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

Octapharma

Status and phase: Completed; Phase 3

Conditions: Severe Haemophilia A

Treatments: Biological: Human cl rhFVIII

Study type: Interventional

Funder types: Industry

Identifiers

NCT02256917

GENA-21B

Details and patient eligibility

About

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.

Enrollment: 58 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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58 participants in 1 patient group

Human-cl rhFVIII

Experimental group

Treatment:

Biological: Human cl rhFVIII