Clinical trials summary

 
The ELOCTA clinical trial programme is one of the largest in haemophilia A

The A-LONG trial demonstrated that ELOCTA was well tolerated and efficacious in the prevention and treatment of bleeding events in adults and adolescents with severe haemophilia A1

Trials
  • A-LONG was an open-label, multinational, phase 3 trial evaluating the safety, efficacy and PK properties of ELOCTA in 165 previously treated males with severe haemophilia A1
  • The individualised prophylaxis regimen was adjusted to maintain trough level >1–3 IU/dL above baseline, as clinically indicated to prevent bleeding. Dose adjusted in a range of 25-65 IU/kg and interval of every 3-5 days1

IU, international units; PK, pharmacokinetics.

Reference

1. Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014 Jan;123(3):317–25.

The Kids A-LONG study demonstrated that twice-weekly injections with ELOCTA was well tolerated and yielded low bleeding rates in children with severe haemophilia A1

Trials

Kids A-LONG was an open-label, multinational, phase 3 trial evaluating the safety, efficacy and pharmacokinetic properties of ELOCTA in 71 previously treated children with severe haemophilia A1

IU, international units; PK, pharmacokinetics.

Reference

1. Young G, Mahlangu J, Kulkarni R, Nolan B, Liesner R, Pasi J, et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe haemophilia A. J Thromb Haemost. 2015 Jun;13(6):967–77.

ASPIRE was a phase 3, open-label, multicentre extension trial evaluating the long-term safety and efficacy of ELOCTA in subjects completing A-LONG and Kids A-LONG1,2

Trials

 IU, international units.

References

1. Nolan B, Mahlangu J, Perry D, Young G, Liesner R, Konkle B, et al. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016 Jan;22(1):72–80.

2. ELOCTA Summary of Product Characteristics. 2020.

Clinical trials summary

 
The ELOCTA clinical trial programme is one of the largest in haemophilia A

The A-LONG trial demonstrated that ELOCTA was well tolerated and efficacious in the prevention and treatment of bleeding events in adults and adolescents with severe haemophilia A1

Trials
  • A-LONG was an open-label, multinational, phase 3 trial evaluating the safety, efficacy and PK properties of ELOCTA in 165 previously treated males with severe haemophilia A1
  • The individualised prophylaxis regimen was adjusted to maintain trough level >1–3 IU/dL above baseline, as clinically indicated to prevent bleeding. Dose adjusted in a range of 25-65 IU/kg and interval of every 3-5 days1

IU, international units; PK, pharmacokinetics.

Reference

1. Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014 Jan;123(3):317–25.

The Kids A-LONG study demonstrated that twice-weekly injections with ELOCTA was well tolerated and yielded low bleeding rates in children with severe haemophilia A1

Trials

Kids A-LONG was an open-label, multinational, phase 3 trial evaluating the safety, efficacy and pharmacokinetic properties of ELOCTA in 71 previously treated children with severe haemophilia A1

IU, international units; PK, pharmacokinetics.

Reference

1. Young G, Mahlangu J, Kulkarni R, Nolan B, Liesner R, Pasi J, et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe haemophilia A. J Thromb Haemost. 2015 Jun;13(6):967–77.

ASPIRE was a phase 3, open-label, multicentre extension trial evaluating the long-term safety and efficacy of ELOCTA in subjects completing A-LONG and Kids A-LONG1,2

Trials

 IU, international units.

References

1. Nolan B, Mahlangu J, Perry D, Young G, Liesner R, Konkle B, et al. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016 Jan;22(1):72–80.

2. ELOCTA Summary of Product Characteristics. 2020.