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RECRUITINGOBSERVATIONAL

Hemophilia A Research Program

Hemophilia A Research Program (HARP): An Observational Intergenerational Cohort Study of Hemophilia A and Factor VIII Immunogenicity

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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 May Be Eligible (Plain English)

Maternal Who May Qualify: 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 willing to sign a consent form - 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 / Who May Qualify: 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 blood relatives (e.g., father, sibling) of the child - Second-degree blood relatives (e.g., aunt, uncle, grandparent, half-sibling) of the child - Any more distant male or female blood relative whose data or samples may be informative for the planned genetic studies of hemophilia and inhibitors Exclusion/Discontinuation Criteria: Maternal: For the pregnant person, exclusion or discontinuation criteria are as follows: - Genetic testing is negative for a severe hemophilia A genotype ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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 blood relatives (e.g., father, sibling) of the child * Second-degree blood relatives (e.g., aunt, uncle, grandparent, half-sibling) of the child * Any more distant male or female blood relative whose data or samples may be informative for the planned genetic studies of hemophilia and inhibitors Exclusion/Discontinuation Criteria: Maternal: For the pregnant person, exclusion or discontinuation criteria are as follows: * Genetic testing is negative for a severe hemophilia A genotype * Prenatal clinical diagnostic testing that indicates there is no fetus affected with severe hemophilia A * Presence of another clinically significant bleeding disorder * Participation in another study for which any blood collection total would exceed safety limits defined in this study * Will deliver outside the United States or plans for regular pediatric care for the child to be delivered outside the United States * Is a prisoner * Any other reason that, in the opinion of the investigator, would render the individual unsuitable for participation in the study * Inability for study team to obtain translated study documents in time for participation if participant is not fluent in English Pediatric: For the child, discontinuation criteria are as follows: * Infant does not have severe hemophilia A defined by a baseline FVIII:C \< 0.01 IU/mL (or FVIII:C \< 1%) or does not have a genotype predicted to cause severe hemophilia A * Mother or child did not have minimal required study samples or data collected before birth, around the time of delivery, or in the neonatal period * Child has another clinically significant bleeding disorder * Child has a clinically severe immune disorder * Participation in another study for which any blood collection total would exceed safety limits defined in this study * Any other reason that, in the opinion of the investigator, would render the individual unsuitable for participation in the study

Locations (1)

University of Washington
Seattle, Washington, United States