Clinical trial NCT01138501: Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
Official title: A Multi-Centre, Open-Label, Non-Controlled Trial on Safety and Efficacy of N8 in Previously Treated Paediatric 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: 2010-06-07
Enrollment: 65
Sex: MALE
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated paediatric subjects with haemophilia A.


Eligibility criteria: Inclusion Criteria:

* Male patients with severe (baseline FVIII less than or equal to 1%) haemophilia A
* Age below 12 years and weight at least 11 kg

Exclusion Criteria:

* Surgery planned to occur during trial participation (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures)
* Congenital or acquired coagulation disorders other than haemophilia A
* Any history of FVIII inhibitors (greater than or equal to 0.6 BU/mL)

Primary outcomes:
measure: The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU))
timeFrame: The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject.
Secondary outcomes:
measure: Frequency of Adverse Events (AEs)
timeFrame: The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novo Nordisk Investigational Site
state: Arizona
city: Orange
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Torrance
country: United States
facility: Novo Nordisk Investigational Site
state: California
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: Iowa City
country: United States
facility: Novo Nordisk Investigational Site
state: Iowa
city: Boston
country: United States
facility: Novo Nordisk Investigational Site
state: Massachusetts
city: Detroit
country: United States
facility: Novo Nordisk Investigational Site
state: Michigan
city: East Lansing
country: United States
facility: Novo Nordisk Investigational Site
state: Michigan
city: Kansas City
country: United States
facility: Novo Nordisk Investigational Site
state: Missouri
city: Omaha
country: United States
facility: Novo Nordisk Investigational Site
state: Nebraska
city: Brooklyn
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Valhalla
country: United States
facility: Novo Nordisk Investigational Site
state: New York
city: Dayton
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
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: Fort Worth
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Houston
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Curitiba
country: Brazil
facility: Novo Nordisk Investigational Site
state: Parana
city: Campinas
country: Brazil
facility: Novo Nordisk Investigational Site
state: Sao Paulo
city: São Paulo
country: Brazil
facility: Novo Nordisk Investigational Site
state: Sao Paulo
city: Rio de Janeiro
country: Brazil
facility: Novo Nordisk Investigational Site
city: Milano
country: Italy
facility: Novo Nordisk Investigational Site
city: Shizuoka-shi, Shizuoka
country: Japan
facility: Novo Nordisk Investigational Site
city: Vilnius
country: Lithuania
facility: Novo Nordisk Investigational Site
city: Skopje
country: Macedonia, The Former Yugoslav Republic of
facility: Novo Nordisk Investigational Site
city: Kuala Lumpur
country: Malaysia
facility: Novo Nordisk Investigational Site
city: Warszawa
country: Poland
facility: Novo Nordisk Investigational Site
city: Wroclaw
country: Poland
facility: Novo Nordisk Investigational Site
city: San Juan
country: Puerto Rico
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: Taipei
country: Taiwan
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
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Kulkarni R, Karim FA, Glamocanin S, Janic D, Vdovin V, Ozelo M, Rageliene L, Carboni E, Laguna P, Dobaczewski G, Seremetis S, Lindblom A, Santagostino E. Results from a large multinational clinical trial (guardian3) using prophylactic treatment with turoctocog alfa in paediatric patients with severe haemophilia A: safety, efficacy and pharmacokinetics. Haemophilia. 2013 Sep;19(5):698-705. doi: 10.1111/hae.12165. Epub 2013 May 8.
citation: Santagostino E, Lentz SR, Misgav M, Brand B, Chowdary P, Savic A, Kilinc Y, Amit Y, Amendola A, Solimeno LP, Saugstrup T, Matytsina I. Safety and efficacy of turoctocog alfa (NovoEight(R)) during surgery in patients with haemophilia A: results from the multinational guardian clinical trials. Haemophilia. 2015 Jan;21(1):34-40. doi: 10.1111/hae.12518. Epub 2014 Oct 2.
citation: Ozelo MC. Updates from guardian: a comprehensive registration programme. Eur J Haematol. 2015 Dec;95 Suppl 81:22-9. doi: 10.1111/ejh.12648.