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A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02053792
Recruitment Status : Completed
First Posted : February 4, 2014
Results First Posted : July 14, 2022
Last Update Posted : July 14, 2022
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemophilia B
Intervention Biological: rIX-FP
Enrollment 97
Recruitment Details  
Pre-assignment Details Approximately 115 male previously treated patients (PTPs) and previously untreated patients (PUPs) with hemophilia B were planned to be enrolled, including all eligible PTPs from CSLB-sponsored rIX-FP lead-in studies, approximately 10 PTPs who required major, nonemergency surgery, and approximately 20 PUPs.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it takes to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data. Previously untreated patients (PUPs) will administer CSL654 (rIX-FP) by intravenous infusion as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the pharmacokinetic data.
Period Title: Overall Study
Started 83 14
Completed 77 10
Not Completed 6 4
Reason Not Completed
Physician Decision             0             1
Development of High titer FIX inhibitor             0             1
Lack of Efficacy             1             0
Adverse Event             1             0
Withdrawal by Subject             2             2
The patient was not compliant to the study procedure             1             0
Patient reached minimum number of exposure days and was allowed to stop             1             0
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs) Total
Hide Arm/Group Description Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data. Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data. Total of all reporting groups
Overall Number of Baseline Participants 83 14 97
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 83 participants 14 participants 97 participants
<=18 years
30
  36.1%
14
 100.0%
44
  45.4%
Between 18 and 65 years
53
  63.9%
0
   0.0%
53
  54.6%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
CSL654 (PTPs) Number Analyzed 83 participants 0 participants 83 participants
27.7  (17.81) 27.7  (17.81)
CSL654 (PUPs) Number Analyzed 0 participants 12 participants 12 participants
1.3  (3.11) 1.3  (3.11)
[1]
Measure Analysis Population Description: Safety Population (SP) was used. It was pre-specified in the Statistical Analysis Plan to collect age data separately for PTPs and PUPs and to not calculate the age for the total overall population.
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 83 participants 14 participants 97 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
83
 100.0%
14
 100.0%
97
 100.0%
Ethnicity (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 83 participants 12 participants 95 participants
Hispanic or Latino
2
   2.4%
0
   0.0%
2
   2.1%
Not Hispanic or Latino
81
  97.6%
12
 100.0%
93
  97.9%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Analysis Population Description: SP was used
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 83 participants 12 participants 95 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
12
  14.5%
1
   8.3%
13
  13.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
3
   3.6%
0
   0.0%
3
   3.2%
White
68
  81.9%
10
  83.3%
78
  82.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   8.3%
1
   1.1%
[1]
Measure Analysis Population Description: SP was used
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Czechia Number Analyzed 83 participants 14 participants 97 participants
3 0 3
United States Number Analyzed 83 participants 14 participants 97 participants
2 4 6
Japan Number Analyzed 83 participants 14 participants 97 participants
9 0 9
Philippines Number Analyzed 83 participants 14 participants 97 participants
1 1 2
Malaysia Number Analyzed 83 participants 14 participants 97 participants
2 0 2
Spain Number Analyzed 83 participants 14 participants 97 participants
6 0 6
Canada Number Analyzed 83 participants 14 participants 97 participants
1 0 1
Austria Number Analyzed 83 participants 14 participants 97 participants
4 1 5
Italy Number Analyzed 83 participants 14 participants 97 participants
10 2 12
South Africa Number Analyzed 83 participants 14 participants 97 participants
2 0 2
Israel Number Analyzed 83 participants 14 participants 97 participants
15 0 15
Bulgaria Number Analyzed 83 participants 14 participants 97 participants
4 0 4
Australia Number Analyzed 83 participants 14 participants 97 participants
2 3 5
France Number Analyzed 83 participants 14 participants 97 participants
14 0 14
Germany Number Analyzed 83 participants 14 participants 97 participants
8 3 11
1.Primary Outcome
Title Total Number of Participants Who Developed Inhibitors Against Factor IX (FIX)
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population (SP) consisted of all participants who received ≥ 1 dose of rIX-FP during this study
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 83 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
1
   8.3%
2.Primary Outcome
Title Mean Incremental Recovery of a 50 IU/kg Dose of CSL654 in Previously Untreated Patients (PUPs)
Hide Description Incremental Recovery: The increase in plasma concentration per IU/kg of factor administered.
Time Frame Approximately 30 minutes after infusion of CSL654
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) Population consisted of participants who received ≥ 1 dose of rIX-FP and had a sufficient number of analyzable PK blood samples (ie, ≥ 1 PK parameter calculated) for the PK assessment of rIX-FP. Participants were excluded from the analyses if an insufficient number of analyzable PK samples was obtained to permit the evaluation of ≥ 1 PK parameter. Only PUPs were analyzed for this endpoint.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 0 8
Mean (Standard Deviation)
Unit of Measure: (IU/dL)/(IU/kg)
Uncorrected FIX Activity 1.295  (0.3578)
Baseline-corrected FIX Activity 1.231  (0.3729)
3.Secondary Outcome
Title Total Annualized Bleeding Rate (ABR) by Prophylaxis Regimen in Previously Treated Patients (PTPs)
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Population (EP) consisted of all participants in the Safety Population (SP). Only PTPs were analyzed for this endpoint. Participants were assigned under multiple regimens during the study, but were counted only once in any given regimen group. Annualized Bleeding Rate was derived only for subjects who were on the given regimen for at least 12 weeks.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 83 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Participant
7-Day Regimen Number Analyzed 41 participants 0 participants
2.89  (3.115)
10-Day Regimen Number Analyzed 23 participants 0 participants
2.72  (2.827)
14-Day Regimen Number Analyzed 48 participants 0 participants
2.72  (3.395)
21-Day Regimen Number Analyzed 11 participants 0 participants
1.19  (1.572)
4.Secondary Outcome
Title Spontaneous ABR by Prophylaxis Regimen in PTPs
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
EP. Only PTPs were analyzed for this endpoint. Participants may be assigned under multiple regimens during the study, but will be counted only once in any given regimen group. Annualized Bleeding Rate is derived only for subjects who are on the given regimen for at least 12 weeks.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 83 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Participant
7-Day Regimen Number Analyzed 41 participants 0 participants
0.95  (1.672)
10-Day Regimen Number Analyzed 23 participants 0 participants
0.98  (1.689)
14-Day Regimen Number Analyzed 48 participants 0 participants
1.32  (2.205)
21-Day Regimen Number Analyzed 11 participants 0 participants
0.60  (1.408)
5.Secondary Outcome
Title Average Amount of CSL654 (rIX-FP) Consumed Per Month Per Subject During Routine Prophylaxis Treatment.
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.
Hide Outcome Measure Data
Hide Analysis Population Description
EP
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 83 12
Mean (Standard Deviation)
Unit of Measure: IU/kg per month
181.8  (35.16) 188.53  (24.096)
6.Secondary Outcome
Title Percentage of Participants With at Least One Treatment Emergent Adverse Event (TEAE) and the Percentage of Participants With at Least One CSL654-related TEAE
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it takes to achieve 50 EDs.
Hide Outcome Measure Data
Hide Analysis Population Description
SP
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 83 12
Measure Type: Number
Unit of Measure: percentage of participants
AEs 89.2 91.7
Related AEs 2.4 16.7
7.Secondary Outcome
Title Number of Participants With Investigator's Overall Clinical Assessment of Hemostatic Efficacy for the Treatment of Major Bleeding Events With CSL654 in PUPs
Hide Description The investigator will rate the efficacy of the rIX-FP treatment based on a hemostatic efficacy four point rating scale of excellent, good, moderate, or poor/no response.
Time Frame Up to 3 years or the time it takes to achieve 50 EDs
Hide Outcome Measure Data
Hide Analysis Population Description
EP. This endpoint was for PUPs only. No major bleeding events were reported. Therefore, no data for this endpoint.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 0 12
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
8.Secondary Outcome
Title Total ABR for On-demand Regimen vs. 14-Day Regimen in PTPs
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
EP. Only PTPs were analyzed for this endpoint. Participants may be assigned under multiple regimens during the study, but will be counted only once in any given regimen group. Number of participants analyzed are those who received at least 12 weeks of treatment in the respective regimen.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 14 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Subject
On-demand Regimen 17.51  (7.130)
14-Day Regimen 3.01  (4.204)
9.Secondary Outcome
Title Spontaneous ABR for On-demand Regimen vs. 14-Day Regimen in PTPs
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
EP. Only PTPs were analyzed for this endpoint. Participants may be assigned under multiple regimens during the study, but will be counted only once in any given regimen group. Number of participants analyzed are those who received at least 12 weeks of treatment in the respective regimen.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 14 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Subject
On-demand Regimen 13.17  (5.873)
14-Day Regimen 1.93  (3.363)
10.Secondary Outcome
Title Total ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
EP. Only PTPs were analyzed for this endpoint. Participants may be assigned under multiple regimens during the study, but will be counted only once in any given regimen group. Number of participants analyzed are those who received at least 12 weeks of treatment in the respective regimen.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 41 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Subject
7-Day Regimen 1.12  (1.697)
14-Day Regimen 2.19  (3.000)
11.Secondary Outcome
Title Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. 14-Day Regimen in PTPs
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
EP. Only PTPs were analyzed for this endpoint. Participants may be assigned under multiple regimens during the study, but will be counted only once in any given regimen group. Number of participants analyzed are those who received at least 12 weeks of treatment in the respective regimen.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 41 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Subject
7-Day Regimen 0.49  (1.135)
14-Day Regimen 1.33  (2.349)
12.Secondary Outcome
Title Total ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
EP. Only PTPs were analyzed for this endpoint. Participants may be assigned under multiple regimens during the study, but will be counted only once in any given regimen group. Number of participants analyzed are those who received at least 12 weeks of treatment in the respective regimen.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 49 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Subject
7-Day Regimen 1.31  (1.868)
(10 or 14)-Day Regimen 2.01  (2.700)
13.Secondary Outcome
Title Spontaneous ABR for Subjects >=12 Years: 7-Day Regimen vs. (10 or 14)-Day Regimen in PTPs
Hide Description [Not Specified]
Time Frame For PTPs: up to 5 years or the time it takes to achieve 100 exposure days (EDs).
Hide Outcome Measure Data
Hide Analysis Population Description
EP. Only PTPs were analyzed for this endpoint. Participants may be assigned under multiple regimens during the study, but will be counted only once in any given regimen group. Number of participants analyzed are those who received at least 12 weeks of treatment in the respective regimen.
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description:
Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
Overall Number of Participants Analyzed 49 0
Mean (Standard Deviation)
Unit of Measure: Bleeds/Year/Subject
7-Day Regimen 0.57  (1.192)
(10 or 14)-Day Regimen 1.05  (2.022)
Time Frame For PTPs: up to 5 years or the time it took to achieve 100 exposure days (EDs). For PUPs: up to 3 years or the time it took to achieve 50 EDs.
Adverse Event Reporting Description Safety Population was used.
 
Arm/Group Title CSL654 (PTPs) CSL654 (PUPs)
Hide Arm/Group Description Previously treated patients (PTPs) will administer CSL654 (rIX-FP) by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 5 years or the time it took to reach 100 exposure days (EDs). The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data. Previously untreated patients (PUPs) administered CSL654 (rIX-FP) intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter up to 3 years or the time it takes to achieve 50 EDs. The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.
All-Cause Mortality
CSL654 (PTPs) CSL654 (PUPs)
Affected / at Risk (%) Affected / at Risk (%)
Total   1/83 (1.20%)      0/12 (0.00%)    
Hide Serious Adverse Events
CSL654 (PTPs) CSL654 (PUPs)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   17/83 (20.48%)      5/12 (41.67%)    
Blood and lymphatic system disorders     
Iron deficiency anaemia  1  2/83 (2.41%)  4 0/12 (0.00%)  0
Anaemia  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Gastrointestinal disorders     
Haemorrhoids  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Large intestine polyp  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Oesophagitis  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Infections and infestations     
Abscess Jaw  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Appendicitis  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Peritonsillar abscess  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Pneumonia  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Device related infection  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Influenza  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Injury, poisoning and procedural complications     
Head injury  1  3/83 (3.61%)  4 1/12 (8.33%)  1
Contusion  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Extradural Haematoma  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Muscle injury  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Road traffic accident  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Femur fracture  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Post procedural haemorrhage  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Investigations     
Serum ferritin decreased  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Anti factor IX antibody increased  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Musculoskeletal and connective tissue disorders     
Haemarthrosis  1  2/83 (2.41%)  2 0/12 (0.00%)  0
Arthralgia  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Arthritis  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Haemophilic arthropathy  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Joint Swelling  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Nervous system disorders     
Haemorrhage intracranial  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Renal and urinary disorders     
Haematuria  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Epistaxis  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Vascular disorders     
Haematoma  1  1/83 (1.20%)  1 0/12 (0.00%)  0
Peripheral Ischaemia  1  1/83 (1.20%)  1 0/12 (0.00%)  0
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
CSL654 (PTPs) CSL654 (PUPs)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   69/83 (83.13%)      11/12 (91.67%)    
Congenital, familial and genetic disorders     
Factor VII deficiency  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Gastrointestinal disorders     
Dental caries  1  10/83 (12.05%)  12 1/12 (8.33%)  1
Diarrhoea  1  6/83 (7.23%)  6 1/12 (8.33%)  1
Toothache  1  5/83 (6.02%)  6 0/12 (0.00%)  0
Constipation  1  4/83 (4.82%)  5 2/12 (16.67%)  3
Teething  1  0/83 (0.00%)  0 2/12 (16.67%)  2
Abdominal pain  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Gingival disorder  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Vomiting  1  3/83 (3.61%)  3 1/12 (8.33%)  1
General disorders     
Pyrexia  1  15/83 (18.07%)  24 6/12 (50.00%)  9
Catheter site bruise  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Influenza like illness  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Immune system disorders     
Hypersensitivity  1  0/83 (0.00%)  0 1/12 (8.33%)  3
Selective IgA immunodeficiency  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Infections and infestations     
Nasopharyngitis  1  15/83 (18.07%)  25 3/12 (25.00%)  14
Gastroenteritis  1  10/83 (12.05%)  10 1/12 (8.33%)  1
Influenza  1  6/83 (7.23%)  6 1/12 (8.33%)  2
Sinusitis  1  5/83 (6.02%)  6 0/12 (0.00%)  0
Tonsillitis  1  5/83 (6.02%)  8 0/12 (0.00%)  0
Bronchitis  1  4/83 (4.82%)  8 2/12 (16.67%)  6
Upper respiratory tract infection  1  4/83 (4.82%)  6 3/12 (25.00%)  7
Ear infection  1  3/83 (3.61%)  3 1/12 (8.33%)  2
Rhinitis  1  3/83 (3.61%)  3 2/12 (16.67%)  2
Viral infection  1  2/83 (2.41%)  2 1/12 (8.33%)  2
Pharyngitis streptococcal  1  0/83 (0.00%)  0 2/12 (16.67%)  3
Hand-foot-and-mouth disease  1  0/83 (0.00%)  0 1/12 (8.33%)  2
Otitis media  1  0/83 (0.00%)  0 1/12 (8.33%)  2
Croup infectious  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Eye infection  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Eyelid boil  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Gastrointestinal infection  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Otitis media chronic  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Otitis media viral  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Paronychia  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Respiratory syncytial virus infection  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Respiratory tract infection viral  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Streptococcal infection  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Urinary tract infection  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Wound infection  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Injury, poisoning and procedural complications     
Contusion  1  7/83 (8.43%)  12 2/12 (16.67%)  6
Laceration  1  6/83 (7.23%)  7 2/12 (16.67%)  2
Fall  1  4/83 (4.82%)  4 4/12 (33.33%)  6
Head injury  1  3/83 (3.61%)  4 3/12 (25.00%)  5
Mouth injury  1  0/83 (0.00%)  0 2/12 (16.67%)  2
Arthropod bite  1  0/83 (0.00%)  0 1/12 (8.33%)  3
Accident  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Skin abrasion  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Soft tissue injury  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Tongue injury  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Metabolism and nutrition disorders     
Dehydration  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Musculoskeletal and connective tissue disorders     
Arthralgia  1  25/83 (30.12%)  35 0/12 (0.00%)  0
Pain in extremity  1  8/83 (9.64%)  10 0/12 (0.00%)  0
Musculoskeletal pain  1  6/83 (7.23%)  8 0/12 (0.00%)  0
Soft tissue swelling  1  0/83 (0.00%)  0 2/12 (16.67%)  5
Haemophilic arthropathy  1  2/83 (2.41%)  3 1/12 (8.33%)  1
Muscle spasms  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Nervous system disorders     
Headache  1  11/83 (13.25%)  21 0/12 (0.00%)  0
Tongue biting  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Psychiatric disorders     
Irritability  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Respiratory, thoracic and mediastinal disorders     
Cough  1  6/83 (7.23%)  7 2/12 (16.67%)  2
Skin and subcutaneous tissue disorders     
Rash  1  2/83 (2.41%)  5 2/12 (16.67%)  4
Dermatitis diaper  1  0/83 (0.00%)  0 2/12 (16.67%)  2
Urticaria  1  0/83 (0.00%)  0 2/12 (16.67%)  2
Ingrowing nail  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Nail bed inflammation  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Palmar-plantar erythrodysaesthesia syndrome  1  0/83 (0.00%)  0 1/12 (8.33%)  1
Vascular disorders     
Hypertension  1  5/83 (6.02%)  5 0/12 (0.00%)  0
1
Term from vocabulary, MedDRA 21.0
Indicates events were collected by systematic assessment
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: CSL Behring
Phone: 610-878-4000
EMail: clinicaltrials@cslbehring.com
Layout table for additonal information
Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT02053792    
Other Study ID Numbers: CSL654_3003
2012-005489-37 ( EudraCT Number )
First Submitted: January 29, 2014
First Posted: February 4, 2014
Results First Submitted: May 17, 2022
Results First Posted: July 14, 2022
Last Update Posted: July 14, 2022