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History of Changes for Study: NCT02053792
A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
Latest version (submitted November 5, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 February 2, 2014 None (earliest Version on record)
2 March 5, 2014 Recruitment Status, Study Status and Contacts/Locations
3 April 7, 2014 Study Status and Contacts/Locations
4 May 5, 2014 Study Status and Contacts/Locations
5 June 4, 2014 Study Status and Contacts/Locations
6 August 27, 2014 Study Status and Contacts/Locations
7 November 12, 2014 Study Status and Contacts/Locations
8 June 10, 2015 Outcome Measures, Study Status, Contacts/Locations, Eligibility, Arms and Interventions, Study Design and Study Description
9 July 2, 2015 Study Status
10 August 31, 2015 Contacts/Locations and Study Status
11 November 24, 2015 Study Status
12 January 26, 2016 Study Status
13 February 26, 2016 Study Status
14 March 8, 2016 Study Status
15 April 25, 2016 Study Status
16 May 26, 2016 Study Status
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18 July 7, 2016 Study Status
19 August 4, 2016 Study Status
20 September 12, 2016 Study Status
21 October 5, 2016 Study Status
22 November 4, 2016 Study Status
23 December 13, 2016 Study Status
24 January 6, 2017 Study Status
25 February 7, 2017 Study Status
26 March 24, 2017 Study Status
27 May 1, 2017 Outcome Measures, Contacts/Locations, Eligibility, Arms and Interventions, Study Description and Study Status
28 May 12, 2017 Contacts/Locations and Study Status
29 August 9, 2017 Contacts/Locations and Study Status
30 September 5, 2017 Study Status
31 October 10, 2017 Study Status
32 November 14, 2017 Study Status and Contacts/Locations
33 January 12, 2018 Study Status
34 March 18, 2018 Contacts/Locations and Study Status
35 April 11, 2018 Study Status
36 June 1, 2018 Study Status
37 June 22, 2018 Contacts/Locations and Study Status
38 June 26, 2018 Contacts/Locations and Study Status
39 July 31, 2018 Study Status
40 August 31, 2018 Study Status
41 September 21, 2018 Contacts/Locations and Study Status
42 October 29, 2018 Study Status and Contacts/Locations
43 November 26, 2018 Study Status
44 January 3, 2019 Study Status
45 March 1, 2019 Study Status
46 March 29, 2019 Study Status
47 April 22, 2019 Contacts/Locations and Study Status
48 June 4, 2019 Study Status and Contacts/Locations
49 July 2, 2019 Study Status
50 August 27, 2019 Study Status
51 October 14, 2019 Study Status
52 November 13, 2019 Contacts/Locations and Study Status
53 January 14, 2020 Study Status
54 February 25, 2020 Recruitment Status, Contacts/Locations and Study Status
55 March 25, 2020 Study Status
56 April 2, 2020 Study Design and Study Status
57 April 21, 2020 Study Status
58 May 1, 2020 Study Status
59 June 29, 2020 Study Status
60 August 6, 2020 Study Status
61 August 31, 2020 Study Status
62 October 12, 2020 Study Status
63 November 21, 2020 Study Status
64 December 21, 2020 Study Status
65 January 21, 2021 Study Status
66 March 1, 2021 Study Status
67 April 2, 2021 Study Status
68 June 2, 2021 Study Status
69 June 16, 2021 Study Status
70 June 29, 2021 Recruitment Status, Study Status and Study Design
71 August 4, 2021 Study Status
72 November 5, 2021 Outcome Measures, Study Status and Study Design
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Study NCT02053792
Submitted Date:  February 2, 2014 (v1)

Open or close this module Study Identification
Unique Protocol ID: CSL654_3003
Brief Title: A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
Official Title: A Phase 3b Open-label, Multicenter, Safety and Efficacy Extension Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B
Secondary IDs: 2012-005489-37 [EudraCT Number]
Open or close this module Study Status
Record Verification: February 2014
Overall Status: Not yet recruiting
Study Start: February 2014
Primary Completion: December 2016 [Anticipated]
Study Completion:
First Submitted: January 29, 2014
First Submitted that
Met QC Criteria:
February 2, 2014
First Posted: February 4, 2014 [Estimate]
Last Update Submitted that
Met QC Criteria:
February 2, 2014
Last Update Posted: February 4, 2014 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: CSL Behring
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring:
Open or close this module Study Description
Brief Summary:

This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B. The study will include subjects who previously completed a CSL-sponsored rIX-FP lead-in study and subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study.

A surgical prophylaxis substudy will examine the efficacy of rIX-FP in subjects with hemophilia B who are undergoing non-emergency major or minor surgery.

Detailed Description:
Open or close this module Conditions
Conditions: Hemophilia B
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 3
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 95 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: rIX-FP

Subjects will administer rIX-FP by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 3 years.

During the initial 6 months of the treatment period, subjects will administer rIX-FP as prophylaxis treatment using a treatment interval based on their experience during a previous CSL-sponsored rIX-FP study. After this initial 6 months, subjects will continue to administer rIX-FP as prophylaxis treatment using either the same treatment interval or a different treatment interval, as determined by the investigator. The treatment interval may be changed at each 6-month follow-up assessment.

On-demand treatment with rIX-FP will be used for all bleeding episodes requiring treatment.

Subjects may participate in a surgical 'substudy' in which rIX-FP may be administered before, during and after surgery.

The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and pharmacokinetic data.

Biological: rIX-FP
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Main study: Total number of subjects who develop inhibitors against factor IX (FIX)
[ Time Frame: Approximately 3 years ]

2. Surgery substudy: Investigator's overall clinical assessment of hemostatic efficacy for surgical prophylaxis
[ Time Frame: Immediately after surgery (0 hours) and at up to 3 timepoints thereafter up to 72 hours or discharge, whichever is earliest. ]

Clinical assessment of hemostatic efficacy to be based on a four point ordinal scale (excellent, good, moderate, poor / none)
Secondary Outcome Measures:
1. Main study: Comparison of annualized bleeding rate between different prophylaxis treatment intervals.
[ Time Frame: Approximately 3 years ]

Comparison of annualized bleeding rate by prophylaxis treatment interval for spontaneous treated bleeds and total treated bleeds.
2. Main study: Comparison of annualized bleeding rate between 2 different prophylaxis treatment intervals and on-demand treatment.
[ Time Frame: At least 3 months and up to 6 months ]

For subjects from the lead-in study (CSL654_3001 [NCT01496274] on-demand arm), comparison of the annualized bleeding rate of spontaneous treated bleeds between routine prophylaxis treatment in this study (CSL654_3003) and:

  • On-demand treatment during the lead-in study (CSL654_3001).
  • Routine prophylaxis treatment with the different treatment interval used by the subject in the lead-in study (CSL654_3001).
3. Main study: rIX-FP consumed per month per subject during routine prophylaxis treatment.
[ Time Frame: Approximately 3 years ]

4. Surgery substudy: The frequency of adverse events (AEs) related to rIX-FP
[ Time Frame: 28 days after surgery (major surgery) or up to hospital discharge (minor surgery) ]

5. Surgery substudy: Total number of subjects who develop inhibitors against FIX
[ Time Frame: 28 days after surgery (major surgery) or up to hospital discharge (minor surgery) ]

6. Surgery substudy: Total number of subjects who develop antibodies against rIX-FP.
[ Time Frame: 28 days after surgery (major surgery) or up to hospital discharge (minor surgery) ]

7. Surgery substudy: Predicted and intraoperative estimated blood loss.
[ Time Frame: Predicted blood loss and intraoperative estimated blood loss to be determined before surgery and at the end of surgery, respectively ]

The predicted blood loss (mL) is based on blood loss expected from a non-hemophilic individual undergoing the same type / extent of procedure.

The estimated blood loss (mL) is the anesthesiologist's record of estimated blood loss during the procedure.

8. Surgery substudy: Predicted and actual transfusion requirements.
[ Time Frame: Predicted and actual transfusion requirements to be determined before and at the end of surgery, respectively ]

9. Surgery substudy: Change in hemoglobin levels between baseline, intraoperatively and postoperatively (major surgery only).
[ Time Frame: Before, during and up to 28 days after surgery ]

Open or close this module Eligibility
Minimum Age:
Maximum Age: 70 Years
Sex: Male
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion criteria:

Main study inclusion criteria:

Either:

  • Completed a CSL-sponsored rIX-FP (CSL654) study, including study CSL654_3001 [NCT01496274] or study CSL654_3002 [NCT01662531].

Or:

  • Scheduled to have a major non-emergency surgery within approximately 8 weeks from the anticipated date of receiving the first rIX-FP injection.
  • Not previously completed a CSL-sponsored rIX-FP lead-in study.
  • Male, 12 to 70 years of age.
  • Documented severe hemophilia B (FIX activity of ≤ 2%), or confirmed at screening by the central laboratory.
  • Subjects who have received FIX products (plasma-derived and / or recombinant FIX) for > 150 exposure days (EDs), confirmed by their treating physician.
  • No confirmed history of FIX inhibitor formation at screening by the central laboratory

Surgery substudy inclusion criterion:

  • Must require non-emergency surgery

Exclusion criteria:

Main study exclusion criteria:

  • Currently receiving a therapy not permitted during the study.
  • Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.

For subjects who have previously completed a CSL-sponsored rIX-FP study:

  • Unwilling to participate in the study for a total of 100 exposure days.

For subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study:

  • Known hypersensitivity (ie, allergic reaction or anaphylaxis) to any FIX product or hamster protein.
  • Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
  • Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
  • Low platelet count, kidney or liver disease.
  • Human immunodeficiency virus positive with a CD4 count < 200/mm3.

The substudy does not have any additional exclusion criteria.

Open or close this module Contacts/Locations
Central Contact Person: Clinical Trial Registration Coordinator
Email: clinicaltrials@cslbehring.com
Study Officials: Program Director
Study Director
CSL Behring
Locations:
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links:
Available IPD/Information:

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