A Safety and Efficacy 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: NCT01496274 |
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Recruitment Status :
Completed
First Posted : December 21, 2011
Results First Posted : May 9, 2016
Last Update Posted : May 9, 2016
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Sponsor:
CSL Behring
Information provided by (Responsible Party):
CSL Behring
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Brief Summary:
This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia B | Biological: rIX-FP | Phase 2 Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 63 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase II/III Open-label, Multicenter, Safety and Efficacy Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) in Subjects With Hemophilia B |
| Study Start Date : | February 2012 |
| Actual Primary Completion Date : | July 2014 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hemophilia
| Arm | Intervention/treatment |
|---|---|
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Experimental: Prophylaxis
Routine weekly prophylaxis and episodic treatment for bleeding episodes. An individualized dosing interval may be tested in sub-group subjects during the 2nd part of the trial. Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention. |
Biological: rIX-FP
Recombinant IX-FP (rIX-FP) is a fusion protein linking coagulation factor IX with albumin, and will be administered by intravenous administration |
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Experimental: On-demand
Episodic treatment for bleeding episodes during the first 6 months then switch to routine weekly prophylaxis for a further 6 months Subjects may participate in a surgical 'sub-study' in which rIX-FP may be administered prior to, during and after surgical intervention.
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Biological: rIX-FP
Recombinant IX-FP (rIX-FP) is a fusion protein linking coagulation factor IX with albumin, and will be administered by intravenous administration |
Primary Outcome Measures :
- Change in Frequency of Spontaneous Bleeding Events Between On-demand and Prophylaxis Treatments (Annualized) [ Time Frame: Up to 26 weeks for on-demand regimen, and between 1 and 17 months for prophylaxis regimen. ]Subjects in the on-demand arm received on-demand dosing with rIX-FP for up to 26 weeks (on-demand regimen), and then received weekly prophylaxis with rIX-FP for the remainder of the study (prophylaxis regimen). The effectiveness of prophylaxis in comparison to on-demand therapy was investigated by comparing the same subject's annualized spontaneous bleeding rate (AsBR) during the on-demand regimen and during the prophylaxis regimen.
- Number of Subjects Developing Inhibitors Against Factor IX (FIX) [ Time Frame: Up to 27.7 months (maximum) ]The number of participants developing inhibitors against factor IX (FIX) along with the 95% Clopper-Pearson confidence interval, are summarized for subjects with 50 or more exposure days (EDs) to rIX-FP, and for all participants in the study.
Secondary Outcome Measures :
- The Frequency of Related Adverse Events [ Time Frame: For the duration of the study; median 20.27 months. ]The percentage of participants experiencing treatment-related adverse-events (TEAEs).
- Number of Subjects Developing Antibodies Against rIX-FP [ Time Frame: For the duration of the study; median 20.27 months. ]
- Proportion of Bleeding Episodes Requiring One or ≤ Two Injections of rIX-FP to Achieve Hemostasis [ Time Frame: For the duration of the study; median 20.27 months. ]Number of injections required to achieve hemostasis expressed as a percentage of the bleeding episodes requiring treatment.
- Investigator's Overall Clinical Assessment of Hemostatic Efficacy for Treatment of Bleeding Episodes, Based on a Four Point Ordinal Scales (Excellent, Good, Moderate, Poor/No Response) [ Time Frame: For the duration of the study; median 20.27 months ]Number of bleeding episodes requiring treatment that resulted in hemostatic efficacy of excellent, good, moderate, poor/no response, according to the Investigator's clinical assessment of hemostatic efficacy, expressed as a percentage of the bleeding episodes requiring treatment.
- rIX-FP Consumed Per Month While Maintaining Assigned Prophylactic Treatment Interval During Routine Prophylaxis. [ Time Frame: Median 269, 240, 386 and 316 days, respectively (see Description) ]Time frame: For Prophylaxis Arm 7-, 10- and 14-day regimens, median 269, 240 and 386 days respectively. For On-demand Arm, prophylaxis regimen, median 316 days.
- Incremental Recovery of rIX-FP [ Time Frame: 336 hours ]Pharmacokinetic (PK) data are presented for a single 50 IU/kg dose of rIX-FP.
- Half-life (t1/2) of a Single Dose of rIX-FP [ Time Frame: 336 hours ]PK data are presented for a single 50 IU/kg dose of rIX-FP.
- Area Under the Curve (AUC) [ Time Frame: 336 hours ]AUC to the last sample with quantifiable drug concentration (AUClast) of a single dose of rIX-FP. PK data are presented for a single 50 IU/kg dose of rIX-FP.
- Clearance of a Single Dose of rIX-FP [ Time Frame: 336 hours ]PK data are presented for a single 50 IU/kg dose of rIX-FP.
- Investigator's (or Surgeon's) Overall Clinical Assessment of Hemostatic Efficacy for Surgical Prophylaxis, Based on a Four Point Ordinal Scale (Excellent, Good, Moderate, Poor/No Response) [ Time Frame: Up to 14 days after surgery ]Number of surgical events treated prophylactically with rIX-FP that resulted in hemostatic efficacy of excellent, good, moderate, poor/no response, according to the Investigator's (surgeon's) overall assessment of hemostatic efficacy for surgical prophylaxis.
- Annualized Spontaneous Bleeding Events Compared Between 7 Day Prophylactic and Extended Regimens [ Time Frame: During treatment, between median 240 and 386 days per subject. ]Median number of spontaneous bleeds per year per subject comparing 7-, 10- and 14- day prophylactic regimens.
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| Ages Eligible for Study: | 12 Years to 65 Years (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Male subjects, 12 to 65 years old
- Severe hemophilia B (FIX activity of ≤ 2%)
- Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs)
- No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX
- Written informed consent for study participation
- On-demand subjects only, who have experienced a minimum average of 2 non-trauma induced bleeding episodes requiring treatment with a FIX product during the previous 6 or 3 months
Exclusion Criteria:
- Known hypersensitivity to any FIX product or hamster protein
- Known congenital or acquired coagulation disorder other than congenital FIX deficiency
- HIV positive subjects with a CD4 count < 200/mm3
- Low platelet count, kidney or liver dysfunction
- Recent life-threatening bleeding episode
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01496274
Locations
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Sponsors and Collaborators
CSL Behring
Investigators
| Study Director: | Program Director | CSL Behring |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | CSL Behring |
| ClinicalTrials.gov Identifier: | NCT01496274 |
| Other Study ID Numbers: |
CSL654_3001 2011-002415-28 ( EudraCT Number ) |
| First Posted: | December 21, 2011 Key Record Dates |
| Results First Posted: | May 9, 2016 |
| Last Update Posted: | May 9, 2016 |
| Last Verified: | April 2016 |
Additional relevant MeSH terms:
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Hemophilia A Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked |