Clinical trial NCT03528551: A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A
Official title: Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Prophylaxis and Treatment of Bleeds in Previously N8-GP Treated 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: 2018-05-18
Enrollment: 160
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will look at how a known study medicine N8-GP works in previously N8-GP treated people with haemophilia A. The aim is to look at how N8-GP works during regular use. Participants will get N8-GP. N8-GP has been tested in more than 200 people with haemophilia A for several years. Participants will get an injection of N8-GP into a blood vessel, one, two or three times weekly. Participants will get more doses if they bleed or if they will need a surgery. The study will last for about 2 years. Participants will have at least 9 visits with the study doctor. If participants agree to be in this study, they will get their first injection (in this study) at the first visit. Participants will also get an injection at visit 3, 5 and 7. Participants will be trained to give all other injections themselves. Participants must not use any clotting factors other than N8-GP or any anticoagulants (blood thinners) during the study.


Eligibility criteria: Inclusion Criteria:

* Male patients of all ages with the diagnosis of severe congenital haemophilia A (coagulation Factor VIII \[FVIII\] activity less than 1%) based on medical records
* On-going participation in NN7088-3859 (pathfinder2), or NN7088-3885 (pathfinder5) at the time of transfer

Exclusion Criteria:

* Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
* Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise patient's safety or compliance with the protocol - Current participation in any clinical trial (except NN7088-3859 (pathfinder2) or NN7088-3885 (pathfinder5)) of an approved or non-approved investigational medicinal product

Primary outcomes:
measure: Number of Adverse Events Reported
timeFrame: Week 0 to week 108
Secondary outcomes:
measure: Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (BU)
timeFrame: Week 0 to week 104
measure: Number of Bleeding Episodes on Prophylaxis
timeFrame: Week 0 to week 104
measure: Number of Spontaneous Bleeding Episodes on Prophylaxis
timeFrame: Week 0 to week 104
measure: Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None
timeFrame: Week 0 to week 104
measure: Mean Number of N8-GP Injections Required Per Bleeding Episode
timeFrame: Week 0 to week 104
measure: Pre-dose FVIII Activity Levels on N8-GP Prophylaxis
timeFrame: Week 0 to week 104
measure: Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score)
timeFrame: Week 0, Week 104
measure: Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None
timeFrame: Week 0 to week 104
measure: Consumption of N8-GP Per Bleed
timeFrame: Week 0 to week 104
measure: Consumption of N8-GP During Prophylaxis Treatment
timeFrame: Week 0 to week 104
measure: Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score)
timeFrame: Week 0, Week 104
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: 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: Baltimore
country: United States
facility: Novo Nordisk Investigational Site
state: Maryland
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: Minneapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Minnesota
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: Portland
country: United States
facility: Novo Nordisk Investigational Site
state: Oregon
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: Houston
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: Norfolk
country: United States
facility: Novo Nordisk Investigational Site
state: Virginia
city: Camperdown
country: Australia
facility: Novo Nordisk Investigational Site
state: New South Wales
city: Parkville
country: Australia
facility: Novo Nordisk Investigational Site
state: Victoria
city: Campinas
country: Brazil
facility: Novo Nordisk Investigational Site
state: Sao Paulo
city: Toronto
country: Canada
facility: Novo Nordisk Investigational Site
state: Ontario
city: Zagreb
country: Croatia
facility: Novo Nordisk Investigational Site
city: Århus N
country: Denmark
facility: Novo Nordisk Investigational Site
city: Bron Cedex
country: France
facility: Novo Nordisk Investigational Site
city: Le Kremlin Bicetre
country: France
facility: Novo Nordisk Investigational Site
city: Nantes Cedex 1
country: France
facility: Novo Nordisk Investigational Site
city: Berlin
country: Germany
facility: Novo Nordisk Investigational Site
city: Frankfurt/M.
country: Germany
facility: Novo Nordisk Investigational Site
city: Homburg
country: Germany
facility: Novo Nordisk Investigational Site
city: Athens
country: Greece
facility: Novo Nordisk Investigational Site
city: Budapest
country: Hungary
facility: Novo Nordisk Investigational Site
city: Debrecen
country: Hungary
facility: Novo Nordisk Investigational Site
city: Tel-Hashomer
country: Israel
facility: Novo Nordisk Investigational Site
city: Milano
country: Italy
facility: Novo Nordisk Investigational Site
city: Vicenza
country: Italy
facility: Novo Nordisk Investigational Site
city: Kitakyusyu-shi, Fukuoka
country: Japan
facility: Novo Nordisk Investigational Site
city: Nara
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Daejeon
country: Korea, Republic of
facility: Novo Nordisk Investigational Site
city: Vilnius
country: Lithuania
facility: Novo Nordisk Investigational Site
city: Selangor Darul Ehsan
country: Malaysia
facility: Novo Nordisk Investigational Site
city: Groningen
country: Netherlands
facility: Novo Nordisk Investigational Site
city: Rotterdam
country: Netherlands
facility: Novo Nordisk Investigational Site
city: Oslo
country: Norway
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: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Málaga
country: Spain
facility: Novo Nordisk Investigational Site
city: Bellinzona
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Lausanne
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Zürich
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Zürich
country: Switzerland
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
city: Samsun
country: Turkey
facility: Novo Nordisk Investigational Site
city: Lviv
country: Ukraine
facility: Novo Nordisk Investigational Site
city: Basingstoke
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Cardiff
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: London
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
city: Oxford
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Sheffield
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor and Disclosure 2834
role: STUDY_DIRECTOR
References: