Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B (Kids B-LONG)

• ClinicalTrials.gov Identifier: NCT01440946
• Recruitment Status: Completed
• First Posted: September 27, 2011
• Results First Posted: June 18, 2015
• Last Update Posted: December 19, 2020
• Sponsor: Bioverativ Therapeutics Inc.
• Collaborator: Swedish Orphan Biovitrum
• Information provided by (Responsible Party): Sanofi ( Bioverativ Therapeutics Inc. )

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Hemophilia B
• Interventions: Drug: rFIXFc; Drug: FIX
• Enrollment: 30

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description	At Baseline and at Day 1, participants received a single intravenous (IV) injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last pharmacokinetic (PK) sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Period Title: Overall Study
Started	15	15
Completed	13	14
Not Completed	2	1
Reason Not Completed
Lost to Follow-up	1	1
Physician Decision	1	0

Baseline Characteristics

Arm/Group Title		Participants < 6 Years Old	Participants 6 to < 12 Years Old	Total
Arm/Group Description		At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	Total of all reporting groups
Overall Number of Baseline Participants		15	15	30
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	15 participants	15 participants	30 participants
		2.6 (0.99)	8.3 (1.45)	5.5 (3.16)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	15 participants	15 participants	30 participants
	Female	0 0.0%	0 0.0%	0 0.0%
	Male	15 100.0%	15 100.0%	30 100.0%

Outcome Measures

1. Primary Outcome

Title	Occurence of Factor IX (FIX) Inhibitor Development
Description	An inhibitor test result ≥ 0.6 Bethesda units (BU)/mL, confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Incidences were summarized for any positive inhibitor for participants with ≥ 50 exposure days (EDs) to rFIXFc. In addition, the incidence for all participants, regardless of their EDs to rFIXFc, was also summarized. An exact 95% CI for the proportion of participants with a confirmed inhibitor was calculated using the Clopper-Pearson exact method for a binomial proportion.
Time Frame	Up to 50 weeks +/- 7 days, or up to 50 EDs if reached prior to Week 50

Outcome Measure Data

Analysis Population Description

Safety Analysis Set: participants who received at least 1 dose of prestudy FIX, or at least 1 dose of rFIXFc; n=number of participants with given number of EDs who had a valid inhibitor test.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old	Total
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	All Participants
Overall Number of Participants Analyzed	15	15	30
Measure Type: Number; Number (95% Confidence Interval); Unit of Measure: percentage of participants
Participants with ≥ 50 EDs; n=10, 13, 23	0; (0 to 30.85)	0; (0 to 23.16)	0; (0 to 14.25)
All participants regardless of # of EDs;n=15,15,30	0; (0 to 21.8)	0; (0 to 21.8)	0; (0 to 11.57)

2. Secondary Outcome

Title	Annualized Bleeding Rate
Description	Annualized bleeding rate = (number of bleeding episodes during the efficacy period / total number of days during the efficacy period)*365.25. The efficacy period begins with the first prophylactic dose of rFIXFc and ends with the last dose (for prophylaxis or a bleeding episode). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. A bleeding episode started from the first sign of bleeding and ended no more than 72 hours after the last treatment for the bleeding episode, 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. Any injection to treat the bleeding episode taken more than 72 hours after the preceding one was considered the first injection to treat a new bleeding episode at the same location. Any bleeding at a different location was considered a separate bleeding episode, regardless of time from last injection.
Time Frame	Up to 50 weeks +/- 7 days (efficacy period as defined in description)

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received at least 1 dose of rFIXFc; based on the number of participants whose efficacy period was of at least 1 day in duration.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	15	15
Median (Inter-Quartile Range); Unit of Measure: bleeding episodes per participant per yr
	1.09; (0.00 to 2.90)	2.13; (0.00 to 4.17)

3. Secondary Outcome

Title	Annualized Joint Bleeding Rate (Spontaneous)
Description	Annualized bleeding rate for spontaneous joint bleeding episode=(number of bleeding episodes meeting those criteria during the efficacy period/total number of days during the efficacy period)*365.25. Efficacy period begins with the first prophylactic dose of rFIXFc and ends with the last dose (for prophylaxis or a bleeding episode). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. A bleeding episode started from the first sign of bleeding and ended ≤ 72 hours after the last treatment for the bleeding episode, within which any symptoms of bleeding at the same location or injections ≤ 72 hours apart were considered the same bleeding episode. Any injection to treat the bleeding episode taken > 72 hours after the preceding 1 was considered the first injection to treat a new bleeding episode at the same location. Any bleeding at a different location was considered a separate bleeding episode, regardless of time from last injection.
Time Frame	Up to 50 weeks +/- 7 days (efficacy period as defined in description)

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received at least 1 dose of rFIXFc; based on the number of participants whose efficacy period is of at least 1 day in duration.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	15	15
Median (Inter-Quartile Range); Unit of Measure: bleeding episodes per participant per yr
	0.00; (0.00 to 1.2)	0.00; (0.00 to 2.2)

4. Secondary Outcome

Title	Participant Assessment of Response to Injections to Treat a Bleeding Episode
Description	Participant's assessment of the response (provided by the caregiver) 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.
Time Frame	Up to 50 weeks +/- 7 days

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received at least 1 dose of rFIXFc and had ≥ 1 bleeding episode; based on the number of injections with an evaluation.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	9	11
Overall Number of Units Analyzed; Type of Units Analyzed: Injections	19	34
Measure Type: Number; Unit of Measure: percent of 1st injections w/ a response
Excellent or Good	89.5	88.2
Excellent	52.6	41.2
Good	36.8	47.1
Moderate	5.3	11.8
No Response	5.3	0

5. Secondary Outcome

Title	Physician's Global Assessment of the Participant's Response to His rFIXFc Regimen
Description	Investigators assessed each participant's response to his rFIXFc regimen using a 4-point scale: excellent=bleeding episodes responded to ≤ the usual number of injections or ≤ the usual dose of rFIXFc or the rate of breakthrough bleeding during prophylaxis was ≤ 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. Percentages are based on the total number of responses; multiple responses per participant are counted.
Time Frame	Up to 50 weeks +/- 7 days

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received ≥ 1 dose of rFIXFc; based on the number of responses.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	15	15
Overall Number of Units Analyzed; Type of Units Analyzed: Responses	48	59
Measure Type: Number; Unit of Measure: percentage of responses
Excellent	85.4	89.8
Effective	14.6	10.2
Partially Effective	0	0
Ineffective	0	0

6. Secondary Outcome

Title	Annualized rFIXFc Consumption by Type of Injection
Description	Annualized consumption of rFIXFc for prevention of bleeding (prophylactic), treatment of bleeding, and other rFIXFc injections. Consumption is calculated for the efficacy period. The efficacy period began with the first prophylactic dose of rFIXFc and ended with the last dose (regardless of the reason for dosing). Surgery/rehabilitation and PK evaluation periods were not included in the efficacy period. Annualized consumption = (total IU/kg of study treatment received during the efficacy period / total number of days during the efficacy period)*365.25. Participants who did not have a particular injection type are counted as having zero injections for that type.
Time Frame	Up to 50 weeks +/- 7 days (efficacy period as defined in description)

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received at least 1 dose of rFIXFc.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	15	15
Mean (Standard Deviation); Unit of Measure: IU/kg rFIXFc per year
Prophylactic injections	3041.5 (577.55)	3185.6 (683.71)
Injections for bleeding	147.9 (209.89)	293.8 (515.59)
Other injections	29.2 (48.93)	16.9 (44.90)

7. Secondary Outcome

Title	Number of Days From the Last Prophylaxis Injection to a Spontaneous Bleeding Episode
Description	The number of days from the last prophylaxis injection to the onset of a new spontaneous bleeding episode, analyzed across all evaluable bleeding episodes per participant and per episode, based on the efficacy period. Evaluable bleeding episodes are those for which both a date and time are available for both the onset of the bleeding episode and the previous prophylactic injection. The efficacy period begins with the first prophylactic dose of rFIXFc and ends with the last dose (for prophylaxis or a bleeding episode). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. For 'Per participant' values, the number of days from the last prophylactic injection to a spontaneous bleeding episode is averaged across all evaluable spontaneous bleeding episodes per participant.
Time Frame	Up to 50 weeks +/- 7 days (efficacy period as defined in description)

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received at least 1 dose of rFIXFc; number of participants and number of episodes were determined for participants with at least 1 evaluable spontaneous bleeding episode.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	4	6
Overall Number of Units Analyzed; Type of Units Analyzed: Evaluable Spontaneous Bleeding Episodes	5	11
Median (Inter-Quartile Range); Unit of Measure: days
Per spontaneous bleeding episode	3.97; (0.71 to 4.27)	5.55; (3.30 to 6.04)
Per participant	4.12; (2.33 to 5.30)	5.52; (4.41 to 6.04)

8. Secondary Outcome

Title	Number of Injections Required for Resolution of a Bleeding Episode
Description	The number of injections required to resolve a bleeding episode per participant and per episode, based on the efficacy period. The efficacy period begins with the first prophylactic dose of rFIXFc and ends with the last dose (for prophylaxis or a bleeding episode). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. All injections given from the initial sign of a bleeding episode, until the last date/time within the bleeding episode window are counted. The resolution of a bleeding episode is defined as no sign of bleeding following injection for the bleeding episode. For 'Per participant' values, the number of injections required to resolve each bleeding episode is averaged across all bleeding episodes per participant.
Time Frame	Up to 50 weeks +/- 7 days (efficacy period as defined in description)

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received at least 1 dose of rFIXFc; number of participants and number of episodes were determined for participants with at least 1 evaluable bleeding episode.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	9	11
Overall Number of Units Analyzed; Type of Units Analyzed: Bleeding Episodes	22	38
Median (Inter-Quartile Range); Unit of Measure: injections
Per bleeding episode	1.0; (1.0 to 1.0)	1.0; (1.0 to 2.0)
Per participant	1.0; (1.0 to 1.2)	1.0; (1.0 to 1.7)

9. Secondary Outcome

Title	Total Dose Required for Resolution of a Bleeding Episode
Description	The total dose required to resolve a bleeding episode per participant and per episode, based on the efficacy period. The efficacy period begins with the first prophylactic dose of rFIXFc and ends with the last dose (for prophylaxis or a bleeding episode). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. For 'Per bleeding episode' values, for each bleeding episode, the total dose is the sum of the doses (IU/kg) administered across all injections given to treat that bleeding episode. For 'Per participant' values, the total dose (IU/kg) used to resolve each bleeding episode is averaged across all bleeding episodes per participant.
Time Frame	Up to 50 weeks +/- 7 days (efficacy period as defined in description)

Outcome Measure Data

Analysis Population Description

Full Analysis Set: participants who received at least 1 dose of rFIXFc; number of participants and number of episodes were determined for participants who had complete information on the dose administered to treat a bleeding episode.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	9	11
Overall Number of Units Analyzed; Type of Units Analyzed: Bleeding Episodes	22	38
Median (Inter-Quartile Range); Unit of Measure: IU/kg
Per bleeding episode	65.37; (50.68 to 125.00)	89.77; (50.92 to 140.86)
Per participant	70.22; (55.40 to 97.22)	52.22; (27.03 to 161.06)

10. Secondary Outcome

Title	Maximum Plasma Activity (Cmax; One-stage Activated Partial Thromboplastin Time [aPTT] Clotting Assay)
Description	Cmax: maximum plasma FIX activity during a dosing interval. The values for Cmax were adjusted to the nominal dose of 50 IU/kg. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Time Frame	Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Outcome Measure Data

Analysis Population Description

PK Analysis Set: all participants with adequate PK data, defined as complete and evaluable PK samples through 168 hours after rFIXFc dosing. Complete means the availability of the 168-hour sample and at least enough other samples to allow for all the PK parameters to be estimated.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	11	13
Geometric Mean (95% Confidence Interval); Unit of Measure: IU/dL
	29.78; (26.18 to 33.87)	35.84; (30.56 to 42.03)

11. Secondary Outcome

Title	Terminal Half Life (t1/2; One-stage aPTT Clotting Assay)
Description	t1/2: time required for the concentration of the drug to reach half of its original value in the body. Non-compartmental methods. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Time Frame	Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Outcome Measure Data

Analysis Population Description

PK Analysis Set: all participants with adequate PK data, defined as complete and evaluable PK samples through 168 hours after rFIXFc dosing. Complete means the availability of the 168-hour sample and at least enough other samples to allow for all the PK parameters to be estimated.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	11	13
Geometric Mean (95% Confidence Interval); Unit of Measure: hours
	66.49; (55.86 to 79.14)	70.34; (60.95 to 81.17)

12. Secondary Outcome

Title	Clearance (CL; One-stage aPTT Clotting Assay)
Description	CL: the measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time. Non-compartmental methods. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Time Frame	Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Outcome Measure Data

Analysis Population Description

PK Analysis Set: all participants with adequate PK data, defined as complete and evaluable PK samples through 168 hours after rFIXFc dosing. Complete means the availability of the 168-hour sample and at least enough other samples to allow for all the PK parameters to be estimated.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	11	13
Geometric Mean (95% Confidence Interval); Unit of Measure: mL/h/kg
	4.365; (3.901 to 4.885)	3.505; (3.006 to 4.087)

13. Secondary Outcome

Title	Volume of Distribution at Steady State (Vss; One-stage aPTT Clotting Assay)
Description	Vss: volume of distribution at steady state. Non-compartmental methods. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Time Frame	Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Outcome Measure Data

Analysis Population Description

PK Analysis Set: all participants with adequate PK data, defined as complete and evaluable PK samples through 168 hours after rFIXFc dosing. Complete means the availability of the 168-hour sample and at least enough other samples to allow for all the PK parameters to be estimated.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	11	13
Geometric Mean (95% Confidence Interval); Unit of Measure: mL/kg
	365.1; (316.2 to 421.6)	289.0; (236.7 to 352.9)

14. Secondary Outcome

Title	Dose Normalized Area Under the Curve (DNAUC; One-stage aPTT Clotting Assay)
Description	DNAUC: dose normalized area under the drug concentration-time curve (extent of unmetabolized drug in circulation). Non-compartmental methods. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Time Frame	Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Outcome Measure Data

Analysis Population Description

PK Analysis Set: all participants with adequate PK data, defined as complete and evaluable PK samples through 168 hours after rFIXFc dosing. Complete means the availability of the 168-hour sample and at least enough other samples to allow for all the PK parameters to be estimated.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	11	13
Geometric Mean (95% Confidence Interval); Unit of Measure: IU*h/dL per IU/kg
	22.71; (20.32 to 25.38)	28.53; (24.47 to 33.27)

15. Secondary Outcome

Title	Mean Residence Time (MRT; One-stage aPTT Clotting Assay)
Description	MRT: the average time for all the drug molecules to reside in the body. Non-compartmental methods. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Time Frame	Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Outcome Measure Data

Analysis Population Description

PK Analysis Set: all participants with adequate PK data, defined as complete and evaluable PK samples through 168 hours after rFIXFc dosing. Complete means the availability of the 168-hour sample and at least enough other samples to allow for all the PK parameters to be estimated.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	11	13
Geometric Mean (95% Confidence Interval); Unit of Measure: hours
	83.65; (71.76 to 97.51)	82.46; (72.65 to 93.60)

16. Secondary Outcome

Title	Incremental Recovery (IR; One-stage aPTT Clotting Assay)
Description	IR for FIX activity following rFIXFc dosing: IU/dL rise in plasma FIX per IU/kg drug administered. Non-compartmental methods. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Time Frame	Baseline (28±7 days before Day 1) Prestudy FIX Dosing: predose; 30±5 min, 3 hrs±30 min, 10±2 hrs, 24±3 hrs, 48±4 hrs postdose. Day 1 rFIXFc Dosing: predose; 30±5 min, 3 hrs ±30 min, 10±2 hrs, 24±3 hrs, 72±7 hrs, 120±12 hrs, 168±16 hrs postdose.

Outcome Measure Data

Analysis Population Description

PK Analysis Set: all participants with adequate PK data, defined as complete and evaluable PK samples through 168 hours after rFIXFc dosing. Complete means the availability of the 168-hour sample and at least enough other samples to allow for all the PK parameters to be estimated.

Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description:	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
Overall Number of Participants Analyzed	11	13
Geometric Mean (95% Confidence Interval); Unit of Measure: IU/dL per IU/kg
	0.5898; (0.5152 to 0.6752)	0.7170; (0.6115 to 0.8407)

Adverse Events

Time Frame	Serious adverse events (AEs): from informed consent up to 21 days after last dose; AEs: from Baseline (28 ± 7 days prior to Day 1) up to 14 (+7) days after last dose.
Adverse Event Reporting Description	Length of rFIXFc dosing was up to 50 weeks ± 7 days. Serious and non-serious AEs that were treatment-emergent with respect to rFIXFc are presented for those subjects in the Safety Analysis Set who were treated with rFIXFc.
Arm/Group Title	Participants < 6 Years Old	Participants 6 to < 12 Years Old
Arm/Group Description	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.	At Baseline and at Day 1, participants received a single IV injection of prestudy FIX and rFIXFc, respectively, over 10 (±5) minutes at a dose of 50 IU/kg. Immediately after the last PK sampling, the first prophylactic dose of approximately 50 to 60 IU/kg was administered in clinic as an IV injection. Dose could be increased or decreased in increments of 10 IU/kg; increases to a maximum of 100 IU/kg and frequency of administration to a maximum of twice weekly, were allowed as indicated.
All-Cause Mortality
	Participants < 6 Years Old	Participants 6 to < 12 Years Old
	Affected / at Risk (%)	Affected / at Risk (%)
Total	--/--	--/--
Serious Adverse Events
	Participants < 6 Years Old	Participants 6 to < 12 Years Old
	Affected / at Risk (%)	Affected / at Risk (%)
Total	3/15 (20.00%)	1/15 (6.67%)
Gastrointestinal disorders
Duodenal Ulcer Haemorrhage † 1	0/15 (0.00%)	1/15 (6.67%)
General disorders
Device Occlusion † 1	1/15 (6.67%)	0/15 (0.00%)
Infections and infestations
Gastroenteritis † 1	1/15 (6.67%)	0/15 (0.00%)
Injury, poisoning and procedural complications
Fall † 1	2/15 (13.33%)	0/15 (0.00%)
Head Injury † 1	2/15 (13.33%)	0/15 (0.00%)
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 15.0
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Participants < 6 Years Old	Participants 6 to < 12 Years Old
	Affected / at Risk (%)	Affected / at Risk (%)
Total	12/15 (80.00%)	14/15 (93.33%)
Blood and lymphatic system disorders
Haemorrhagic Anaemia † 1	0/15 (0.00%)	1/15 (6.67%)
Iron Deficiency Anaemia † 1	1/15 (6.67%)	0/15 (0.00%)
Ear and labyrinth disorders
Ear Pain † 1	1/15 (6.67%)	1/15 (6.67%)
Gastrointestinal disorders
Cheilitis † 1	1/15 (6.67%)	0/15 (0.00%)
Constipation † 1	2/15 (13.33%)	0/15 (0.00%)
Dental Caries † 1	2/15 (13.33%)	0/15 (0.00%)
Diarrhoea † 1	1/15 (6.67%)	0/15 (0.00%)
Duodenal Ulcer † 1	0/15 (0.00%)	1/15 (6.67%)
Duodenal Ulcer Haemorrhage † 1	0/15 (0.00%)	1/15 (6.67%)
Faeces Discoloured † 1	1/15 (6.67%)	0/15 (0.00%)
Haematochezia † 1	1/15 (6.67%)	0/15 (0.00%)
Stomatitis † 1	0/15 (0.00%)	1/15 (6.67%)
Toothache † 1	0/15 (0.00%)	1/15 (6.67%)
Vomiting † 1	2/15 (13.33%)	0/15 (0.00%)
General disorders
Fatigue † 1	0/15 (0.00%)	1/15 (6.67%)
Pyrexia † 1	4/15 (26.67%)	0/15 (0.00%)
Immune system disorders
Seasonal Allergy † 1	1/15 (6.67%)	1/15 (6.67%)
Infections and infestations
Croup Infectious † 1	2/15 (13.33%)	0/15 (0.00%)
Ear Infection † 1	2/15 (13.33%)	0/15 (0.00%)
Fungal Skin Infection † 1	0/15 (0.00%)	1/15 (6.67%)
Gastroenteritis Norovirus † 1	0/15 (0.00%)	2/15 (13.33%)
Gastroenteritis Viral † 1	1/15 (6.67%)	0/15 (0.00%)
Impetigo † 1	1/15 (6.67%)	0/15 (0.00%)
Nail Infection † 1	0/15 (0.00%)	1/15 (6.67%)
Nasopharyngitis † 1	1/15 (6.67%)	6/15 (40.00%)
Oral Bacterial Infection † 1	0/15 (0.00%)	1/15 (6.67%)
Otitis Media † 1	2/15 (13.33%)	0/15 (0.00%)
Pharyngitis † 1	1/15 (6.67%)	0/15 (0.00%)
Pharyngitis Streptococcal † 1	1/15 (6.67%)	1/15 (6.67%)
Respiratory Tract Infection † 1	1/15 (6.67%)	0/15 (0.00%)
Sinusitis † 1	0/15 (0.00%)	1/15 (6.67%)
Tonsillitis † 1	1/15 (6.67%)	1/15 (6.67%)
Upper Respiratory Tract Infection † 1	1/15 (6.67%)	1/15 (6.67%)
Upper Respiratory Tract Infection Bacterial † 1	0/15 (0.00%)	1/15 (6.67%)
Urinary Tract Infection † 1	1/15 (6.67%)	0/15 (0.00%)
Varicella † 1	1/15 (6.67%)	0/15 (0.00%)
Viral Infection † 1	4/15 (26.67%)	0/15 (0.00%)
Injury, poisoning and procedural complications
Accidental Drug Intake By Child † 1	1/15 (6.67%)	0/15 (0.00%)
Bite † 1	1/15 (6.67%)	0/15 (0.00%)
Contusion † 1	1/15 (6.67%)	0/15 (0.00%)
Excoriation † 1	1/15 (6.67%)	0/15 (0.00%)
Face Injury † 1	2/15 (13.33%)	1/15 (6.67%)
Fall † 1	2/15 (13.33%)	2/15 (13.33%)
Head Injury † 1	1/15 (6.67%)	1/15 (6.67%)
Joint Injury † 1	0/15 (0.00%)	1/15 (6.67%)
Ligament Sprain † 1	0/15 (0.00%)	1/15 (6.67%)
Limb Injury † 1	0/15 (0.00%)	1/15 (6.67%)
Lip Injury † 1	1/15 (6.67%)	0/15 (0.00%)
Traumatic Haematoma † 1	1/15 (6.67%)	0/15 (0.00%)
Metabolism and nutrition disorders
Decreased Appetite † 1	1/15 (6.67%)	0/15 (0.00%)
Food Intolerance † 1	0/15 (0.00%)	1/15 (6.67%)
Musculoskeletal and connective tissue disorders
Arthralgia † 1	0/15 (0.00%)	1/15 (6.67%)
Musculoskeletal Stiffness † 1	0/15 (0.00%)	1/15 (6.67%)
Nervous system disorders
Headache † 1	0/15 (0.00%)	2/15 (13.33%)
Tremor † 1	0/15 (0.00%)	1/15 (6.67%)
Psychiatric disorders
Abnormal Behaviour † 1	1/15 (6.67%)	0/15 (0.00%)
Respiratory, thoracic and mediastinal disorders
Asthma † 1	1/15 (6.67%)	1/15 (6.67%)
Cough † 1	2/15 (13.33%)	0/15 (0.00%)
Epistaxis † 1	1/15 (6.67%)	0/15 (0.00%)
Upper-Airway Cough Syndrome † 1	1/15 (6.67%)	0/15 (0.00%)
Skin and subcutaneous tissue disorders
Dermatitis Contact † 1	1/15 (6.67%)	0/15 (0.00%)
Eczema † 1	0/15 (0.00%)	1/15 (6.67%)
Rash † 1	2/15 (13.33%)	0/15 (0.00%)
Rash Maculo-Papular † 1	0/15 (0.00%)	1/15 (6.67%)
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 15.0

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.

Results Point of Contact

• Name/Title: Bioverativ Study Medical Director
• Organization: Bioverativ
• EMail: clinicaltrials@bioverativ.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Fischer K, Kulkarni R, Nolan B, Mahlangu J, Rangarajan S, Gambino G, Diao L, Ramirez-Santiago A, Pierce GF, Allen G. Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study. Lancet Haematol. 2017 Feb;4(2):e75-e82. doi: 10.1016/S2352-3026(16)30193-4.

• Responsible Party: Sanofi ( Bioverativ Therapeutics Inc. )
• ClinicalTrials.gov Identifier: NCT01440946
• Other Study ID Numbers: 9HB02PED; 2011-003076-36 ( EudraCT Number )
• First Submitted: September 16, 2011
• First Posted: September 27, 2011
• Results First Submitted: June 1, 2015
• Results First Posted: June 18, 2015
• Last Update Posted: December 19, 2020