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Trial record 63 of 398 for:    bleeding episodes

A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B

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ClinicalTrials.gov Identifier: NCT01361126
Recruitment Status : Completed
First Posted : May 26, 2011
Results First Posted : May 9, 2016
Last Update Posted : May 9, 2016
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hemophilia B
Intervention Biological: Recombinant Coagulation Factor IX Albumin Fusion Protein
Enrollment 17
Recruitment Details  
Pre-assignment Details A total of 17 subjects were screened and enrolled in the study. Fifteen subjects participated in the pharmacokinetic (PK) evaluation period and were administered a dose of 25 IU/kg rIX-FP. Two subjects did not participate in the PK evaluation period because PK data for these 2 subjects were available from the previous phase 1 study (CSL654_2001).
Arm/Group Title Prophylactic On-demand
Hide Arm/Group Description For the PK evaluation, subjects received a single intravenous (IV) infusion of Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) at a dose of 25 IU/kg. After completion of the PK evaluation period, subjects entered the treatment period and were administered a single IV infusion of rIX-FP once a week at a dose of 15 to 35 IU/kg, or at a dose determined by the investigator. The dose was adjusted up to 75 IU/kg to maintain the trough FIX activity level > 1% between infusions. For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg. After completion of the PK evaluation period, subjects entered the treatment period and were administered 1 or more IV infusions of rIX-FP at a dose of at least 25 IU/kg to treat minor, moderate or major bleeding episodes. The dose of rIX-FP was calculated by the investigator.
Period Title: Overall Study
Started 13 4
Completed 13 2
Not Completed 0 2
Reason Not Completed
Sponsor decision             0             2
Arm/Group Title Prophylactic On-demand Total
Hide Arm/Group Description For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg. After completion of the PK evaluation period, subjects entered the treatment period and were administered a single IV infusion of rIX-FP once a week at a dose of 15 to 35 IU/kg, or at a dose determined by the investigator. The dose was adjusted up to 75 IU/kg to maintain the trough FIX activity level > 1% between infusions. For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg. After completion of the PK evaluation period, subjects entered the treatment period and were administered 1 or more IV infusions of rIX-FP at a dose of at least 25 IU/kg to treat minor, moderate or major bleeding episodes. The dose of rIX-FP was calculated by the investigator. Total of all reporting groups
Overall Number of Baseline Participants 13 4 17
Hide Baseline Analysis Population Description
Safety Population
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 13 participants 4 participants 17 participants
23.2  (9.4) 35.8  (9.7) 26.1  (10.7)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 13 participants 4 participants 17 participants
< 18 years 3 0 3
≥ 18 years 10 4 14
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 13 participants 4 participants 17 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
13
 100.0%
4
 100.0%
17
 100.0%
1.Primary Outcome
Title Number of Subjects With Treatment-related Adverse Events
Hide Description The causal relationship of each adverse event to rIX-FP was assessed by the Investigator.
Time Frame Approximately 20 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title All Subjects
Hide Arm/Group Description:
Subjects received rIX-FP as prophylactic treatment once a week or on-demand to treat bleeding episodes administered by IV infusion for 20 weeks.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: participants
0
2.Primary Outcome
Title Number of Subjects With Inhibitors Against Factor IX (FIX)
Hide Description The presence of inhibitors against FIX was assessed by the central laboratory by a FIX potency assay. To quantify anti-FIX neutralizing antibodies, the Bethesda assay with the Nijmegen modification was used, and the results expressed as Bethesda Units per mL (BU/mL). A positive inhibitor test is >=0.6 BU/mL.
Time Frame Baseline, Day 10 and Weeks 4, 12 and 20
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title All Subjects
Hide Arm/Group Description:
Subjects received rIX-FP as prophylactic treatment once a week or on-demand to treat bleeding episodes administered by IV infusion for 20 weeks.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: participants
0
3.Primary Outcome
Title Number of Subjects Who Developed Antibodies to rIX-FP
Hide Description Antibodies against rIX-FP were detected using a direct binding enzyme-linked immunosorbent assay (ELISA).
Time Frame Pre-dose, Day 10 and Weeks 4, 12, and 20
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title All Subjects
Hide Arm/Group Description:
Subjects received rIX-FP as prophylactic treatment once a week or on-demand to treat bleeding episodes administered by IV infusion for 20 weeks.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: participants
0
4.Secondary Outcome
Title Area Under the Curve to the Last Sample With Quantifiable Drug Concentration (AUC0-t) After a Single Dose of rIX-FP
Hide Description The plasma concentrations of rIX-FP were measured as FIX activity using a validated, 1-stage assay in a central laboratory for a quantification range from 0.25 to 150% (or 0.25 IU/dL to 150 IU/dL). The PK population comprised all subjects who received at least 1 dose of rIX-FP and for whom a sufficient number of analyzable PK samples had been obtained in order to permit the evaluation of the PK profile of rIX-FP, and who did not receive a dose of rIX-FP or any other FIX product for the treatment of a bleed during the PK sampling period.
Time Frame Pre-dose and up to 14 days after rIX-FP infusion.
Hide Outcome Measure Data
Hide Analysis Population Description
PK population
Arm/Group Title Prophylactic On-demand
Hide Arm/Group Description:
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
Overall Number of Participants Analyzed 10 3
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: h*IU/dL
2915
(11.3%)
2960
(12.4%)
5.Secondary Outcome
Title Half-life (t1/2) of a Single Dose of rIX-FP
Hide Description [Not Specified]
Time Frame Pre-dose and up to 14 days after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
PK population
Arm/Group Title Prophylactic On-demand
Hide Arm/Group Description:
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
Overall Number of Participants Analyzed 10 3
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: hours
77.7
(25.2%)
138
(37.1%)
6.Secondary Outcome
Title Incremental Recovery of rIX-FP at 30 Minutes Following Infusion of rIX-FP
Hide Description Incremental recovery (IU/mL/IU/kg) is defined as FIX activity (IU/mL) obtained 30 minutes following infusion, per dose of (IU/kg) infusion. FIX activity was measured at a central laboratory using validated one-stage clotting method.
Time Frame 30 minutes after infusion
Hide Outcome Measure Data
Hide Analysis Population Description
PK population
Arm/Group Title Prophylactic On-demand
Hide Arm/Group Description:
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
Overall Number of Participants Analyzed 10 3
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: IU/dL/IU/kg
1.47
(8.2%)
1.35
(4.9%)
7.Secondary Outcome
Title Clearance of a Single Dose of rIX-FP
Hide Description [Not Specified]
Time Frame Pre-dose and up to 14 days after rIX-FP infusion
Hide Outcome Measure Data
Hide Analysis Population Description
PK population
Arm/Group Title Prophylactic On-demand
Hide Arm/Group Description:
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg.
Overall Number of Participants Analyzed 10 3
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: mL/h/kg
0.749
(14.0%)
0.699
(10.6%)
8.Secondary Outcome
Title Breakthrough Bleeding Events
Hide Description Number of breakthrough bleeding events (spontaneous bleeding events) requiring treatment per subject in subjects receiving prophylactic treatment regimen with rIX-FP
Time Frame Week 9 to approximately Week 20
Hide Outcome Measure Data
Hide Analysis Population Description
Per protocol population
Arm/Group Title Prophylactic
Hide Arm/Group Description:
For the PK evaluation, subjects received a single IV infusion of rIX-FP at a dose of 25 IU/kg. After completion of the PK evaluation period, subjects entered the treatment period and were administered a single IV infusion of rIX-FP once a week at a dose of 15 to 35 IU/kg, or at a dose determined by the investigator. The dose was adjusted up to 75 IU/kg to maintain the trough FIX activity level > 1% between infusions.
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: Events per subject
0.2  (0.38)
Time Frame 20 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title All Subjects
Hide Arm/Group Description Subjects received rIX-FP administered by IV infusion as prophylactic treatment once a week or on demand to treat bleeding episodes for 20 weeks.
All-Cause Mortality
All Subjects
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
All Subjects
Affected / at Risk (%) # Events
Total   0/17 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
All Subjects
Affected / at Risk (%) # Events
Total   14/17 (82.35%)    
Eye disorders   
Conjunctivitis  1  1/17 (5.88%)  1
Eye pain  1  1/17 (5.88%)  1
Gastrointestinal disorders   
Dyspepsia  1  1/17 (5.88%)  1
General disorders   
Injection site swelling  1  1/17 (5.88%)  3
Injection site erythema  1  1/17 (5.88%)  1
Injection site haemorrhage  1  1/17 (5.88%)  1
Infections and infestations   
Upper respiratory tract infection  1  2/17 (11.76%)  4
Pharyngitis  1  1/17 (5.88%)  1
Tonsillitis  1  1/17 (5.88%)  1
Injury, poisoning and procedural complications   
Hand fracture  1  2/17 (11.76%)  2
Laceration  1  2/17 (11.76%)  2
Contusion  1  1/17 (5.88%)  1
Head injury  1  1/17 (5.88%)  1
Injury  1  1/17 (5.88%)  1
Limb injury  1  1/17 (5.88%)  1
Radius fracture  1  1/17 (5.88%)  1
Investigations   
Weight decreased  1  1/17 (5.88%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  5/17 (29.41%)  11
Synovitis  1  1/17 (5.88%)  2
Bone pain  1  1/17 (5.88%)  1
Chondropathy  1  1/17 (5.88%)  1
Joint range of motion decreased  1  1/17 (5.88%)  1
Muscle spasms  1  1/17 (5.88%)  1
Nervous system disorders   
Headache  1  3/17 (17.65%)  3
Dizziness  1  1/17 (5.88%)  1
Skin and subcutaneous tissue disorders   
Rash follicular  1  1/17 (5.88%)  1
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.
CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.
Results Point of Contact
Name/Title: Clinical Trial Disclosure Manager
Organization: CSL Behring
Phone: Use email contact
Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01361126     History of Changes
Other Study ID Numbers: CSL654_2004
2010-023793-39 ( EudraCT Number )
First Submitted: May 25, 2011
First Posted: May 26, 2011
Results First Submitted: April 3, 2016
Results First Posted: May 9, 2016
Last Update Posted: May 9, 2016