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

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ClinicalTrials.gov Identifier: NCT01440946
Recruitment Status : Completed
First Posted : September 27, 2011
Results First Posted : June 18, 2015
Last Update Posted : September 25, 2018
Sponsor:
Collaborator:
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemophilia B
Interventions Drug: rFIXFc
Drug: FIX
Enrollment 30
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Participants < 6 Years Old Participants 6 to < 12 Years Old
Hide 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
Arm/Group Title Participants < 6 Years Old Participants 6 to < 12 Years Old Total
Hide 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
Hide 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%
1.Primary Outcome
Title Occurence of Factor IX (FIX) Inhibitor Development
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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)
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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)
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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
Hide Outcome Measure Data
Hide 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
Hide 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
Time Frame Up to 50 weeks +/- 7 days (efficacy period as defined in description)
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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)
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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)
Hide Outcome Measure Data
Hide 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
Hide 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
Hide 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)
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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.
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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.
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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.
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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.
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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.
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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.
Hide Outcome Measure Data
Hide 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
Hide 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)
Hide 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.
Hide Outcome Measure Data
Hide 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
Hide 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)
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
Hide 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   --/--   --/-- 
Show Serious Adverse Events Hide 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
Show Other (Not Including Serious) Adverse Events Hide 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
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
Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01440946     History of Changes
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: September 25, 2018