Clinical trial NCT01561924: Prediction of the Efficacy of Activated Recombinant Human Factor VII in Adult Congenital Haemophilia A or B Patients With Inhibitors by Use of Thromboelastography
Official title: An Exploratory Non-interventional Study of Ex-vivo Spiking Followed by Thromboelastography as a Method for Predicting the Efficacy of Recombinant Activated Human FVII (rFVIIa, NovoSeven®) in Adult Congenital Haemophilia A or B Patients With Inhibitors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Congenital Bleeding Disorder
Haemophilia A With Inhibitors
Haemophilia B With Inhibitors
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2012-03-23
Enrollment: 17
Sex: MALE
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: This trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state.

Detailed description: The TEG parameters are: R time (Reaction Time), K time (K Time (arbitrary measurement)), a (a angle), MA (Maximum Amplitude) and LY30 (Lysis 30 min after MA) while the ROTEM parameters are: CT (Clotting Time), CFT (Clot Formation Time), a (a angle), MCF (Maximum Clot Firmness) and LI60 (Lysis index 60 min after CT)

Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of congenital haemophilia A or B with a FVIII:C clot activity or FIX:C clot activity, respectively, less than 5%
* Presently use of activated recombinant human factor VII (NovoSeven®) as haemostatic agent for preventive treatment or treatment of bleeding episodes
* A documented historical or present record for the presence of inhibitors to factor VIII or IX, respectively
* A documented history of 2 or more joint bleeding episodes during the preceding 12 months
* Subjects must be in a non-bleeding state (i.e. no clinical manifestations of active bleeds)

Exclusion Criteria:

* Subjects who have received any haemostatic treatment for a bleeding episode within the last 7 days prior to this trial
* Immune tolerance therapy within the last 30 days prior to this trial
* Clinically relevant coagulation disorders other than congenital haemophilia A or B
* Thrombocytopenia (platelet count below 60,000 platelets/mcl)
* Prophylactic haemostatic treatment within 3 days prior to this trial

Primary outcomes:
measure: TEG parameters obtained at baseline and with activated recombinant human factor VII
measure: ROTEM parameters obtained at baseline and with activated recombinant human factor VII
Secondary outcomes:
Other outcomes:
Locations:
city: Berkeley
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Los Angeles
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Iowa City
country: United States
facility: Novo Nordisk Investigational Site
state: Iowa
city: Philadelphia
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
city: Århus N
country: Denmark
facility: Novo Nordisk Investigational Site
city: Lyon
country: France
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: