Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Clinical Trial
. 2020 Jan-Dec:26:1076029620950836.
doi: 10.1177/1076029620950836.

Safety, Immunogenicity, and Hemostatic Efficacy of Nonacog Gamma in Patients With Severe or Moderately Severe Hemophilia B: A Continuation Study

Jerzy Windyga  1 , Oleksandra Stasyshyn  2 , Toshko Lissitchkov  3 , Vasily Mamonov  4 , Margit Serban  5 , Luminita Rusen  6 , Bettina Ploder  7 , Srilatha Tangada  8
Affiliations
Clinical Trial

Safety, Immunogenicity, and Hemostatic Efficacy of Nonacog Gamma in Patients With Severe or Moderately Severe Hemophilia B: A Continuation Study

Jerzy Windyga et al. Clin Appl Thromb Hemost. 2020 Jan-Dec.

Abstract

This phase 3, prospective, open-label, multicenter, continuation study (NCT01286779) investigated the use of a recombinant factor IX (FIX), nonacog gamma (BAX 326, RIXUBIS®) in patients with severe or moderately severe hemophilia B. The study population included 85 patients transitioning from a phase 1/3 pivotal study (NCT01174446), a pediatric study (NCT01488994), and 30 newly recruited patients, naïve to nonacog gamma. Patients received nonacog gamma as prophylaxis treatment (standard, modified or PK-tailored) or on-demand, as determined by the investigator. Treatment was assessed for safety, immunogenicity, hemostatic efficacy and consumption. In this study, after ≥100 exposure days, nonacog gamma resulted in no treatment-related serious adverse events, and no patients developed inhibitory antibodies to FIX. Nonacog gamma was efficacious at controlling bleeding episodes, with an 89.1% overall hemostatic efficacy rating of excellent or good, and 56% of bleeds resolved with one infusion. The annualized bleeding rate was considerably lower during prophylactic treatment (median ABR of 1.3 in 108 patients) than during on-demand treatment (median ABR of 16.5 in 13 patients). These results show that in previously treated patients and nonacog gamma-naïve patients, long-term use of nonacog gamma had acceptable safety and tolerability, and was efficacious as a prophylactic treatment for the management of bleeding episodes.NCT01286779, EudraCT: 2010-022726-33.

Keywords: continuation study; factor IX; hemophilia B; hemostatic efficacy; nonacog gamma; safety.

PubMed Disclaimer

Conflict of interest statement

Declaration of Conflicting Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

Figures

Figure 1.
Figure 1.
Patient participation flowchart. After enrollment, but prior to treatment, 2 patients discontinued: 1 patient, patient decision; 1 patient, owing to screen failure. After treatment, 19 patients discontinued: 9 patients, withdrawal by patient; 5 patients, protocol violation; 2 patients, physician decision; 1 patient, scheduled surgery; 1 patient, emigrated; 1 patient, discontinued by sponsor. Abbreviation: FAS, full analysis set.
Figure 2.
Figure 2.
Hemostatic efficacy rating. The rating scale for hemostatic efficacy is described in Supplemental Table 1.
Figure 3.
Figure 3.
Number of infusions required to resolve a bleed.
See this image and copyright information in PMC

References

    1. Srivastava A, Brewer A, Mauser-Bunschoten E, et al. Treatment Guidelines Working Group on Behalf of the World Federation of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1–e47. - PubMed
    1. Key NS. Inhibitors in congenital coagulation disorders. Br J Haematol. 2004;127(4):379–391. - PubMed
    1. US Food and Drug Administration. RIXUBIS. Product information. Published 2014 Accessed October 1, 2019 http://www.fda.gov/vaccines-blood-biologics/approved-blood-products/rixubis.
    1. European Medicines Agency. RIXUBIS. Summary of product characteristics. Accessed October 1, 2019 https://www.ema.europa.eu/documents/product-information/rixubis-epar-pro....
    1. Windyga J, Lissitchkov T, Stasyshyn O, et al. Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicenter phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level ≤2%) haemophilia B. Haemophilia. 2014;20(1):15–24. - PubMed

Publication types

Associated data