Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B

Grifols

Status and phase: Completed; Phase 2

Conditions: Hemophilia B

Treatments: Drug: BeneFIX

Study type: Interventional

Funder types: Industry

Identifiers

NCT03091751

IG-404-1

Details and patient eligibility

About

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.

Full description

Two pharmacokinetic assessments (studies) were carried out in the same subjects during a previous clinical trial. The first pharmacokinetic study (PK1) was performed after a single dose of AlphaNine. The second pharmacokinetic study (PK2) was performed following 26 Weeks of AlphaNine treatment after PK1. To compare AlphaNine with BeneFIX, a third pharmacokinetic study (PK3) (current study) was performed after a single dose of BeneFIX administered following a 7- to 15-day wash-out period.

The main objective of the PK3 study was to assess the pharmacokinetic profile of BeneFIX and compare to the pharmacokinetic profile of AlphaNine from the PK2 study.

Enrollment: 13 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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13 participants in 1 patient group

BeneFIX

Experimental group

Description:

BeneFIX is a recombinant FIX provided in a vial containing 100 IU/mL lyophilized nonacog alfa accompanied with solvent for reconstitution and injection.

Treatment:

Drug: BeneFIX