Trial · NCT01027364

Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B

NCT01027364, Phase 3, Completed, Bioverativ Therapeutics Inc.; results first posted 2014-09-04

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B. Trials, record NCT01027364. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01027364

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
NCT number: NCT01027364
Status: Completed
Phases: Phase 3
Enrollment: 123 participants, actual
Lead sponsor: Bioverativ Therapeutics Inc.
Conditions: Severe Hemophilia B
Interventions: Factor IX (rFIXFc), rFIX
First posted: 2009-12-07
Last updated: 2020-12-19
Other registries' IDs: eudract:2009-014295-21
Results first posted: 2014-09-04

Source ClinicalTrials.gov

Registered outcomes38

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	up to 52 weeks ± 1 week	Clinical laboratory evaluations included hematology and blood chemistry. Table does not include laboratory tests evaluated during the surgical/rehabilitation period. Because the perioperative management period represents a unique clinical situation, safety data obtained during the surgical/rehabilitation period for participants in Arm 4 were included in listings and reviewed separately. Review of the listing was sufficient to assess this endpoint. ULN=upper limit of normal.
Primary	Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	up to 52 weeks + 30 days ± 1 week	AE=any untoward medical occurrence that did not necessarily have a causal relationship with this treatment, and could be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study product, whether related or not. TE=event present prior to receiving the first injection of BeneFIX or rFIXFc that subsequently worsened in severity or not present prior to receiving the first injection but subsequently appeared before last visit on study. Serious AE (SAE)=AE resulting in death, immediate risk of death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, or a congenital/anomaly/birth defect, or any other medically important event. Related=related, possibly related, and relationship missing. Data include AEs emergent during the surgical/rehabilitation period; AE data are included in each treatment arm only for the time each participant was enrolled in that arm.
Primary	Number of Participants With Non-serious Treatment-emergent Adverse Events (TEAEs) During the Surgical / Rehabilitation Period	up to 52 weeks + 30 days ± 1 week	AE=any untoward medical occurrence that did not necessarily have a causal relationship with this treatment, and could be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study product, whether related or not. TEAE=AE present prior to receiving the first injection of BeneFIX or rFIXFc that subsequently worsened in severity or was not present prior to receiving the first injection but subsequently appeared before last visit on study. Participants are counted once if they report multiple events in the same system organ class (SOC) or preferred term (PT). Coded using the Medical Dictionary for Regulatory Activities (MedDRA), version 15.0 dictionary. The following SOCs are abbreviated in the table: Immune System (IS); Injury, Poisoning, and Procedural (IPP); Metabolism and Nutrition (MN); Musculoskeletal and Connective Tissue (MCT); Respiratory, Thoracic and Mediastinal (RTM); Skin and Subcutaneous Tissue (SST).
Primary	Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) During the Surgical / Rehabilitation Period	up to 52 weeks + 30 days ± 1 week	SAE=AE resulting in death, immediate risk of death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, or a congenital/anomaly/birth defect, or any other medically important event. TESAE=SAE present prior to receiving the first injection of BeneFIX or rFIXFc that subsequently worsened in severity or was not present prior to receiving the first injection but subsequently appeared before last visit on study. Participants are counted once if they report multiple events in the same system organ class (SOC) or preferred term (PT). Coded using the Medical Dictionary for Regulatory Activities (MedDRA), version 15.0 dictionary. The following SOC is abbreviated in the table: Injury, Poisoning, and Procedural (IPP).
Primary	Incidence Rate of FIX Inhibitor Development	up to 52 weeks ± 1 week	An inhibitor test result ≥0.6 Bethesda units (BU)/mL, identified and confirmed by re-testing of a second sample obtained within 2 to 4 weeks, was considered positive. Both tests were to be performed using the Nijmegen-modified Bethesda Assay by the central laboratory. The incidence rates along with the 95% CI were summarized for all titers for subjects with 50 or more exposure days (EDs) to rFIXFc and a valid inhibitor test after the 50th exposure. In addition, the incidence rates for all subjects regardless of their exposure days to rFIXFc were also summarized. The 95% CI was calculated using Clopper-Pearson exact method.
Primary	Annualized Bleeding Rate	up to 52 weeks ± 1 week (efficacy period as defined in description)	Annualized bleeding episodes = (number of bleeding episodes / number of days in the respective period)\*365.25. In Arms 1 and 2, the efficacy period (EP) started with date and time of first prophylactic dose following a completed pharmacokinetic (PK) sampling period and ended with last dose administered (for prophylaxis or a bleeding episode). In Arm 3, the EP started following last PK sampling timepoint and ended with either date of last contact or date of last entry into the eDiary, whichever was later. The EP was interrupted for the repeat PK period in Arm 1 (sequential PK subgroup) and for all surgical/rehabilitation periods (for both major and minor surgeries) in all 3 arms. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode.
Primary	Comparison of Annualized Bleeding Rates	up to 52 weeks ± 1 week (efficacy period as defined in description)	Estimated with a factor for arm, based on whole study duration for all participants. Annualized bleeding episodes = (number of bleeding episodes / number of days in the respective period)\*365.25. In Arms 1 and 2, the efficacy period (EP) started with date and time of first prophylactic dose following a completed PK sampling period and ended with last dose administered (for prophylaxis or a bleeding episode). In Arm 3, the EP started following last PK sampling timepoint and ended with either date of last contact or date of last entry into the eDiary, whichever was later. The EP was interrupted for the repeat PK period in Arm 1 and for all surgical/rehabilitation periods in all 3 arms. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode.
Secondary	Participant Assessment of Response to Injections to Treat a Bleeding Episode	up to 52 weeks ± 1 week	Participant's assessment of the response to the first rFIXFc injection for each bleeding episode. Percentages were based on the number of bleeding episodes for which a response was provided for the first injection, using the following 4-point scale: excellent=abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hours after the initial injection; good=definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after an injection, but possibly requiring more than one injection after 24 to 48 hours for complete resolution; moderate=probable or slight beneficial effect within 8 hours after the initial injection and requiring more than one injection; no response=no improvement, or condition worsened, within approximately 8 hours after the initial injection.
Secondary	Physicians' Global Assessments of Participants' Response to Treatment With rFIXFc	up to 52 weeks ± 1 week	Physicians assessed each participant's response to rFIXFc using a 4-point scale: excellent=bleeding episodes responded to less than or equal to the usual number of injections or less than or equal to the usual dose of rFIXFc, or the rate of breakthrough bleeding during prophylaxis was less than or equal to that usually observed; effective=most bleeding episodes responded to the same number of injections and dose, but some required more injections or higher doses, or there was a minor increase in the rate of breakthrough bleeding; partially effective=bleeding episodes most often required more injections and/or higher doses than expected, or adequate breakthrough bleeding prevention during prophylaxis required more frequent injections and/or higher doses; ineffective=routine failure to control hemostasis or hemostatic control required additional agents. Percentage of the total count of scale responses for all participants is presented. Multiple responses per participant are counted.
Secondary	Annualized rFIXFc Consumption Per Participant	up to 52 weeks ± 1 week (efficacy period as defined in description)	Consumption is calculated for the efficacy period (EP). In Arms 1 and 2, the EP started with date and time of first prophylactic dose following a completed PK sampling period and ended with last dose administered (for prophylaxis or a bleeding episode). In Arm 3, the EP started following last PK sampling timepoint and ended with either date of last contact or date of last entry into the eDiary, whichever was later. The EP was interrupted for the repeat PK period in Arm 1 (sequential PK subgroup) and for all surgical/rehabilitation periods (for both major and minor surgeries) in all 3 arms. Overall units (IU/kg) of annualized rFIXFc consumption = \[Total rFIXFc IU/kg received during the EP / number of days in EP\]\*365.25.

1 to 10 of 38Source ClinicalTrials.gov

See all 38 registered outcomes

Outcomes38

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	up to 52 weeks ± 1 week	Safety Analysis Set: participants who received at least 1 dose of BeneFIX or rFIXFc; n=the number of participants with at least one post-baseline value. For this study, a table was not generated for potentially clinically significant laboratory abnormalities for participants in the perioperative management/surgical arm (Arm 4).	participants	NUMBER
PRIMARY	Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)	up to 52 weeks + 30 days ± 1 week	Safety Analysis Set: participants who received at least 1 dose of BeneFIX or rFIXFc. A participant may have been in more than one group (i.e., participants in Arm 4 who were also in Arm 1, 2, or 3; please see Participant Flow for details). Participants with at least one TESAE reported are included in the TEAE count.	participants	NUMBER
PRIMARY	Number of Participants With Non-serious Treatment-emergent Adverse Events (TEAEs) During the Surgical / Rehabilitation Period	up to 52 weeks + 30 days ± 1 week	Safety Analysis Set: participants who received at least 1 dose of BeneFIX or rFIXFc. A participant may have been in more than one group (i.e., participants in Arm 4 who were also in Arm 1, 2, or 3; please see Participant Flow for details).	participants	NUMBER
PRIMARY	Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) During the Surgical / Rehabilitation Period	up to 52 weeks + 30 days ± 1 week	Safety Analysis Set: participants who received at least 1 dose of BeneFIX or rFIXFc. A participant may have been in more than one group (i.e., participants in Arm 4 who were also in Arm 1, 2, or 3; please see Participant Flow for details).	participants	NUMBER
SECONDARY	Participant Assessment of Response to Injections to Treat a Bleeding Episode	up to 52 weeks ± 1 week	Full Analysis Set: participants who received at least 1 dose of rFIXFc and had a bleeding episode; participants with a non-evaluable bleeding episode are counted in the 'number of participants analyzed,' but not the percentages.	percentage of responses	NUMBER
SECONDARY	Physicians' Global Assessments of Participants' Response to Treatment With rFIXFc	up to 52 weeks ± 1 week	Full Analysis Set: participants who received at least 1 dose of rFIXFc, had evaluable efficacy assessments, and had nonmissing observations at time point.	percentage of responses	NUMBER
SECONDARY	Annualized rFIXFc Consumption Per Participant	up to 52 weeks ± 1 week (efficacy period as defined in description)	Full Analysis Set: participants who received at least 1 dose of rFIXFc with evaluable data in the efficacy period. 'Overall' n=all participants in the Full Analysis Set with evaluable data in the efficacy period; 'Last 3 Months on Study' n=all participants in the Full Analysis Set with evaluable data and \>=6 months on study.	IU/kg rFIXFc per participant per year	MEAN
SECONDARY	Average Weekly Dose For the Fixed Weekly Interval Prophylaxis Arm	up to 52 weeks ± 1 week (efficacy period as defined in description)	Full Analysis Set: participants in Arm 1 who received at least 1 dose of rFIXFc with evaluable data. 'Overall' n=participants with evaluable data; 'Last 3 Months on Study' n=participants with evaluable data and \>=6 months on study.	IU/kg	MEAN
SECONDARY	Average Dosing Interval For the Individualized Interval Prophylaxis Arm	up to 52 weeks ± 1 week (efficacy period as defined in description)	Full Analysis Set: participants in Arm 2 who received at least 1 dose of rFIXFc with \>=6 months on study and evaluable data.	days	MEDIAN
SECONDARY	Annualized Bleeding Rate by Type of Bleed (Spontaneous and Traumatic)	up to 52 weeks ± 1 week (efficacy period as defined in description)	Full Analysis Set: participants who received at least 1 dose of rFIXFc with evaluable data.	episodes per participant per year	MEDIAN

1 to 10 of 38Source ClinicalTrials.gov

See all 38 outcomes

Results by group426

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 1: Weekly Prophylaxis	White Blood Cells <3.0*10^9/L; n=62, 28, 27	2	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 2: Individualized Interval Prophylaxis	White Blood Cells <3.0*10^9/L; n=62, 28, 27	0	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 3: Episodic (On Demand)	White Blood Cells <3.0*10^9/L; n=62, 28, 27	2	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 1: Weekly Prophylaxis	White Blood Cells >=16*10^9/L; n=62, 28, 27	0	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 2: Individualized Interval Prophylaxis	White Blood Cells >=16*10^9/L; n=62, 28, 27	0	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 3: Episodic (On Demand)	White Blood Cells >=16*10^9/L; n=62, 28, 27	0	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 1: Weekly Prophylaxis	Lymphocytes <0.8*10^9/L; n=60, 28, 26	1	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 2: Individualized Interval Prophylaxis	Lymphocytes <0.8*10^9/L; n=60, 28, 26	2	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 3: Episodic (On Demand)	Lymphocytes <0.8*10^9/L; n=60, 28, 26	3	participants
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities	Arm 1: Weekly Prophylaxis	Lymphocytes >12*10^9/L; n=60, 28, 26	0	participants

1 to 10 of 426Source ClinicalTrials.gov

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Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Arm 1: Weekly Prophylaxis	63
Overall Study	STARTED	Arm 2: Individualized Interval Prophylaxis	29
Overall Study	STARTED	Arm 3: Episodic (On Demand)	27
Overall Study	STARTED	Arm 4: Perioperative Management	4
Overall Study	Enrolled in Sequential PK Subgroup	Arm 1: Weekly Prophylaxis	22
Overall Study	Enrolled in Sequential PK Subgroup	Arm 2: Individualized Interval Prophylaxis	0
Overall Study	Enrolled in Sequential PK Subgroup	Arm 3: Episodic (On Demand)	0
Overall Study	Enrolled in Sequential PK Subgroup	Arm 4: Perioperative Management	0
Overall Study	Joined Arm 4 for Surgery Then Returned	Arm 1: Weekly Prophylaxis	5
Overall Study	Joined Arm 4 for Surgery Then Returned	Arm 2: Individualized Interval Prophylaxis	0

1 to 10 of 24Source ClinicalTrials.gov

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Adverse events84

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Nasopharyngitis	Infections and infestations	Arm 1: Weekly Prophylaxis	13	63
other	Nasopharyngitis	Infections and infestations	Arm 2: Individualized Interval Prophylaxis	4	29
other	Nasopharyngitis	Infections and infestations	Arm 3: Episodic (On Demand)	1	27
other	Influenza	Infections and infestations	Arm 1: Weekly Prophylaxis	5	63
other	Influenza	Infections and infestations	Arm 2: Individualized Interval Prophylaxis	0	29
other	Influenza	Infections and infestations	Arm 3: Episodic (On Demand)	4	27
other	Upper respiratory tract infection	Infections and infestations	Arm 1: Weekly Prophylaxis	4	63
other	Upper respiratory tract infection	Infections and infestations	Arm 2: Individualized Interval Prophylaxis	2	29
other	Upper respiratory tract infection	Infections and infestations	Arm 3: Episodic (On Demand)	1	27
other	Sinusitis	Infections and infestations	Arm 1: Weekly Prophylaxis	3	63

1 to 10 of 84Source ClinicalTrials.gov

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Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Arm 1: Weekly Prophylaxis
Total, all-cause mortality	Arm 2: Individualized Interval Prophylaxis
Total, all-cause mortality	Arm 3: Episodic (On Demand)
Total, other adverse events	Arm 1: Weekly Prophylaxis	29	63
Total, other adverse events	Arm 2: Individualized Interval Prophylaxis	17	29
Total, other adverse events	Arm 3: Episodic (On Demand)	11	27
Total, serious adverse events	Arm 1: Weekly Prophylaxis	5	63
Total, serious adverse events	Arm 2: Individualized Interval Prophylaxis	4	29
Total, serious adverse events	Arm 3: Episodic (On Demand)	4	27

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
24304002	RESULT	PubMed
36848635	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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