A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
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| ClinicalTrials.gov Identifier: NCT01662531 |
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Recruitment Status :
Completed
First Posted : August 10, 2012
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 pharmacokinetics, safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children who have previously received factor replacement therapy for hemophilia B.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia B | Biological: rIX-FP | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 27 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase III Open-label, Multicenter, Pharmacokinetic, Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Previously Treated Children With Hemophilia B |
| Study Start Date : | January 2013 |
| Actual Primary Completion Date : | October 2014 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hemophilia
| Arm | Intervention/treatment |
|---|---|
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Experimental: rIX-FP
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) will be administered by IV infusion as routine weekly prophylaxis and episodic treatment for bleeding episodes.
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Biological: rIX-FP
Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) |
Primary Outcome Measures :
- Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product [ Time Frame: 30 minutes after infusion ]Incremental recovery (IU/dL/IU/kg) is defined as the FIX activity (IU/dL) 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. Recovery values were baseline-corrected for pre-infusion plasma FIX activity. Incremental recovery was measured following a single intravenous dose of 50 IU/kg rIX-FP on Day 1. Analysis of previous FIX product was conducted at the beginning of the study in a subset of subjects who had no historical pharmacokinetic (PK) data of their previous FIX product. For the PK assessment, the previous FIX product was administered by IV infusion after approximately 4 days following the last FIX treatment, prior to any dosing of rIX-FP. The formal PK population consisted of subjects who received at least 1 dose of rIX-FP for PK assessment and for whom a sufficient number of analyzable PK samples had been obtained to permit the evaluation of the PK profile of rIX-FP.
- Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values.
- Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast) [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]
AUClast following a single intravenous dose of 50 IU/kg rIX-FP or previous FIX product.
FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values.
- Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]FIX activity was measured at a central laboratory using validated one-stage clotting method. FIX levels were not corrected for baseline values. Clearance is normalized for body weight.
- Number of Subjects Developing Inhibitors to Factor IX (FIX) [ Time Frame: 12 months ]Inhibitor formation was defined as any inhibitor (≥0.6 BU [Bethesda Units]/mL) identified and confirmed by retesting.
Secondary Outcome Measures :
- Number of Subjects With Treatment-related Adverse Events [ Time Frame: 12 months ]
- Number of Subjects Developing Antibodies Against rIX-FP [ Time Frame: 12 months ]Antibodies to rIX-FP were measured using a direct-binding enzyme-linked immunosorbent assay (ELISA).
- Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis [ Time Frame: Approximately 12 months ]For each bleeding episode that required treatment, the number of episodes that required one, two or more than two infusions of rIX-FP to achieve hemostasis
- Consumption of rIX-FP During Routine Prophylaxis [ Time Frame: 12 months ]Consumption of rIX-FP during routine prophylaxis is expressed as the total prophylaxis dose per month.
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| Ages Eligible for Study: | up to 11 Years (Child) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Male subjects, younger than 12 years old.
- Severe hemophilia B (Factor IX [FIX] activity of ≤ 2%).
- Body weight ≥ 10 kg.
- Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for > 150 exposure days (EDs) (6 to < 12 years), and > 50 EDs (< 6 years).
- 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.
Exclusion Criteria:
- Known hypersensitivity to any FIX product or hamster protein.
- Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
- Kidney or liver disease.
- 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): NCT01662531
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: | NCT01662531 |
| Other Study ID Numbers: |
CSL654_3002 2011-006032-23 ( EudraCT Number ) |
| First Posted: | August 10, 2012 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 |