Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

Brief summary (registry, in English)

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.

What you commit to

• Study type: Observational
• Participants enrolled: 71
• From 1 Aug 2024 to the primary completion date, 18 Jul 2025
• 10 sites in 1 country
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: - to 36 months · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (10)

Sites in registry order. Pick your city on the disease page to sort them by distance.