Assess the Safety and Efficacy of Individually Tailored Prophylaxis With 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

NCT01863758

GENA-21

Details and patient eligibility

About

To compare the number of breakthrough bleeds under tailored prophylaxis with Human cell line recombinant factor FVIII (Human-cl rhFVIII) with the historical bleeding rate from patients who received Human-cl rhFVIII as on demand treatment.

Full description

There were 3 phases in this study: (1) An initial pharmacokinetic (PK) assessment in which participants received a single infusion of 60±5 IU/kg of Human-cl rhFVIII; blood samples were collected for 72 hours following the infusion. (2) Prophylactic Treatment-Phase I during which participants received infusions of 30-40 IU/kg of human-cl rhFVIII every other day or 3x/week for 1-3 months. (3) Prophylactic Treatment-Phase II during which the dose and dosing interval were determined individually from data gathered in the initial PK assessment. The maximum dosing interval with a dose of ≤ 60-80 IU/kg that maintains a trough level of ≥ 0.01 IU/mL was determined. Participants were treated for 6 months.

Enrollment

66 patients

Sex

Male

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Severe haemophilia A (FVIII:C < 1%) according to medical history.
Male patients ≥ 18 years old.
Previous treatment with a FVIII concentrate (regular prophylaxis with good compliance or on-demand treatment) for at least 150 exposure days (EDs).
Good documentation regarding dosing and bleeding frequency in the 6 months preceding study start.
Immunocompetence (CD4+ count > 200/microliter).
HIV-negative, if positive, viral load < 200 particles/microliter or < 400,000 copies/mL.
Freely given written informed consent

Exclusion criteria