A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
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ClinicalTrials.gov Identifier: NCT02053792 |
Recruitment Status :
Completed
First Posted : February 4, 2014
Results First Posted : July 14, 2022
Last Update Posted : July 14, 2022
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Sponsor:
CSL Behring
Information provided by (Responsible Party):
CSL Behring
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Study Type | Interventional |
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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 |
Participant Flow
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) |
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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 |
Baseline Characteristics
Arm/Group Title | CSL654 (PTPs) | CSL654 (PUPs) | Total | |
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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 | |
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[Not Specified]
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 83 participants | 14 participants | 97 participants | |
<=18 years |
30 36.1%
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14 100.0%
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44 45.4%
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Between 18 and 65 years |
53 63.9%
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0 0.0%
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53 54.6%
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>=65 years |
0 0.0%
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0 0.0%
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0 0.0%
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Age, Continuous
[1] Mean (Standard Deviation) Unit of measure: Years |
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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.
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 83 participants | 14 participants | 97 participants | |
Female |
0 0.0%
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0 0.0%
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0 0.0%
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Male |
83 100.0%
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14 100.0%
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97 100.0%
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Ethnicity (NIH/OMB)
[1] Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 83 participants | 12 participants | 95 participants | |
Hispanic or Latino |
2 2.4%
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0 0.0%
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2 2.1%
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Not Hispanic or Latino |
81 97.6%
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12 100.0%
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93 97.9%
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Unknown or Not Reported |
0 0.0%
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0 0.0%
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0 0.0%
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[1]
Measure Analysis Population Description: SP was used
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Race (NIH/OMB)
[1] Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 83 participants | 12 participants | 95 participants | |
American Indian or Alaska Native |
0 0.0%
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0 0.0%
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0 0.0%
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Asian |
12 14.5%
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1 8.3%
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13 13.7%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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0 0.0%
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0 0.0%
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Black or African American |
3 3.6%
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0 0.0%
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3 3.2%
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White |
68 81.9%
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10 83.3%
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78 82.1%
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More than one race |
0 0.0%
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0 0.0%
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0 0.0%
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Unknown or Not Reported |
0 0.0%
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1 8.3%
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1 1.1%
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[1]
Measure Analysis Population Description: SP was used
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
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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 |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Results Point of Contact
Name/Title: | Study Director |
Organization: | CSL Behring |
Phone: | 610-878-4000 |
EMail: | clinicaltrials@cslbehring.com |
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 |