Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
Completed
Conditions studied: Hemophilia A
In brief
Hemophilia A (HA) is a rare congenital bleeding disorder characterized by coagulation factor VIII deficiency. In severe HA, defined as plasma FVIII clotting activity \< 1%, bleeding may frequently occur spontaneously, most commonly in joints, leading to painful hemophilic arthropathy and loss of joint function. Patients with moderate or mild hemophilia A, defined as FVIII clotting activity between 1-\<5% and 5-40% respectively, are less likely to have spontaneous bleeding however can have significant bleeding with trauma or surgery. Perioperative management by a hematologist who specializes in hemophilia is needed to ensure hemostasis during surgery. Hemophilia is an X-linked recessive disorder affecting 1 in 5000 to 10,000 males.
Key facts
- Study ID
- NCT05248594
- Run by
- Montefiore Medical Center
- People needed
- 71
- Starts
- 2024-08-01
- Expected to finish
- 2025-07-18
- Last updated by the study team
- 2025-09-08
Who can join
Age: any, up to 3. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Patients with acquired Hemophilia A
- Patients with Hemophilia A and another congenital or acquired bleeding disorder.
Where it is running
- Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center — Hartford, Connecticut, United States
- Yale Hemophilia Treatment Center — New Haven, Connecticut, United States
- Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital — Lebanon, New Hampshire, United States
- Newark Beth Israel — Newark, New Jersey, United States
- Western NY Blood Care - Research Foundation for SUNY — Buffalo, New York, United States
- Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell) — New York, New York, United States
- Mary M Gooley Hemophilia Center, Inc. — Rochester, New York, United States
- Northwell Health Hemostasis and Thrombosis Center — Staten Island, New York, United States
- SUNY Upstate Hemophilia Treatment Center - HTC 058 — Syracuse, New York, United States
- Children's Hospital at Montefiore — The Bronx, New York, United States
Full record on ClinicalTrials.gov
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