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

Completed

• Conditions: Hemophilia A

• Registration Number: NCT05248594
• Lead Sponsor: Montefiore Medical Center

Brief Summary

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.

Detailed Description

A questionnaire to elucidate the dosing, frequency and indication for the use of emicizumab in patients with Hemophilia A (mild, moderate or severe) ages 0-3 years has been developed. Data on any pre-, peri and post-surgical practices while on emicizumab is being collected. Pediatricians are being asked if there are plans to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when this is planned on being done. The hope is that this data will help inform understanding of current use of emicizumab in infants and young children as a form of primary prophylaxis, especially when venous access has historically been a limiting factor.

This will be a multi-institutional, retrospective review of pediatric patients ages 0 to 36 months of age who are currently receiving and/or have received emicizumab-kxwh as part of their treatment for hemophilia A with or without inhibitors. Participating sites are part of the New England Region (plus New Jersey and New York-Region II) of hemophilia treatment centers. Each institution will be contributing subjects who have been treated or who are currently being treated with emicizumab, from October 4th, 2018 up to the point of IRB approval. Additionally, each institution will determine the best way to identify eligible patients and keep track of patients enrolled in the study. The Children's Hospital at Montefiore will be the coordinating center.

Study Timeline

• Study First Submitted: 2022-02-10
• Study First Submitted QC: 2022-02-10
• Study First Posted: 2022-02-21
• Study Start: 2024-08-01
• Primary Completion: 2025-07-18
• Study Completion: 2025-07-18
• Status Verified: 2025-09
• Last Update Submitted: 2025-09-02
• Last Update Posted: 2025-09-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 71

Inclusion Criteria

Patients must meet the following criteria for study entry:

• Patients who have been prescribed Emicizumab
• Patients who are 0-36 months of age at the time of starting treatment with Emicizumab
• Diagnosis of congenital mild, moderate or severe hemophilia with or without an inhibitor

Exclusion Criteria

• Patients with acquired Hemophilia A
• Patients with Hemophilia A and another congenital or acquired bleeding disorder.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The proportion of patients that are being treated with emicizumab	Within a year.	The primary outcome will include the proportion of patients that are being treated with emicizumab for primary prophylaxis, as well as identifying breakthrough bleeding while on emicizumab.

Secondary Outcome Measures

Name	Time	Method
Patients who receive additional doses of factor concentrate while on emicizumab	Within a year.	Data collected will also include proportion of patients who receive additional doses of factor concentrate while on emicizumab for trauma or surgical interventions.

Trial Locations

Locations (10)

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