Dosage and PD Study of Eftrenonacog-alfa

NCT04590950 | Completed

• 1 other identifier: APHP191124
• Study Type: observational
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the performance of different methods for measuring factor IX activity levels in haemophilia B patients treated with eftrenonacog-alfa and assess its pharmacodynamics (PD) in a real-life setting.

Conditions

Severe Haemophilia B

Keywords

Chromogenic assay; Extended half-life product; Fc fusion protein; One-stage clotting assay; Pharmacodynamics

Outcome Measures

Primary Outcomes (1)

• FIX activity levels

  2 chromogenic assays (Bio phen FIX and Rox FIX) and 2 chronometric assays (CK Prest with standard human plasma or FIX deficient plasma supplemented with eftrenonacog-alfa as calibrators)

  One month

Secondary Outcomes (5)

• Thrombin generation assay

  One month

• Thrombin generation assay

  One month

• Thrombin generation assay

  One month

• Thrombin generation assay

  One month

• Thrombin generation assay

  One month

Study Arms (1)

Single-group study

Severe haemophilia B patients substituted with eftrenonacog-alfa

Other: Non interventional study

Interventions

Non interventional study OTHER

Non interventional study

Single-group study

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Severe haemophilia B patients

You may qualify if:

• severe haemophilia B patients
• treated with eftrenonacog-alfa

You may not qualify if:

• any other haemostatic or replacement therapy

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Service d'hématologie biologique - Hôpital Cochin

Paris, 75014, France

Location

Related Publications (8)

• Peyvandi F, Garagiola I, Boscarino M, Ryan A, Hermans C, Makris M. Real-life experience in switching to new extended half-life products at European haemophilia centres. Haemophilia. 2019 Nov;25(6):946-952. doi: 10.1111/hae.13834. Epub 2019 Aug 16.

  PMID: 31418967 BACKGROUND

• Sommer JM, Buyue Y, Bardan S, Peters RT, Jiang H, Kamphaus GD, Gray E, Pierce GF. Comparative field study: impact of laboratory assay variability on the assessment of recombinant factor IX Fc fusion protein (rFIXFc) activity. Thromb Haemost. 2014 Nov;112(5):932-40. doi: 10.1160/TH13-11-0971. Epub 2014 Aug 21.

  PMID: 25144892 BACKGROUND

• Kitchen S, Tiefenbacher S, Gosselin R. Factor Activity Assays for Monitoring Extended Half-Life FVIII and Factor IX Replacement Therapies. Semin Thromb Hemost. 2017 Apr;43(3):331-337. doi: 10.1055/s-0037-1598058. Epub 2017 Mar 6.

  PMID: 28264199 BACKGROUND

• Dargaud Y, Beguin S, Lienhart A, Al Dieri R, Trzeciak C, Bordet JC, Hemker HC, Negrier C. Evaluation of thrombin generating capacity in plasma from patients with haemophilia A and B. Thromb Haemost. 2005 Mar;93(3):475-80. doi: 10.1160/TH04-10-0706.

  PMID: 15735797 BACKGROUND

• Bowyer AE, Shepherd MF, Kitchen S, Maclean RM, Makris M. Measurement of extended half-life recombinant factor IX products in clinical practice. Int J Lab Hematol. 2019 Apr;41(2):e46-e49. doi: 10.1111/ijlh.12953. Epub 2018 Dec 7. No abstract available.

  PMID: 30536630 BACKGROUND

• Collins PW, Fischer K, Morfini M, Blanchette VS, Bjorkman S; International Prophylaxis Study Group Pharmacokinetics Expert Working Group. Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia. 2011 Jan;17(1):2-10. doi: 10.1111/j.1365-2516.2010.02370.x. Epub 2010 Aug 22.

  PMID: 20731726 BACKGROUND

• Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. Haemophilia. 2013 Jan;19(1):e1-47. doi: 10.1111/j.1365-2516.2012.02909.x. Epub 2012 Jul 6.

  PMID: 22776238 BACKGROUND

• Atsou S, Furlan F, Duchemin J, Ellouze S, Sourdeau E, Launois A, Roussel-Robert V, Stieltjes N, Combe S, Fontenay M, Curis E, Jourdi G. Pharmacodynamics of eftrenonacog-alfa (rFIX-Fc) in severe hemophilia B patients: A real-life study. Eur J Pharmacol. 2021 Jan 15;891:173764. doi: 10.1016/j.ejphar.2020.173764. Epub 2020 Nov 27.

  PMID: 33249076 RESULT

Biospecimen

Retention: SAMPLES WITHOUT DNA

Citrated plasma samples

MeSH Terms

Conditions

Hemophilia B

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2020
• First Posted: October 19, 2020
• Study Start: December 18, 2019
• Primary Completion: March 31, 2020
• Study Completion: March 31, 2020
• Last Updated: March 4, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)