Clinical trial NCT01863758: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Severe Haemophilia A
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2013-05-29
Enrollment: 66
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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.

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.

Primary outcomes:
measure: Annualized Number of Bleeding Episodes (BE) in Phase II
timeFrame: Beginning to the end of Phase II (6 months)
Secondary outcomes:
measure: Annualized Number of Spontaneous Bleeding Episodes (BE) in Phase II
timeFrame: Beginning to the end of Phase II (6 months)
measure: Annualized Number of Bleeding Episodes (BE) in Phase II in Participants With ≤ 2 Treatments/Week
timeFrame: Beginning to the end of Phase II (6 months)
measure: Median Dosing Interval During Individually Tailored Prophylaxis
timeFrame: Beginning to the end of Phase II (6 months)
measure: Dosage Per Week in Phase II
timeFrame: Beginning to the end of Phase II (6 months)
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Medical University Vienna
city: Plovdiv
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment
city: Sofia
country: Bulgaria
facility: Specialized Hospital for Active Treatment
city: Varna
country: Bulgaria
facility: Multiprofile Hospital for Active Treatment
city: Berlin
country: Germany
facility: Vivantes Hospital in Friedrichshain
city: Heidelberg
country: Germany
facility: SRH Kurpfalzklinik Heidelberg GMBH
city: Budapest
country: Hungary
facility: Hungarian National Healthcare Center
city: Debrecen
country: Hungary
facility: University of Debrecen, Medical and Health Science Center
city: Białystok
country: Poland
facility: University Teaching Hospital in Bialystok, Teaching Department of Hematology with a Subdepartment of Vascular Diseases
city: Gdańsk
country: Poland
facility: University Clinical Center, Teaching Department of Hematology and Transplantology
city: Torun
country: Poland
facility: Nicolaus Copernicus Municipal Specialist Hospital, Department of Hematology
city: Warsaw
country: Poland
facility: Institute of Hematology and Transfusion Medicine, Depart. of Hemostatic Disorders and Internal Diseases
city: Bucharest
country: Romania
facility: Sanador SRL
city: Timisoara
country: Romania
facility: Louis Turcanu Emergency Clinical Children's Hospital
city: Bratislava
country: Slovakia
facility: University Hospital Saint Cyril and Metod
city: Martin
country: Slovakia
facility: University Hospital Martin, Department of Hematology and Transfusiology
city: Basingstoke
country: United Kingdom
facility: Basingstoke and North Hampshire Hospital, Hemophilia, Hemostasis and Thrombosis Center
city: London
country: United Kingdom
facility: Royal London Hospital, Barts and the London Hemophilia Center
city: Manchester
country: United Kingdom
facility: Manchester Royal Infirmary, Department of Clinical Hematology
city: Sheffield
country: United Kingdom
facility: Royal Hallamshire Hospital
Study officials:
References: