A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors (NCT03417245) | Clinical Trial Compass
CompletedPhase 3
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
United States, Australia, China120 participantsStarted 2018-03-01
Plain-language summary
Primary Objective:
-To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes.
Secondary Objectives:
* To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by:
* The frequency of spontaneous bleeding episodes.
* The frequency of joint bleeding episodes.
* Health-related quality of life (HRQOL) in participants \>=17 years of age.
* To determine the frequency of bleeding episodes during the onset period.
* To determine the safety and tolerability of fitusiran.
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:
* Males, \>=12 years of age.
* Severe hemophilia A or B without inhibitors.
* Severity confirmed by a central laboratory where FVIII level was less than (\<) 1 percent (%) or Factor IX (FIX) level was less than or equal to (\<=) 2% at Screening.
* On-demand use of factor concentrate to manage bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion:
* Nijmegen modified Bethesda assay inhibitor titer of \<0.6 Bethesda units per milliliter (BU/mL) at Screening.
* No use of Bypassing agents to treat bleeding episodes for at least the last 6 months prior to Screening.
* No history of immune tolerance induction therapy within the last 3 years prior to Screening.
* A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening.
* Willing and complied with the study requirements and to provide written informed consent and assent.
Exclusion Criteria:
* Known co-existing bleeding disorders other than hemophilia A or B, i.e., Von Willebrand's disease, additional factor deficiencies, or platelet disorders.
* Antithrombin (AT) activity \<60% at Screening.
* Co-existing thrombophilic disorder.
* Clinically significant liver disease.
* Active hepatitis C virus infection.
* HIV positive with a cluster of differentiation-4 count of \<200 cells/microliter.
* History of arterial or venous thromboembolism.
* Inadequate renal function.
* History of mu…
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
Estimated Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period
Timeframe: From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest
2
Observed Annualized Bleeding Rate (ABR) for Treated Bleeds During the Efficacy Period
Timeframe: From Day 29 up to Day 246 or up to the last day of bleeding follow up (any day up to Day 246), whichever was the earliest