רדאר קליני AI | ||
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הניסוי הקליני NCT07414511 (HARP) עבור המופיליה A, המופיליה A חמורה, Factor VIII (FVIII), FVIII Deficiency, Carrier of Hemophilia A, Inhibitors, הריון, Maternal Blood Loss, סיבוכי הריון, הפרעת דימום, דימום לאחר לידה, Alloimmunization, X-Linked הוא מגייס. לכל הפרטים, עיינו בתצוגת הכרטיסים של רדאר ניסויים קליניים ובכלי הגילוי של AI. אפשר גם לשאול כל דבר כאן. | ||
Hemophilia A Research Program (HARP) 500 ילדים לאחר לידה
Hemophilia A is an inherited X-linked bleeding disorder caused by deficiency in coagulation factor VIII (FVIII). Severe hemophilia A is defined by a FVIII level < 0.01 IU/dL (or < 1%)....
הצג עודHemophilia A Research Program (HARP): An Observational Intergenerational Cohort Study of Hemophilia A and Factor VIII Immunogenicity
- HARP
- STUDY00020465
- 1UG3HL165064-01 (מענק/חוזה של NIH בארה"ב)
Inhibitor
Bleeding
Bleeding Disorder
Maternal Child Health
Hemophilia
Hemophilia A
Hemophilia A Carrier
Hemophilia A Symptomatic Carrier
Coagulation
Factor VIII
| קבוצת משתתפים/זרוע | התערבות/טיפול |
|---|---|
Maternal Cohort Inclusion Criteria (mother):
* Currently pregnant and prior to 37 weeks gestation
* Known or at-risk of having a severe hemophilia A genotype
* Pregnant with a fetus at-risk of inheriting severe hemophilia A
* Ability to understand and willingness to provide informed consent
* 18 years of age or older | לא ישים |
Pediatric Cohort Continuation Criteria (Child) * Severe hemophilia A defined by a baseline factor VIII activity (FVIII:C) \<0.01 international units (IU)/mL (or FVIII:C \< 1%) or a genotype predicted to result in severe hemophilia A
* Born to a pregnant mother participating in the study
* Absence of discontinuation criteria | לא ישים |
Blood Relatives of the Child (may be offered participation) * 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 | לא ישים |
| מדד תוצאה | תיאור המדידה | טווח זמן |
|---|---|---|
Primary Endpoint(s)/Outcome(s) | The study primary endpoints/outcomes for the maternal and pediatric cohorts are as follows:
• Maternal: Rates of primary PPH, defined as
* estimated or quantified blood loss \> 1,000 mL in the first 24 hours PP, or
* unplanned transfusion of blood products related to blood loss in the first 24 hours PP.
As a subset of primary PPH, severe primary PPH is defined as
* estimated or quantified blood loss \> 1,500 mL or requirement of \> 2 units packed red blood cells within 24 hours PP, or
* primary PPH with estimated or quantified blood loss \> 1,000 mL and evidence of maternal hemodynamic instability (tachycardia, hypotension) or end organ damage with no other etiology (oliguria, creatinine \> 0.8, etc.).
• Pediatric: Rate of development of humoral immune response to FVIII and proportion that progress to clinical inhibitors, defined as
* clinical FVIII inhibitor, or
* detection of an antibody specific to FVIII. | 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. |
| מדד תוצאה | תיאור המדידה | טווח זמן |
|---|---|---|
Secondary Endpoint(s)/Outcome(s) | Prioritized Secondary Maternal and Pediatric Endpoints/Outcomes
* Maternal: Rate of secondary PPH
* Maternal: Development of other clinically significant other bleeding
* Pediatric: Development of clinical FVIII inhibitor
* Pediatric: Development of IgG1 antibody to FVIII
* Pediatric: Development of high-affinity IgG3 or IgG4 antibody to FVIII
Additional descriptions of study outcomes and hypotheses to be tested are detailed in the study protocol. | 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. |
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
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