Comparison of Pulmonary Function, Exercise Capacity, and Physical Activity Level Between Children with Hemophilia and Healthy Controls
Study Details
- Hemophilia
- Unmapped
- Hemophilia group
- Inclusion criteria:
- Having been diagnosed with hemophilia and being clinically stable,
- Being between the ages of 8-18,
- Being on prophylaxis treatment (a routine treatment),
- Being 125 cm or taller,
- Being inhibitor negative at the time of enrollment,
- To be able to cooperate with the tests to be performed,
- Being willing to participate in the study.
- Exclusion criteria:
- Being inhibitor positive at the time of enrollment,
- Having a history of acute joint bleeding,
- Being unable to bear weight on the extremities,
- Having a history of acute intramuscular bleeding,
- Having undergone radionuclide synovectomy,
- Having a history of surgery on any of the lower extremity joints,
- Having a severe cardiovascular, orthopedic or neurological problem that may affect the assessments.
- Control group
- Inclusion criteria:
- Not having any known internal or chronic disease
- Being male between the ages of 8-18
- Having full range of motion in the upper extremity
- Having full range of motion in the lower extremity
- Not having any history of injury or surgery in the lower extremity
- Being 125 cm or taller
- Being willing to participate in the study
- Exclusion criteria:
- Having any problem that may affect the tests
- Not having full range of motion in the upper extremity
- Having a history of injury or surgery in any of the lower extremity joints
- Having a neurological deficit
- Having pain or movement limitation in the lower extremity
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.