Phase 2
N=22
Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B
Hemophilia B
Bottom Line
View on ClinicalTrials.gov: NCT03091751 ↗Enrolled (actual)
22
Serious AEs
0.0%
Results posted
Jul 2017
Primary outcome: Primary: Mean Difference of Area Under the Curve (AUC): BeneFIX Compared to AlphaNine — 10.41 IU x hour/dL — p=0.77
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- BeneFIX (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Grifols Biologicals, LLC
- Primary completion
- Oct 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Difference of Area Under the Curve (AUC): BeneFIX Compared to AlphaNine |
10.41 | 0.77 |
| PRIMARY Mean Difference of In Vivo Recovery: BeneFIX Compared to AlphaNine |
0.3068 | 0.002 sig |
| PRIMARY Mean Difference of Terminal Half-Life: BeneFIX Compared to AlphaNine |
-3.3 | 0.30 |
| PRIMARY Mean Difference of Clearance: BeneFIX Compared to AlphaNine |
-0.0058 | 0.16 |
| PRIMARY Mean Difference of Mean Residence Time (MRT): BeneFIX Compared to AlphaNine |
-7.609 | 0.02 sig |
Summary
The goal of this non-randomized, multi-center study in subjects with severe hereditary haemophilia B was to determine and compare the pharmacokinetic and safety profiles of BeneFIX in subjects having had 2 prior pharmacokinetic assessments with AlphaNine.
Eligibility Criteria
Key Inclusion Criteria
- Participated in the previous study "Efficacy and safety of factor IX (FIX) contained in Alphananine in patients with severe hereditary haemophilia B":
- Congenital deficiency in Factor IX (FIX)
- FIX residual activity of ≤2% of normal
- Had required FIX-containing products in the past and in clinical records that were collected data to assess a reliable estimation of at least 150 treatment exposure days to previous products
- Was able to receive treatment for more than 10 days for a 6-month period
Key Exclusion Criteria
- Received a dose of FIX in the 7 days prior to the infusion
- FIX inhibitor level of >0.5 Bethesda units (BU) or clinically relevant presence in the past (≥5 BU)
- Active bleeding at the moment of infusion
- Had a known allergic reaction to any BeneFIX component
- Exhibited symptoms of any intercurrent infection (ie, fever, chills, nausea) at the time of the first infusion
- Had any disease that might affect the distribution or metabolism of FIX and which could affect interpretation of the study (such as non-controlled diabetes mellitus)
- Had non-controlled arterial hypertension
- Had abnormal renal function (creatinine >1.5 mg/dL)
- Had documented liver cirrhosis or any hepatic disorder with alanine aminotransferase (ALT) levels 2.5x upper limit of normal (ULN )
- Prevision to be concomitantly treated with other FIX-containing products
- Had conditions that might affect subject compliance (survival-limiting [in 2 year time] diseases, alcohol or other drug abuse, etc.)
- Unable to provide a storage plasma sample before the first dose of BeneFIX
Data sourced from ClinicalTrials.gov (NCT03091751). 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.