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

NCT03091751 | Completed Phase 2 Results FDA Drug

• 1 other identifier: IG-404-1
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Hemophilia B

Keywords

Factor IX (FIX)

Outcome Measures

Primary Outcomes (5)

• Mean Difference of Area Under the Curve (AUC): BeneFIX Compared to AlphaNine

  BeneFIX pharmacokinetic parameter of area under the curve (AUC 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 study).

  Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.

• Mean Difference of In Vivo Recovery: BeneFIX Compared to AlphaNine

  BeneFIX pharmacokinetic parameter of in vivo recovery was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).

  Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.

• Mean Difference of Terminal Half-Life: BeneFIX Compared to AlphaNine

  BeneFIX pharmacokinetic parameter of terminal half-life was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).

  Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.

• Mean Difference of Clearance: BeneFIX Compared to AlphaNine

  BeneFIX pharmacokinetic parameter of clearance was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).

  Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.

• Mean Difference of Mean Residence Time (MRT): BeneFIX Compared to AlphaNine

  BeneFIX pharmacokinetic parameter of mean residence time (MRT 0-inf) was assessed and compared to the AlphaNine pharmacokinetic parameter (PK2 Study).

  Baseline (prior to the infusion), and at 15 and 30 minutes, 1, 3, 6, 9, 24, 48, 72, and 74 hours following a single dose infusion of BeneFIX administered following a 7 to 15 day wash-out period.

Study Arms (1)

BeneFIX

EXPERIMENTAL

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

Drug: BeneFIX

Interventions

BeneFIX DRUG

BeneFIX is a recombinant FIX that contains nonacog alfa, reconstituted in solvent and administered as a single dose of 65-75 IU/kg.

Also known as: recombinant coagulation factor IX

BeneFIX

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Grifols Biologicals, LLC: lead

Study Sites (3)

Medical University Pleven

Pleven, 5800, Bulgaria

Location

National Center of Haematology

Sofia, 1000, Bulgaria

Location

Medical University, University Hospital "Sveta Marina",

Varna, 9010, Bulgaria

Location

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Henry Li, PhD
• Organization: Grifols Therapeutics Inc

henry.li@grifols.com

+1 919 316 6042

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2017
• First Posted: March 27, 2017
• Study Start: August 1, 2005
• Primary Completion: October 1, 2009
• Study Completion: October 1, 2009
• Last Updated: July 25, 2017
• Results First Posted: July 25, 2017

Record last verified: 2017-06

Locations

BU (3)