Brief Title
Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A
Official Title
Prospective, Open-label, Multicenter Phase 3b Study to Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Previously Treated Adult Patients With Severe Haemophilia A
Brief Summary
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.
Detailed 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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Annualized Number of Bleeding Episodes (BE) in Phase II
Secondary Outcome
Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II
Condition
Severe Haemophilia A
Intervention
Human-cl rhFVIII
Study Arms / Comparison Groups
Human-cl rhFVIII
Description: Up to 60-80 IU/kg of intravenous Human-cl rhFVIII was administered at an individually determined dose and dose interval.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
66
Start Date
August 2013
Completion Date
January 2015
Primary Completion Date
January 2015
Eligibility Criteria
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: - Any coagulation disorder other than haemophilia A. - Present or past FVIII inhibitor activity (> 0.6 Bethesda Unit [BU]) - Severe liver or kidney disease.
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Austria
Location Countries
Austria
Administrative Informations
NCT ID
NCT01863758
Organization ID
GENA-21
Responsible Party
Sponsor
Study Sponsor
Octapharma
Study Sponsor
, ,
Verification Date
July 2017