Clinical trial NCT01392547: Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors
Official title: Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients With Congenital Haemophilia and Inhibitors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Haemophilia A With Inhibitors
Haemophilia B With Inhibitors
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2011-07-12
Enrollment: 72
Sex: MALE
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: This trial is conducted globally. The purpose of this trial is to confirm the efficacy and safety of NNC 0078-0000-0007 in patients with congenital haemophilia and inhibitors.

Detailed description: Scheduled dose visit in a non-bleeding state. Single dose of NNC 0078-0000-0007 (vatreptocog alfa (activated)) every 3 months.

Eligibility criteria: Inclusion Criteria:

* Male patient with clinical diagnosis of congenital haemophilia A or B and inhibitors to coagulation factors VIII or IX
* Minimum of five bleeds requiring haemostatic drug treatment within the previous 12 months at trial entry

Exclusion Criteria:

* Previous participation in this trial defined as withdrawal after administration of trial product
* Patient has received an investigational medicinal product within 30 days prior to this trial
* Congenital or acquired coagulation disorders other than haemophilia A or B
* Any clinical signs or known history of arterial thrombotic events or of deep venous thrombosis or pulmonary embolism (as defined by available medical records)
* Platelet count of less than 50,000 platelets/mcL (at the screening visit)
* ALAT (alanine-transaminase) of more than 3 times the upper normal limit (according to laboratory reference ranges)
* Factor VIII/IX Immune Tolerance Induction regimen planned to occur during the trial
* Ongoing bleeding prophylaxis regimens or planned bleeding prophylaxis to occur during the trial
* HIV (Human Immunodeficiency Virus) positive with current CD4+ count of less than 200/mcL (defined by medical records)

Primary outcomes:
measure: Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given
timeFrame: Within 12 hours of first trial product administration
Secondary outcomes:
measure: Effective and Sustained Bleeding Control
timeFrame: Up to 48 hours after first trial product administration
measure: Number of Doses of Trial Product Given for Each Acute Bleed
timeFrame: Up to 6 hours after first trial product administration
measure: Number of Adverse Events
timeFrame: Adverse events were captured from the time of consent to 1 month (+14 days) after last administration of trial product.
measure: Immunogenicity (Inhibitor Development)
timeFrame: Adverse events were captured from the time of consent to the end of trial visit 1 month (+14 days) after last administration of trial product.
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Arizona
city: Los Angeles
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: California
city: Los Angeles
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: California
city: Orange
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: California
city: Aurora
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Colorado
city: Tampa
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Florida
city: Atlanta
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Georgia
city: Augusta
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Georgia
city: Iowa City
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Iowa
city: Boston
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Massachusetts
city: Detroit
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Michigan
city: Brooklyn
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: New York
city: Portland
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Oregon
city: Richmond
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Virginia
city: Linz
country: Austria
city: Campinas
country: Brazil
state: Sao Paulo
city: Zagreb
country: Croatia
city: Athens
country: Greece
city: Budapest
country: Hungary
city: Milano
country: Italy
city: Shinjuku-ku, Tokyo
country: Japan
city: Kuala Lumpur
country: Malaysia
city: Warszawa
country: Poland
city: San Juan
country: Puerto Rico
facility: Novo Nordisk Clinical Trial Call Center
city: Timisoara
country: Romania
state: Timis
city: Saint-Petersburg
country: Russian Federation
city: Novi Sad
country: Serbia
city: Parktown, Johannesburg
country: South Africa
state: Gauteng
city: Changhua
country: Taiwan
city: Bangkok
country: Thailand
city: Bornova-IZMIR
country: Turkey
city: Oxford
country: United Kingdom
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: