Clinical trial NCT03741881: A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6)
Official title: A Prospective, Multi-national, Non-interventional Study in Haemophilia A and B Patients With or Without Inhibitors Treated According to Routine Clinical Treatment Practice (Explorer™6)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Haemophilia A
Haemophilia A With Inhibitors
Haemophilia B
Haemophilia B With Inhibitors
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2018-11-15
Enrollment: 231
Sex: MALE
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with haemophilia A or B and with or without inhibitors

Brief summary: This study will collect data on bleeds and data related to quality of life in people with severe congenital (a disease existing from birth) haemophilia A and B, with or without inhibitors. The aim for the study is to look at the number of bleeds when on usual treatment for haemophilia. Participants will be asked to keep an electronic diary to track the number of bleeds and the treatment of their bleeds. Participants will be asked to wear an activity tracker on their wrist to capture their level of activity every day for up to 12 weeks. While taking part in this study, participants will keep getting their usual treatment as given to them by their doctor. All study visits at the clinic are done in the same way as the participants are used to. In the time between the participants' visits to the clinic, the study staff at the clinic may call or email the participant. The study will last for about 2½ years.


Eligibility criteria: Inclusion Criteria:

1. Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine eligibility for the study.
2. Male, age equal to or above 12 years at the time of signing informed consent.
3. Patients with congenital haemophilia with inhibitors treated with FEIBA® prophylaxis: equal to or above 2 treated bleeding episodes within 24 weeks before screening (visit 1).

   (For Turkey only: Patients with congenital haemophilia with inhibitors treated with by-passing agents prophylaxis: equal to or above 2 treated bleeding episodes within 24 weeks before screening (visit 1)).
4. Severe (FVIII activity below 1%) congenital haemophilia A or severe/moderate (FIX activity equal to or below 2%) congenital haemophilia B, or congenital haemophilia A or B of any severity, with a presence or history of inhibitor (equal to or above 0.6 Bethesda Unit (BU)), based on medical records
5. Patients with CHwI treated on-demand: equal to or above 6 treated (with bypassing agent) bleeding episodes within 24 weeks (or equal to or above 12 during 52 weeks) before screening (visit 1) and patients with severe congenital HA/HB treated on-demand: equal to or above 5 treated (with factor product) bleeding episodes within 24 weeks (or equal to or above 10 during 52 weeks) before screening (visit 1).

Exclusion Criteria:

1. Known or suspected hypersensitivity to monoclonal antibodies.
2. Previous participation in this study. Participation is defined as signed informed consent.
3. Any disorder, except for conditions associated with congenital haemophilia, which in the physician's opinion might jeopardise patient's safety or compliance with the protocol.
4. Previous treatment with concizumab. Previous treatment is defined as two or more doses administered.
5. Planned FVIII/FIX Immune Tolerance Induction (ITI) regimens during the study.
6. Current or planned treatment with emicizumab.
7. Any known congenital or acquired coagulation disorder other than congenital haemophilia.
8. History of thromboembolic disease, current clinical signs of or treatment for thromboembolic disease, or at high risk of thromboembolic disease as judged by the investigator.
9. Presence or history of malignant neoplasm within 5 years prior to the day of screening.

Primary outcomes:
measure: The number of treated bleeding episodes
timeFrame: From enrolment (week 0) and up to a maximum of 115 weeks
Secondary outcomes:
measure: The number of all bleeding episodes
timeFrame: From enrolment (week 0) and up to a maximum of 115 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Sacramento
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Aurora
country: United States
facility: Novo Nordisk Investigational Site
state: Colorado
city: Washington
country: United States
facility: Novo Nordisk Investigational Site
state: District of Columbia
city: Atlanta
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
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: Chicago
country: United States
facility: Novo Nordisk Investigational Site
state: Illinois
city: Indianapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Indiana
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: Saint Louis
country: United States
facility: Novo Nordisk Investigational Site
state: Missouri
city: Las Vegas
country: United States
facility: Novo Nordisk Investigational Site
state: Nevada
city: Newark
country: United States
facility: Novo Nordisk Investigational Site
state: New Jersey
city: Albuquerque
country: United States
facility: Novo Nordisk Investigational Site
state: New Mexico
city: Charlotte
country: United States
facility: Novo Nordisk Investigational Site
state: North Carolina
city: Charlotte
country: United States
facility: Novo Nordisk Investigational Site
state: North Carolina
city: Cleveland
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Dayton
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Hershey
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
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: Houston
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
city: San Antonio
country: United States
facility: Novo Nordisk Investigational Site
state: Texas
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: Milwaukee
country: United States
facility: Novo Nordisk Investigational Site
state: Wisconsin
city: Algiers
country: Algeria
facility: Novo Nordisk Investigational Site
city: Constantine
country: Algeria
facility: Novo Nordisk Investigational Site
city: Melbourne
country: Australia
facility: Novo Nordisk Investigational Site
state: Victoria
city: Parkville
country: Australia
facility: Novo Nordisk Investigational Site
state: Victoria
city: Murdoch
country: Australia
facility: Novo Nordisk Investigational Site
state: Western Australia
city: Amstetten
country: Austria
facility: Novo Nordisk Investigational Site
city: Banja Luka
country: Bosnia and Herzegovina
facility: Novo Nordisk Investigational Site
city: Plovdiv
country: Bulgaria
facility: Novo Nordisk Investigational Site
city: Sofia
country: Bulgaria
facility: Novo Nordisk Investigational Site
city: Varna
country: Bulgaria
facility: Novo Nordisk Investigational Site
city: Calgary
country: Canada
facility: Novo Nordisk Investigational Site
state: Alberta
city: Saint John
country: Canada
facility: Novo Nordisk Investigational Site
state: New Brunswick
city: St. John's
country: Canada
facility: Novo Nordisk Investigational Site
state: Newfoundland and Labrador
city: Hamilton
country: Canada
facility: Novo Nordisk Investigational Site
state: Ontario
city: Hamilton
country: Canada
facility: Novo Nordisk Investigational Site
state: Ontario
city: Zagreb
country: Croatia
facility: Novo Nordisk Investigational Site
city: Zagreb
country: Croatia
facility: Novo Nordisk Investigational Site
city: Tallinn
country: Estonia
facility: Novo Nordisk Investigational Site
city: Helsinki
country: Finland
facility: Novo Nordisk Investigational Site
city: Brest
country: France
facility: Novo Nordisk Investigational Site
city: Bron Cedex
country: France
facility: Novo Nordisk Investigational Site
city: Caen
country: France
facility: Novo Nordisk Investigational Site
city: Kremlin-Bicêtre
country: France
facility: Novo Nordisk Investigational Site
city: Lille
country: France
facility: Novo Nordisk Investigational Site
city: Nantes Cedex 1
country: France
facility: Novo Nordisk Investigational Site
city: Paris
country: France
facility: Novo Nordisk Investigational Site
city: Rennes
country: France
facility: Novo Nordisk Investigational Site
city: Saint Etienne
country: France
facility: Novo Nordisk Investigational Site
city: Bonn
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: Thessaloniki
country: Greece
facility: Novo Nordisk Investigational Site
city: Budapest
country: Hungary
facility: Novo Nordisk Investigational Site
city: Bangalore
country: India
facility: Novo Nordisk Investigational Site
state: Karnataka
city: Pune
country: India
facility: Novo Nordisk Investigational Site
state: Maharashtra
city: New Dehli
country: India
facility: Novo Nordisk Investigational Site
state: New Delhi
city: Vellore
country: India
facility: Novo Nordisk Investigational Site
state: Tamil Nadu
city: Kolkatta
country: India
facility: Novo Nordisk Investigational Site
state: West Bengal
city: Tel-Hashomer
country: Israel
facility: Novo Nordisk Investigational Site
city: Castelfranco Veneto
country: Italy
facility: Novo Nordisk Investigational Site
city: Firenze
country: Italy
facility: Novo Nordisk Investigational Site
city: Roma
country: Italy
facility: Novo Nordisk Investigational Site
city: Udine
country: Italy
facility: Novo Nordisk Investigational Site
city: Verona
country: Italy
facility: Novo Nordisk Investigational Site
city: Aichi
country: Japan
facility: Novo Nordisk Investigational Site
city: Hiroshima
country: Japan
facility: Novo Nordisk Investigational Site
city: Hyogo
country: Japan
facility: Novo Nordisk Investigational Site
city: Kagoshima
country: Japan
facility: Novo Nordisk Investigational Site
city: Kanagawa
country: Japan
facility: Novo Nordisk Investigational Site
city: Kyoto
country: Japan
facility: Novo Nordisk Investigational Site
city: Mie
country: Japan
facility: Novo Nordisk Investigational Site
city: Nara
country: Japan
facility: Novo Nordisk Investigational Site
city: Niigata
country: Japan
facility: Novo Nordisk Investigational Site
city: Osaka
country: Japan
facility: Novo Nordisk Investigational Site
city: Saitama
country: Japan
facility: Novo Nordisk Investigational Site
city: Saitama
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: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Busan
country: Korea, Republic of
facility: Novo Nordisk Investigational Site
city: Daejeon
country: Korea, Republic of
facility: Novo Nordisk Investigational Site
city: Daejeon
country: Korea, Republic of
facility: Novo Nordisk Investigational Site
city: Jeju-do
country: Korea, Republic of
facility: Novo Nordisk Investigational Site
city: Seoul
country: Korea, Republic of
facility: Novo Nordisk Investigational Site
city: Seoul
country: Korea, Republic of
facility: Novo Nordisk Investigational Site
city: Vilnius
country: Lithuania
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: Monterrey
country: Mexico
facility: Novo Nordisk Investigational Site
state: Nuevo León
city: Groningen
country: Netherlands
facility: Novo Nordisk Investigational Site
city: Oslo
country: Norway
facility: Novo Nordisk Investigational Site
city: Krakow
country: Poland
facility: Novo Nordisk Investigational Site
city: Lublin
country: Poland
facility: Novo Nordisk Investigational Site
city: Lublin
country: Poland
facility: Novo Nordisk Investigational Site
city: Warszawa
country: Poland
facility: Novo Nordisk Investigational Site
city: Porto
country: Portugal
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: 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: Belgrade
country: Serbia
facility: Novo Nordisk Investigational Site
city: Kragujevac
country: Serbia
facility: Novo Nordisk Investigational Site
city: Novi Sad
country: Serbia
facility: Novo Nordisk Investigational Site
city: Bratislava
country: Slovakia
facility: Novo Nordisk Investigational Site
city: Parktown, Johannesburg
country: South Africa
facility: Novo Nordisk Investigational Site
state: Gauteng
city: Durban
country: South Africa
facility: Novo Nordisk Investigational Site
state: KwaZulu-Natal
city: Polokwane
country: South Africa
facility: Novo Nordisk Investigational Site
state: Limpopo
city: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Málaga
country: Spain
facility: Novo Nordisk Investigational Site
city: Oviedo
country: Spain
facility: Novo Nordisk Investigational Site
city: Sevilla
country: Spain
facility: Novo Nordisk Investigational Site
city: Valencia
country: Spain
facility: Novo Nordisk Investigational Site
city: Malmö
country: Sweden
facility: Novo Nordisk Investigational Site
city: Solna
country: Sweden
facility: Novo Nordisk Investigational Site
city: Zürich
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Adana
country: Turkey
facility: Novo Nordisk Investigational Site
city: Ankara
country: Turkey
facility: Novo Nordisk Investigational Site
city: Ankara
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: Capa-ISTANBUL
country: Turkey
facility: Novo Nordisk Investigational Site
city: Edirne
country: Turkey
facility: Novo Nordisk Investigational Site
city: Istanbul
country: Turkey
facility: Novo Nordisk Investigational Site
city: Samsun
country: Turkey
facility: Novo Nordisk Investigational Site
city: Kyiv
country: Ukraine
facility: Novo Nordisk Investigational Site
city: Lviv
country: Ukraine
facility: Novo Nordisk Investigational Site
city: Belfast
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Birmingham
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: Manchester
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Nottingham
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 (1452)
role: STUDY_DIRECTOR
References: