Trial Outcomes & Findings for Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B (NCT NCT03091751)
NCT ID: NCT03091751
Last Updated: 2017-07-25
Results Overview
BeneFIX pharmacokinetic parameter of area under the curve (AUC 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 study).
COMPLETED
PHASE2
13 participants
Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.
2017-07-25
Participant Flow
Participant milestones
| Measure |
BeneFIX
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
BeneFIX: BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B
Baseline characteristics by cohort
| Measure |
BeneFIX
n=22 Participants
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
BeneFIX: BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
|
|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
21 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
|
22 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
|
22 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
|
PRIMARY outcome
Timeframe: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.Population: Thirteen subjects with 2 prior pharmacokinetic assessments with AlphaNine (PK1, PK2) had a third PK assessment with BeneFIX (PK3). Nine subjects had PK3 analysis with BeneFIX as part of a previous study. In total, 22 subjects treated with BeneFIX (PK3) were analyzed jointly and compared to 25 subjects treated with AlphaNine (PK2).
BeneFIX pharmacokinetic parameter of area under the curve (AUC 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 study).
Outcome measures
| Measure |
BeneFIX
n=22 Participants
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
BeneFIX: BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
|
|---|---|
|
Mean Difference of Area Under the Curve (AUC): BeneFIX Compared to AlphaNine
|
10.41 IU x hour/dL
Standard Deviation 563.081
|
PRIMARY outcome
Timeframe: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.Population: Thirteen subjects with 2 prior pharmacokinetic assessments with AlphaNine (PK1, PK2) had a third PK assessment with BeneFIX (PK3). Nine subjects had PK3 analysis with BeneFIX as part of a previous study. In total, 22 subjects treated with BeneFIX (PK3) were analyzed jointly and compared to 25 subjects treated with AlphaNine (PK2).
BeneFIX pharmacokinetic parameter of in vivo recovery was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
Outcome measures
| Measure |
BeneFIX
n=22 Participants
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
BeneFIX: BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
|
|---|---|
|
Mean Difference of In Vivo Recovery: BeneFIX Compared to AlphaNine
|
0.3068 kg/dL
Standard Deviation 0.37505
|
PRIMARY outcome
Timeframe: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.Population: Thirteen subjects with 2 prior pharmacokinetic assessments with AlphaNine (PK1, PK2) had a third PK assessment with BeneFIX (PK3). Nine subjects had PK3 analysis with BeneFIX as part of a previous study. In total, 22 subjects treated with BeneFIX (PK3) were analyzed jointly and compared to 25 subjects treated with AlphaNine (PK2).
BeneFIX pharmacokinetic parameter of terminal half-life was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
Outcome measures
| Measure |
BeneFIX
n=22 Participants
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
BeneFIX: BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
|
|---|---|
|
Mean Difference of Terminal Half-Life: BeneFIX Compared to AlphaNine
|
-3.3 hours
Standard Deviation 13.48
|
PRIMARY outcome
Timeframe: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.Population: Thirteen subjects with 2 prior pharmacokinetic assessments with AlphaNine (PK1, PK2) had a third PK assessment with BeneFIX (PK3). Nine subjects had PK3 analysis with BeneFIX as part of a previous study. In total, 22 subjects treated with BeneFIX (PK3) were analyzed jointly and compared to 25 subjects treated with AlphaNine (PK2).
BeneFIX pharmacokinetic parameter of clearance was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
Outcome measures
| Measure |
BeneFIX
n=22 Participants
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
BeneFIX: BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
|
|---|---|
|
Mean Difference of Clearance: BeneFIX Compared to AlphaNine
|
-0.0058 mL/min x kg
Standard Deviation 0.0145
|
PRIMARY outcome
Timeframe: Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.Population: Thirteen subjects with 2 prior pharmacokinetic assessments with AlphaNine (PK1, PK2) had a third PK assessment with BeneFIX (PK3). Nine subjects had PK3 analysis with BeneFIX as part of a previous study. In total, 22 subjects treated with BeneFIX (PK3) were analyzed jointly and compared to 25 subjects treated with AlphaNine (PK2).
BeneFIX pharmacokinetic parameter of mean residence time (MRT 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).
Outcome measures
| Measure |
BeneFIX
n=22 Participants
BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.
BeneFIX: BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.
|
|---|---|
|
Mean Difference of Mean Residence Time (MRT): BeneFIX Compared to AlphaNine
|
-7.609 hours
Standard Deviation 13.7121
|
Adverse Events
BeneFIX
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Site may publish results from the study, after providing Sponsor 30 days' notice prior to submitting a manuscript or other materials related to the study to any outside party. At Sponsor's request, site will remove any confidential information (other than study results) and site will, upon Sponsor's request, delay publication or presentation for a period of up to 120 days to allow Sponsor to protect its interests in any Sponsor interventions.
- Publication restrictions are in place
Restriction type: OTHER