Brief Title
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)
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.
Study Type
Observational
Primary Outcome
The number of treated bleeding episodes
Secondary Outcome
The number of all bleeding episodes
Condition
Haemophilia A
Intervention
No treatment given
Study Arms / Comparison Groups
Patients with haemophilia
Description: Patients with haemophilia A or B and with or without inhibitors
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
230
Start Date
December 18, 2018
Completion Date
March 17, 2021
Primary Completion Date
March 17, 2021
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.
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Reporting Anchor and Disclosure (1452), ,
Location Countries
Algeria
Location Countries
Algeria
Administrative Informations
NCT ID
NCT03741881
Organization ID
NN7415-4322
Secondary IDs
U1111-1182-3359
Responsible Party
Sponsor
Study Sponsor
Novo Nordisk A/S
Study Sponsor
Clinical Reporting Anchor and Disclosure (1452), Study Director, Novo Nordisk A/S
Verification Date
July 2020