Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

Summary

The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses. Because trough FVIII levels are likely to be important predictors of the efficacy of prophylaxis, the focus of this study is on pharmacokinetic (PK) data.

Timeline

Start

2015-05

Primary completion

2018-09

Completion

2018-09

Outcome

Met primary endpoint

registry analysis (other test); Individualized Prophylaxis in GENA-21b vs GENA-01; p <0.0001; Rate ratio 11.89 (95% CI 7.50 to 18.86); Negative binomial regression model NCT02256917 ↗

Drugs

Indications