Clinical trial NCT01493778: Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
Official title: Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients With Haemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2011-12-16
Enrollment: 60
Sex: MALE
Minimum age (years): 0
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia A.


Eligibility criteria: Inclusion Criteria:

* Age below 6 years
* Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)
* Male patients diagnosed with congenital severe haemophilia A (FVIII level equal to or below 1%)
* No prior use of purified clotting factor products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma, is accepted) including commercially available NovoEight® /Novoeight®

Exclusion Criteria:

* Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
* Previous participation in this trial defined as withdrawal after administration of trial product
* Congenital or acquired coagulation disorders other than haemophilia A
* Any history of Factor VIII inhibitor
* Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids)

Primary outcomes:
measure: Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial
timeFrame: From Visit 2 (21 days after screening) to Visit 5 (50-55 exposure day)
Secondary outcomes:
measure: Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Annualised Bleeding Rate (ABR)
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Number of Turoctocog Alfa (N8) Injections Required Per Bleed
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Month
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Year
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Consumption of Turoctocog Alfa (N8) (IU/kg/Bleed) Per Bleed
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Consumption of Turoctocog Alfa (N8) (IU/kg/Months) for Bleed Prevention
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs) Reported During the Trial Period
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Incidence Rate of Clinically Relevant Inhibitors Defined as an Inhibitor Titre (≥ 0.6 BU/mL) Combined With a Decreased Recovery (<66% of Expected Level)
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Incidence Rate of High-titre Inhibitors Defined as Inhibitor Titre ≥ 5 BU (Bethesda Units)/mL)
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Change in Total Scores for Parent Reported Treatment Satisfaction
timeFrame: Visit 3 (10th-15th ED); Visit 5 (50th-55th ED); End of trial (within 8 weeks of their last scheduled visit in the extension phase)
measure: Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Month Per Patient)
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
measure: Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Year Per Patient)
timeFrame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novo Nordisk Investigational Site
state: Arizona
city: Long Beach
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Sacramento
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Washington
country: United States
facility: Novo Nordisk Investigational Site
state: District of Columbia
city: Gainesville
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Orlando
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Tampa
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Tampa
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Augusta
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Macon
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Savannah
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Oak Lawn
country: United States
facility: Novo Nordisk Investigational Site
state: Illinois
city: Detroit
country: United States
facility: Novo Nordisk Investigational Site
state: Michigan
city: Las Vegas
country: United States
facility: Novo Nordisk Investigational Site
state: Nevada
city: New Brunswick
country: United States
facility: Novo Nordisk Investigational Site
state: New Jersey
city: Brooklyn
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Brooklyn
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Charlotte
country: United States
facility: Novo Nordisk Investigational Site
state: North Carolina
city: Cincinnati
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Dayton
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Oklahoma City
country: United States
facility: Novo Nordisk Investigational Site
state: Oklahoma
city: Portland
country: United States
facility: Novo Nordisk Investigational Site
state: Oregon
city: Charleston
country: United States
facility: Novo Nordisk Investigational Site
state: South Carolina
city: Salt Lake City
country: United States
facility: Novo Nordisk Investigational Site
state: Utah
city: Charlottesville
country: United States
facility: Novo Nordisk Investigational Site
state: Virginia
city: Algiers
country: Algeria
facility: Novo Nordisk Investigational Site
city: Annaba
country: Algeria
facility: Novo Nordisk Investigational Site
city: Graz
country: Austria
facility: Novo Nordisk Investigational Site
city: Innsbruck
country: Austria
facility: Novo Nordisk Investigational Site
city: Klagenfurt
country: Austria
facility: Novo Nordisk Investigational Site
city: Linz
country: Austria
facility: Novo Nordisk Investigational Site
city: Salzburg
country: Austria
facility: Novo Nordisk Investigational Site
city: St. Poelten
country: Austria
facility: Novo Nordisk Investigational Site
city: Wien
country: Austria
facility: Novo Nordisk Investigational Site
city: Curitiba
country: Brazil
facility: Novo Nordisk Investigational Site
state: Parana
city: Campinas
country: Brazil
facility: Novo Nordisk Investigational Site
state: Sao Paulo
city: Beijing
country: China
facility: Novo Nordisk Investigational Site
state: Beijing
city: Chonqqing
country: China
facility: Novo Nordisk Investigational Site
state: Chongqing
city: Guangzhou
country: China
facility: Novo Nordisk Investigational Site
state: Guangdong
city: Tianjing
country: China
facility: Novo Nordisk Investigational Site
state: Tianjin
city: Hangzhou
country: China
facility: Novo Nordisk Investigational Site
state: Zhejiang
city: Beijing
country: China
facility: Novo Nordisk Investigational Site
city: København Ø
country: Denmark
facility: Novo Nordisk Investigational Site
city: Athens
country: Greece
facility: Novo Nordisk Investigational Site
city: Thessaloniki
country: Greece
facility: Novo Nordisk Investigational Site
city: Shatin, New Territories
country: Hong Kong
facility: Novo Nordisk Investigational Site
city: Budapest
country: Hungary
facility: Novo Nordisk Investigational Site
city: Debrecen
country: Hungary
facility: Novo Nordisk Investigational Site
city: Aichi
country: Japan
facility: Novo Nordisk Investigational Site
city: Hyogo
country: Japan
facility: Novo Nordisk Investigational Site
city: Shizuoka
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Vilnius
country: Lithuania
facility: Novo Nordisk Investigational Site
city: Bydgoszcz
country: Poland
facility: Novo Nordisk Investigational Site
city: Lublin
country: Poland
facility: Novo Nordisk Investigational Site
city: Porto
country: Portugal
facility: Novo Nordisk Investigational Site
city: San Juan
country: Puerto Rico
facility: Novo Nordisk Investigational Site
city: Krasnodar
country: Russian Federation
facility: Novo Nordisk Investigational Site
city: Moscow
country: Russian Federation
facility: Novo Nordisk Investigational Site
city: Saint-Petersburg
country: Russian Federation
facility: Novo Nordisk Investigational Site
city: Belgrade
country: Serbia
facility: Novo Nordisk Investigational Site
city: Barcelona
country: Spain
facility: Novo Nordisk Investigational Site
city: El Palmar
country: Spain
facility: Novo Nordisk Investigational Site
city: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Adana
country: Turkey
facility: Novo Nordisk Investigational Site
city: Antalya
country: Turkey
facility: Novo Nordisk Investigational Site
city: Bornova-IZMIR
country: Turkey
facility: Novo Nordisk Investigational Site
city: Izmit
country: Turkey
facility: Novo Nordisk Investigational Site
city: Samsun
country: Turkey
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: