Prospective, Open-label, Multi-centre Phase 3b Study to Assess the Efficacy and Safety of Personalized Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A

Octapharma20 sites in 8 countries58 target enrollmentMay 2015

ConditionsSevere Haemophilia A

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Severe Haemophilia A
Sponsor: Octapharma
Enrollment: 58
Locations: 20
Primary Endpoint: Annualized Total Bleeding Rate of Individually Tailored Prophylaxis
Status: Completed
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

May 2015

End Date

September 2018

Last Updated

5 years ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Octapharma

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Severe Haemophilia A (FVIII:C \< 1%)
•Male patients \>= 18 years of age
•Previous treatment with a FVIII concentrate for at least 150 EDs
•Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start
•Immunocompetence (CD4+ count \> 200/uL)

Exclusion Criteria

•Any coagulation disorder other than Haemophilia A
•Present of past FVIII inhibitor activity
•Severe liver or kidney disease

Outcomes

Primary Outcomes

Annualized Total Bleeding Rate of Individually Tailored Prophylaxis

Time Frame: 6 months

Total annualized bleeding rate (ABR) of individually tailored prophylaxis (GENA-21b) compared to historical bleeding rate in patients having received on-demand treatment (GENA-01) with Human-cl rhFVIII

Secondary Outcomes

Annualized Total Bleeding Rate in Patients With 2x/Week (or Less) Prophylaxis(6 months)
Annualized Spontaneous Bleeding Rate of Individually Tailored Prophylaxis(6 months)
Mean Prophylactic Dosing Interval(6 months)
Mean Residence Time (MRT) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
AUC Divided by the Dose (AUCnorm) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
Clearance (CL) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
In-vivo Recovery (IVR) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
Number of Patients With Adverse Events (AEs)(At each study visit over the study duration (7-9 months))
Median Prophylactic Dosing Interval(6 months)
Volume of Distribution at Steady State (Vss) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)
Usage of Human-cl rhFVIII (FVIII IU/kg BW Per Week Per Patient)(6 months)
Half Life (t1/2) of Human-cl rhFVIII(Before injection (within 1 h before injection) and up to 72 h (± 2 h) after the end of injection)