A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6) (explorer™6)

• Sponsor: Novo Nordisk A/S
• Information provided by (Responsible Party): Novo Nordisk A/S

Study Description

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 1½ years.

Condition or disease	Intervention/treatment
Haemophilia A; Haemophilia A With Inhibitors; Haemophilia B; Haemophilia B With Inhibitors	Other: No treatment given

Study Design

• Study Type: Observational
• Estimated Enrollment: 265 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• 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)
• Actual Study Start Date: December 18, 2018
• Estimated Primary Completion Date: July 20, 2020
• Estimated Study Completion Date: July 20, 2020

Groups and Cohorts

Group/Cohort	Intervention/treatment
Patients with haemophilia; Patients with haemophilia A or B and with or without inhibitors	Other: No treatment given; Participants are treated with their usual prescribed treatment, either given regularly as a prevention (prophylaxis) or when needed (on-demand).

Outcome Measures

Primary Outcome Measures :

1. The number of treated bleeding episodes [ Time Frame: From enrolment (week 0) and up to a maximum of 89 weeks ]
   Count of treated bleeding episodes

Secondary Outcome Measures :

1. The number of all bleeding episodes [ Time Frame: From enrolment (week 0) and up to a maximum of 89 weeks ]
   Count of bleeding episodes

Eligibility Criteria

• Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

Patients with haemophilia A or B and with or without inhibitors

Criteria

Inclusion Criteria:

• 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.
• Male, age greater than or equal to 12 years at the time of signing informed consent.
• Severe (FVIII [factor 8] activity less than 1%) congenital haemophilia A or severe (FIX [factor 9] activity less than 1%) congenital haemophilia B or congenital haemophilia A or B with inhibitors, based on medical records at the full discretion of the treating physician.
• Patients with congenital haemophilia A or B with inhibitors (CHwI) treated on-demand: greater than or equal to 6 treated (with bypassing agent) bleeding episodes within 24 weeks before screening (visit 1) and patients with severe congenital haemophilia A (HA)/ haemophilia B (HB) treated on-demand: greater than or equal to 5 treated (with factor product) bleeding episodes within 24 weeks before screening (visit 1).
• Patients with congenital haemophilia with inhibitors treated with FEIBA® prophylaxis: greater than or equal to 2 treated bleeding episodes within 24 weeks before screening (visit 1).

Exclusion Criteria:

• Known or suspected hypersensitivity to monoclonal antibodies.
• Previous participation in this study. Participation is defined as signed informed consent.
• 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.
• Previous treatment with concizumab. Previous treatment is defined as two or more doses administered.
• Planned FVIII/FIX Immune Tolerance Induction (ITI) regimens during the study.
• Current or planned treatment with emicizumab.
• Any known congenital or acquired coagulation disorder other than congenital haemophilia

Contacts and Locations

  Show 124 Study Locations

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

• Study Director: Clinical Reporting Anchor and Disclosure (1452); Novo Nordisk A/S

More Information

• Responsible Party: Novo Nordisk A/S
• ClinicalTrials.gov Identifier: NCT03741881 History of Changes
• Other Study ID Numbers: NN7415-4322; U1111-1182-3359 ( Other Identifier: World Health Organization (WHO) )
• First Posted: November 15, 2018
• Last Update Posted: August 22, 2019
• Last Verified: August 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
• URL: http://novonordisk-trials.com

• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hemophilia A; Hemophilia B; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Factor VIII; Coagulants