Hemophilia A Research Program
Study Details
- Hemophilia A
- Hemophilia A, Severe
- Unmapped
- Hemophilia A With Inhibitor(s)
- Hemophilia
- 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
Protocol Summary
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.