A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Trea… (NCT03741881) | Clinical Trial Compass
CompletedNot Applicable
A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6)
United States, Algeria, Australia231 participantsStarted 2018-12-18
Plain-language 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.
Who can participate
Age range
12 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 equal to or above 12 years at the time of signing informed consent.
. 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).
. 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
. 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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The number of treated bleeding episodes
Timeframe: From enrolment (week 0) and up to a maximum of 115 weeks
. 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.
. 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.