Clinical trial NCT01712438: Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients
Official title: Immunogenicity, Efficacy and Safety of Treatment With Human-cl-rhFVIII in Previously Untreated Patients With Severe Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Severe Hemophilia A
Keywords: Previously untreated patients
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2012-10-23
Enrollment: 110
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.


Eligibility criteria: Inclusion Criteria:

* Male patients
* Severe Hemophilia A (FVIII:C \<1%)
* No previous treatment with FVIII concentrates or other blood products containing FVIII

Exclusion Criteria:

* Diagnosis with a coagulation disorder other than Hemophilia A
* Severe liver or kidney disease
* Concomitant treatment with any systemic immunosuppressive drug

Primary outcomes:
measure: Immunogenicity of Human-cl rhFVIII: Incidence of Inhibitors
timeFrame: maximum 5 years (100 exposure days)
Secondary outcomes:
measure: Frequency of Spontaneous Break-through Bleeds
timeFrame: Maximum 5 years (100 exposure days)
measure: Efficacy of Human-cl rhFVIII for the Treatment of Bleeds
timeFrame: Maximum 5 years (100 exposure days)
measure: Efficacy of Human-cl rhFVIII for Surgical Prophylaxis
timeFrame: Maximum 5 years (100 exposure days)
Other outcomes:
measure: The Occurrence of Any Adverse Event (AE)
timeFrame: 5 years
Locations:
city: Sacramento
country: United States
facility: UC Davis
state: California
city: Saint Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Boston
country: United States
facility: Harvard Children's Hospital Boston
state: Massachusetts
city: Minsk
country: Belarus
facility: Republican Scientific Practical Center for Pediatric Oncology and Hematology
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Vancouver
country: Canada
facility: BC Children's Hospital
state: British Columbia
city: Hamilton
country: Canada
facility: Mc Master Children's Hospital
state: Ontario
city: Toronto
country: Canada
facility: Hospital for Sick Children
state: Ontario
city: Montreal
country: Canada
facility: Hopital Ste-Justine
state: Quebec
city: Caen
country: France
facility: L'hôpital Côte de Nacre - CHU de Caen
city: Le Kremlin-Bicêtre
country: France
facility: Centre de traitement de l'hemophilie, Hôpital Bicêtre
city: Marseille
country: France
facility: Hopital de la Timone
city: Nantes
country: France
facility: Hôtel-Dieu de Nantes, Centre Regional de Traitement de l'hemophilie
city: Paris
country: France
facility: Hôpital Necker
city: Rennes
country: France
facility: CHU de Rennes - Hôpital Pontchaillou
city: Tours
country: France
facility: Hopital Trousseau - CHU Tours
city: Tbilisi
country: Georgia
facility: Institute of Haematology and Transfusiology
city: Bonn
country: Germany
facility: Institut für Experimentelle Hämatologie und Transfusionsmedizin (IHT)
city: Frankfurt
country: Germany
facility: University Hospital Frankfurt/M
city: Mainz
country: Germany
facility: Universitätsmedizin der Johannes-Gutenberg-Universität Mainz
city: Manipala
country: India
facility: Kasturba Medical College, Dr. TMA Pai Hospital
state: Karnataka
city: Pune
country: India
facility: Sahyadri Speciality Hospital, Haematology & BMT Unit
city: Vellore
country: India
facility: Christian Medical College & Hospital, Dept of Haematology
city: Perugia
country: Italy
facility: Univ. Di Perugia
city: Torino
country: Italy
facility: Centro di Referimento per le Malattie Emorragiche e Trombotiche
city: Chisinau
country: Moldova, Republic of
facility: Scientific Research Institute of Mother and Child Health Care
city: Rabat
country: Morocco
facility: Centre Hospitalier Ibn Sina
city: Warsaw
country: Poland
facility: University Medical School Warsaw
city: Porto
country: Portugal
facility: HSJ - Hospital de São João, EPE
city: Moscow
country: Russian Federation
facility: Morozovsky Children's Hospital
city: Ljubljana
country: Slovenia
facility: Haemophilia Centre, University Clinical Centre
city: Barcelona
country: Spain
facility: Unitat d'hemofilia, Hospital Universitari Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Kiev
country: Ukraine
facility: National Children's Specialized Hospital "OHMATDET"
city: Lviv
country: Ukraine
facility: Institute of Blood Pathology and Transfusion Medicine
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
Study officials:
affiliation: Octapharma
name: Sigurd Knaub
role: STUDY_DIRECTOR
References: