Clinical trial NCT01731600: A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
Official title: A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe 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: 2012-11-22
Enrollment: 68
Sex: MALE
Minimum age (years): 0
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.


Eligibility criteria: Inclusion Criteria:

* Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
* Weight above or equal to 10 kg - Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years

Exclusion Criteria:

- Any history of FVIII inhibitors

Primary outcomes:
measure: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units
timeFrame: During the main phase of the trial (from 0-26 weeks of treatment)
Secondary outcomes:
measure: Frequency of Adverse Events Including Serious Adverse Events Reported During the Trial Period
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes and Assessed as: Excellent, Good, Moderate, or None
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Number of Bleeding Episodes During Prophylactic Treatment With N8-GP (Annualised Bleeding Rate)
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Consumption of N8-GP Per Bleeding Episode (Number of Injections)
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Consumption of N8-GP Per Bleeding Episode (U/kg)
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Consumption of N8-GP During Prophylaxis (Number of Injections)
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Consumption of N8-GP During Prophylaxis (U/kg Per Month)
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Consumption of N8-GP During Prophylaxis (U/kg Per Year)
timeFrame: Main phase: (from 0-26 weeks of treatment) and full trial: (0 weeks to last patient's completion of the trial, an average of 4.5 years)
measure: Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for Previous FVIII Product
timeFrame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
measure: Incremental Recovery (Defined as the Peak Level Recorded 60 Min After End of Injection) Evaluated for N8-GP
timeFrame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
measure: Area Under the Curve Evaluated for Previous FVIII Product
timeFrame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
measure: Area Under the Curve Evaluated for N8-GP
timeFrame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
measure: Terminal Half-life Evaluated for Previous FVIII Product
timeFrame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
measure: Terminal Half-life Evaluated for N8-GP
timeFrame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP
measure: Clearance Evaluated for Previous FVIII Product
timeFrame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product
measure: Clearance Evaluated for N8-GP
timeFrame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP.
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: Orange
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: Orlando
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Tampa
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Boise
country: United States
facility: Novo Nordisk Investigational Site
state: Idaho
city: Iowa City
country: United States
facility: Novo Nordisk Investigational Site
state: Iowa
city: New Orleans
country: United States
facility: Novo Nordisk Investigational Site
state: Louisiana
city: Boston
country: United States
facility: Novo Nordisk Investigational Site
state: Massachusetts
city: Minneapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Minnesota
city: Omaha
country: United States
facility: Novo Nordisk Investigational Site
state: Nebraska
city: New Hyde Park
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: Philadelphia
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
city: Philadelphia
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
city: Charleston
country: United States
facility: Novo Nordisk Investigational Site
state: South Carolina
city: Nashville
country: United States
facility: Novo Nordisk Investigational Site
state: Tennessee
city: Dallas
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Houston
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Charlottesville
country: United States
facility: Novo Nordisk Investigational Site
state: Virginia
city: Spokane
country: United States
facility: Novo Nordisk Investigational Site
state: Washington
city: Rio de Janeiro
country: Brazil
facility: Novo Nordisk Investigational Site
city: Toronto
country: Canada
facility: Novo Nordisk Investigational Site
state: Ontario
city: Bron Cedex
country: France
facility: Novo Nordisk Investigational Site
city: Lille
country: France
facility: Novo Nordisk Investigational Site
city: Paris
country: France
facility: Novo Nordisk Investigational Site
city: Athens
country: Greece
facility: Novo Nordisk Investigational Site
city: Thessaloniki
country: Greece
facility: Novo Nordisk Investigational Site
city: Tel-Hashomer
country: Israel
facility: Novo Nordisk Investigational Site
city: Vicenza
country: Italy
facility: Novo Nordisk Investigational Site
city: Kitakyusyu, Fukuoka
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: Kuala Lumpur
country: Malaysia
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: Bellinzona
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Luzern 16
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Zürich
country: Switzerland
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
city: Donetsk
country: Ukraine
facility: Novo Nordisk Investigational Site
city: Lviv
country: Ukraine
facility: Novo Nordisk Investigational Site
city: Leicester
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Oxford
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:
citation: Tosetto A, Neff A, Lentz SR, Santagostino E, Nemes L, Sathar J, Meijer K, Chowdary P, Shen C, Landorph A, Hampton K. Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials. Haemophilia. 2020 May;26(3):450-458. doi: 10.1111/hae.13980. Epub 2020 Apr 15.
citation: Saulyte Trakymiene S, Economou M, Kenet G, Landorph A, Shen C, Kearney S. Long-term safety and efficacy of N8-GP in previously treated pediatric patients with hemophilia A: Final results from pathfinder5. J Thromb Haemost. 2020 Sep;18 Suppl 1(Suppl 1):15-25. doi: 10.1111/jth.15036.