Phase 3
N=27
A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT01662531 ↗Enrolled (actual)
27
Serious AEs
14.8%
Results posted
May 2016
Primary outcome: Primary: Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product — 1.0114; 0.9506; 1.0600; 0.7379 (IU/dL)/(IU/kg)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rIX-FP (Biological)
- Age
- Pediatric
- Sex
- Male
- Sponsor
- CSL Behring
- Primary completion
- Oct 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product |
1.0114; 0.9506; 1.0600; 0.7379; 0.6764; 0.7925 | — |
| PRIMARY Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product |
91.4492; 89.6124; 92.7962; 18.6291; 19.8816; 17.6550 | — |
| PRIMARY Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast) |
4156.7037; 3891.4820; 4368.8810; 718.9386; 676.5414; 751.9143 | — |
| PRIMARY Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product |
1.1119; 1.1841; 1.0589; 6.4007; 7.1576; 5.8119 | — |
| PRIMARY Number of Subjects Developing Inhibitors to Factor IX (FIX) |
— | — |
| SECONDARY Number of Subjects With Treatment-related Adverse Events |
26; 0 | — |
| SECONDARY Number of Subjects Developing Antibodies Against rIX-FP |
— | — |
| SECONDARY Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis |
94; 40; 54; 9; 5; 4 | — |
| SECONDARY Consumption of rIX-FP During Routine Prophylaxis |
205.071; 213.517; 198.314 | — |
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.
Eligibility 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 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.
Data sourced from ClinicalTrials.gov (NCT01662531). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.