard Deviation 7.42
|
20.87 Percentage
Standard Deviation 6.32
|
19.84 Percentage
Standard Deviation 7.20
|
21.24 Percentage
Standard Deviation 9.32
|
23.00 Percentage
Standard Deviation 8.98
|
23.32 Percentage
Standard Deviation 6.69
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.Change in Mean Residence Time at each stage for each dose group
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Comparative MarzAA Activity of Intravenous and Subcutaneous - Mean Residence Time
|
3.77 hours
Standard Deviation 0.42
|
28.25 hours
Standard Deviation 9.18
|
28.10 hours
Standard Deviation 9.94
|
23.79 hours
Standard Deviation 5.40
|
20.96 hours
Standard Deviation 5.58
|
23.42 hours
Standard Deviation 7.47
|
22.80 hours
Standard Deviation 5.9211
|
29.04 hours
Standard Deviation 7.88
|
29.32 hours
Standard Deviation 8.60
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.PK analysis by route of administration, dose level/stage of the study based on examination of AUC for each of the dose groups. Split dose (2\*30 µg/kg) vs. (60 µg/kg)
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=8 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Effect of Split Injections on MarzAA Activity by Dose Level/Stage - AUC From T1 to T2 Norm by Dose
|
15.3687 h*kg/mL
Standard Deviation 3.20630
|
15.5688 h*kg/mL
Standard Deviation 5.08859
|
—
|
—
|
—
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing, depending on participation in all 9 stages and time elapsed between each study stage.PK analysis by route of administration, dose level/stage of the study based on examination of AUC for each of the dose groups. Split dose (2\*30 µg/kg) vs. (60 µg/kg)
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=8 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Effect of Split Injections on MarzAA Activity by Dose Level/Stage - AUC Infinity Obs and AUC to Last Nonzero Conc
AUC to Last Nonzero Conc
|
922.1 h*ng/mL
Standard Deviation 192.38
|
934.1 h*ng/mL
Standard Deviation 305.32
|
—
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Effect of Split Injections on MarzAA Activity by Dose Level/Stage - AUC Infinity Obs and AUC to Last Nonzero Conc
AUC Infinity Obs
|
1060.0 h*ng/mL
Standard Deviation 205.86
|
1025.6 h*ng/mL
Standard Deviation 328.31
|
—
|
—
|
—
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-7 (SC).Maximum change in PT from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Coagulation Parameters - Prothrombin Time (PT)
|
-1.36 seconds
Standard Deviation 0.57
|
-1.41 seconds
Standard Deviation 0.86
|
-1.16 seconds
Standard Deviation 0.68
|
-1.09 seconds
Standard Deviation 0.58
|
-1.29 seconds
Standard Deviation 0.86
|
-1.07 seconds
Standard Deviation 0.79
|
-1.37 seconds
Standard Deviation 0.54
|
-1.36 seconds
Standard Deviation 0.60
|
-1.35 seconds
Standard Deviation 0.62
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in aPTT from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Coagulation Parameters - Activated Partial Thromboplastin Time (aPTT)
|
-11.69 seconds
Standard Deviation 3.71
|
1.91 seconds
Standard Deviation 3.21
|
-2.33 seconds
Standard Deviation 4.28
|
-2.18 seconds
Standard Deviation 2.63
|
-2.51 seconds
Standard Deviation 2.09
|
-2.85 seconds
Standard Deviation 1.82
|
-2.10 seconds
Standard Deviation 3.02
|
-2.92 seconds
Standard Deviation 3.01
|
-5.13 seconds
Standard Deviation 2.59
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in TGT parameter from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Peak
|
64.0 nM
Standard Deviation 65.32
|
14.9 nM
Standard Deviation 31.69
|
28.3 nM
Standard Deviation 21.04
|
42.3 nM
Standard Deviation 32.13
|
45.4 nM
Standard Deviation 30.84
|
33.0 nM
Standard Deviation 25.16
|
44.3 nM
Standard Deviation 46.10
|
45.6 nM
Standard Deviation 19.18
|
41.3 nM
Standard Deviation 30.47
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in TGT parameters from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Lag and TGT-Time to Peak
TGT-Lag
|
-1.30 minutes
Standard Deviation 1.231
|
-1.21 minutes
Standard Deviation 0.613
|
-1.11 minutes
Standard Deviation 0.391
|
-1.42 minutes
Standard Deviation 0.645
|
-1.38 minutes
Standard Deviation 0.311
|
-1.07 minutes
Standard Deviation 0.655
|
-1.33 minutes
Standard Deviation 0.396
|
-1.54 minutes
Standard Deviation 0.507
|
-1.13 minutes
Standard Deviation 0.688
|
|
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Lag and TGT-Time to Peak
TGT-Time to Peak
|
-3.88 minutes
Standard Deviation 3.302
|
-2.22 minutes
Standard Deviation 2.309
|
-2.76 minutes
Standard Deviation 1.226
|
-3.53 minutes
Standard Deviation 2.313
|
-3.68 minutes
Standard Deviation 1.290
|
-3.73 minutes
Standard Deviation 1.926
|
-3.73 minutes
Standard Deviation 2.024
|
-3.70 minutes
Standard Deviation 1.681
|
-3.25 minutes
Standard Deviation 1.756
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in TGT parameter from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Coagulation Parameters - Thrombin Generation Time (TGT) - TGT-Endogenous Thrombin Potential
|
215.9 nM•minutes
Standard Deviation 337.55
|
158.6 nM•minutes
Standard Deviation 247.55
|
94.3 nM•minutes
Standard Deviation 148.51
|
149.5 nM•minutes
Standard Deviation 256.34
|
211.0 nM•minutes
Standard Deviation 190.22
|
133.0 nM•minutes
Standard Deviation 193.85
|
216.9 nM•minutes
Standard Deviation 184.54
|
164.6 nM•minutes
Standard Deviation 58.49
|
-187.3 nM•minutes
Standard Deviation 474.61
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in thrombogenicity parameter from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Thrombogenicity Parameter - Fibrinogen
|
-13.2 mg/dL
Standard Deviation 23.83
|
13.3 mg/dL
Standard Deviation 29.65
|
-23.6 mg/dL
Standard Deviation 34.04
|
16.0 mg/dL
Standard Deviation 37.67
|
7.6 mg/dL
Standard Deviation 51.69
|
-31.0 mg/dL
Standard Deviation 42.02
|
21.6 mg/dL
Standard Deviation 56.61
|
-12.5 mg/dL
Standard Deviation 32.74
|
-8.0 mg/dL
Standard Deviation 33.72
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in thrombogenicity parameter from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Thrombogenicity Parameter - Prothrombin Fragments 1 + 2
|
1903.5 pmol/L
Standard Deviation 3809.47
|
206.3 pmol/L
Standard Deviation 545.97
|
56.0 pmol/L
Standard Deviation 158.02
|
76.5 pmol/L
Standard Deviation 118.78
|
383.9 pmol/L
Standard Deviation 324.41
|
974.5 pmol/L
Standard Deviation 2407.09
|
1744.9 pmol/L
Standard Deviation 4367.48
|
236.5 pmol/L
Standard Deviation 459.58
|
284.9 pmol/L
Standard Deviation 672.80
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in thrombogenicity parameter from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Thrombogenicity Parameter - Thrombin/Antithrombin
|
105.09 µg/L
Standard Deviation 254.951
|
62.34 µg/L
Standard Deviation 156.899
|
10.41 µg/L
Standard Deviation 19.586
|
63.57 µg/L
Standard Deviation 181.409
|
138.19 µg/L
Standard Deviation 202.946
|
56.79 µg/L
Standard Deviation 146.603
|
10.46 µg/L
Standard Deviation 15.648
|
18.90 µg/L
Standard Deviation 38.191
|
3.75 µg/L
Standard Deviation 3.231
|
SECONDARY outcome
Timeframe: From predose to Day 2 at stage 1 (IV). From predose to Day 3 at stages 2-9 (SC).Maximum change in thrombogenicity parameter from pre-dose
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Change in Thrombogenicity Parameter - D-Dimer
|
0.141 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.214
|
-0.054 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.104
|
0.110 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.080
|
0.039 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.073
|
0.285 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.605
|
0.200 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.175
|
0.211 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.285
|
0.111 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.125
|
0.256 mg/L Fibrinogen Equivalent Unit
Standard Deviation 0.173
|
SECONDARY outcome
Timeframe: From time of first dose of MarzAA until date of first occurrence of clinical event, assessed up to End of Study. Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing.Occurrence of an antibody response to MarzAA and whether it is inhibitory and cross-reactive to wild-type recombinant coagulation FVII (wt-rFVII) or wt-FVIIa
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Occurrence of an Antibody Response to MarzAA
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
SECONDARY outcome
Timeframe: From the date of first dose of MarzAA until date of first occurrence of clinical event, assessed up to End of Study. Dosing period for each stage was approximately 3 days, with a maximum of approximately 8 weeks of dosing.Occurrence of clinical thrombotic event not attributable to another cause
Outcome measures
| Measure |
Stage 1 MarzAA 18 µg/kg IV
n=10 Participants
Single intravenous dose of MarzAA, coagulation factor VIIa variant
|
Stage 2 MarzAA 30 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 3 MarzAA 45 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 4 MarzAA 60 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant
|
Stage 6 MarzAA 90 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 7 MarzAA 120 µg/kg SC
n=8 Participants
Single subcutaneous injection of MarzAA, coagulation factor VIIa variant
|
Stage 8 2×60 µg/kg SC Q3H
n=8 Participants
Two subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
Stage 9 3×60 µg/kg SC Q3H
n=8 Participants
Three subcutaneous injections of MarzAA, coagulation factor VIIa variant, every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
Occurrence of Clinical Thrombotic Event
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
0 participants with an occurrence
|
Adverse Events
Stage 1 MarzAA 18 µg/kg SC
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Stage 2 MarzAA 30 µg/kg SC
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Stage 3 MarzAA 45 µg/kg SC
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Stage 4 MarzAA 60 µg/kg SC
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Stage 5 MarzAA 2×30 µg/kg SC
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Stage 6 MarzAA 90 µg/kg SC
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Stage 7 MarzAA 120 µg/kg SC
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Stage 8 MarzAA 2×60 µg/kg SC Q3H
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Stage 9 MarzAA 3×60 µg/kg SC Q3H
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Stage 1 MarzAA 18 µg/kg SC
n=11 participants at risk
Single intravenous dose of MarzAA
|
Stage 2 MarzAA 30 µg/kg SC
n=8 participants at risk
Single subcutaneous injection of MarzAA
|
Stage 3 MarzAA 45 µg/kg SC
n=8 participants at risk
Single subcutaneous injection of MarzAA
|
Stage 4 MarzAA 60 µg/kg SC
n=8 participants at risk
Single subcutaneous injection of MarzAA
|
Stage 5 MarzAA 2×30 µg/kg SC
n=8 participants at risk
Two subcutaneous injections of MarzAA
|
Stage 6 MarzAA 90 µg/kg SC
n=8 participants at risk
Single subcutaneous injection of MarzAA
|
Stage 7 MarzAA 120 µg/kg SC
n=8 participants at risk
Single subcutaneous injection of MarzAA
|
Stage 8 MarzAA 2×60 µg/kg SC Q3H
n=8 participants at risk
Two subcutaneous injections of MarzAA every 3 hours
|
Stage 9 MarzAA 3×60 µg/kg SC Q3H
n=8 participants at risk
Three subcutaneous injections of MarzAA every 3 hours
|
|---|---|---|---|---|---|---|---|---|---|
|
General disorders
Injection site reaction
|
0.00%
0/11 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
12.5%
1/8 • Number of events 1 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
25.0%
2/8 • Number of events 2 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
37.5%
3/8 • Number of events 4 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
25.0%
2/8 • Number of events 2 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
|
General disorders
Pyrexia
|
0.00%
0/11 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
12.5%
1/8 • Number of events 1 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
12.5%
1/8 • Number of events 1 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/11 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
12.5%
1/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
12.5%
1/8 • Number of events 1 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/11 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
12.5%
1/8 • Number of events 1 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
0.00%
0/8 • Treatment-emergent adverse events were defined as any event that either began or worsened after the first administration of study drug and within 28 days of the last dose.
An adverse event (AE) was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a drug, without any judgment about causality. An AE could arise from any use of the drug (eg, off label use, use in combination with another drug) and from any route of administration, formulation, or dose, including an overdose.
|
Additional Information
Howard Levy, Chief Medical Officer
Catalyst Biosciences
Phone: +1.650.266.6871
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place