A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With H… (NCT06684314) | Clinical Trial Compass
RecruitingNot Applicable
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
Taiwan100 participantsStarted 2024-11-11
Plain-language summary
This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants. The data related to efanesoctocog alfa effectiveness, safety and usage will be recorded prospectively during routine visits for up to 5 years following enrollment initiation and the retrospective data will be collected at least 12 months and up to 24 months prior to efanesoctocog alfa initiation. Joint imaging data will be collected in centers performing Joint U/S and/or MRI (≥6 years old). At least 12 months of retrospective data will also be collected from medical records, as available. Prospectively collected data will be recorded at routine clinical visits during a five-year follow-up period. The end of study is defined as the last participant's last visit. No intervention will be administered, and no study related visits are required.
Who can participate
Sex
ALL
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:
* Participants with all ages and diagnosis of moderate-severe hemophilia A without current and/or at least three years of un-detectable inhibitor (\<0.6 BU)
* Participants with moderate to severe hemophilia A as defined by FVIII level ≤ 5%
* Participants starting efanesoctocog alfa prophylaxis treatment as per standard of care no more than three months prior to the enrollment date
* Participants aged 6 years and older are able to undergo MRI examinations (sedation given, if necessary, and per investigator discretion)
* Participants are able to undergo joint examinations
* Physician's decision to treat the participant with efanesoctocog alfa is made prior to and independently of participation in the study
* Signed and dated informed consent provided by the participant, or by the participant's legally acceptable representative for participants under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric participants according to local regulations
Exclusion Criteria:
* Participants with coagulation disorders other than hemophilia A
* Participants diagnosed with other known bleeding disorder
* Participants currently receive factor therapy and have signs of decreased response to FVIII therapy
* Participants with a baseline Radiological Pettersson score (PS) of greater than 6 for each individual ankle
* Enrollment in another concurrent clinical interventional study, or intake of an Investigational Medicinal P…
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
Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints
Timeframe: At 1, 2, 3, 4 and 5 years
2
Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds
Timeframe: At 1, 2, 3, 4 and 5 years
3
Number of target joint development, resolution and/or recurrence
Timeframe: At 1, 2 ,3, 4 and 5 years
Trial details
NCT IDNCT06684314
SponsorSanofi
Sponsor typeINDUSTRY
Study typeOBSERVATIONAL
Primary completion2031-08-28
Contact for this trial
Trial Transparency email recommended (Toll free for US & Canada)