Hemophilia A Research Program (NCT07414511) | Clinical Trial Compass
RecruitingNot Applicable
Hemophilia A Research Program
United States500 participantsStarted 2024-07-31
Plain-language summary
This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood. Because the study follows mother-child pairs, the study includes both a maternal cohort and a pediatric cohort. Each cohort has a primary goal: for the mother with a severe hemophilia genotype, the overarching primary goal is to understand the risks for pregnancy-associated bleeding and postpartum hemorrhage (PPH); for the child, the overarching primary goal is to understand the risks, timing, and circumstances of development of anti-FVIII antibodies. From a longitudinal perspective, risks for both bleeding in the mother and anti-FVIII antibody development in the child are expected to be influenced over time by genetic and environmental factors that begin early in (or before) pregnancy. Enrollment of blood relatives is offered to improve power to better understand inherited contributions to bleeding and inhibitor development in the mother-baby pairs.
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.
Maternal Inclusion Criteria:
Pregnant individuals who meet the following criteria are eligible for enrollment as study participants:
* Currently pregnant and prior to 37 weeks gestation
* Known to have or at-risk of having a severe hemophilia A genotype
* Pregnant with at least one fetus at-risk of inheriting severe hemophilia A
* Ability to understand and willingness to provide informed consent
* 18 years of age or older
Before the 38th week of pregnancy, enrolled participants must meet all the following criteria to continue to remain in the study:
* The pregnant mother has a severe hemophilia A genotype.
* A fetus is determined to have a \>/= 25% risk of inheriting severe hemophilia A, or prenatal testing indicates a fetus is affected by severe hemophilia A.
* No other discontinuation criteria have been identified.
Pediatric Continuation / Inclusion Criteria:
Eligibility of the child to continue is assessed by age 8 weeks. Mother-child pairs in which a child meets the following criteria will remain in the study:
* Severe hemophilia A defined by a baseline FVIII:C \< 0.01 IU/mL (or FVIII:C \< 1%) or a genotype predicted to cause severe hemophilia A
* Born to a mother participating in the study
Thereafter, mothers and their children will continue in the study as long as no new discontinuation criteria occur.
Inclusion Criteria for Blood Relatives:
Blood relatives of the child may be offered participation if one of the following criteria are met:
* First-degree bloo…
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
Primary Endpoint(s)/Outcome(s)
Timeframe: For mother-child pairs, from enrollment until the child with severe hemophilia A is at least 2 years old*, or until a study discontinuation criteria is met. *This is the minimum duration, the mother-child pairs will be followed for as long as feasible.