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

NCT ID: NCT05248594

Last Updated: 2025-09-08

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 71 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-08-01
• Study Completion Date: 2025-07-18

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.

Conditions

Hemophilia A

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Interventions

HEMLIBRA

Emicizumab (Hemlibra, also known as ACE910 and RO5534262) is a humanized monoclonal modified immunoglobulin G4 (IgG4) antibody with a bispecific antibody structure produced by recombinant DNA technology in Chinese hamster ovary (CHO) cells.

Intervention Type: DRUG

Other Intervention Names

Emicizumab-kxwh

Eligibility Criteria

Inclusion Criteria

• 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.

• Maximum Eligible Age: 36 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genentech, Inc.

INDUSTRY

Sponsor Role: collaborator

Regional Comprehensive Hemophilia Treatment Center (Mount Sinai)

UNKNOWN

Sponsor Role: collaborator

Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center

UNKNOWN

Sponsor Role: collaborator

Rutgers Robert Wood Johnson Medical School

OTHER

Sponsor Role: collaborator

Montefiore Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jennifer Davila, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital at Montefiore

Locations

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

Countries

United States

Other Identifiers

ML43506

Identifier Type: OTHER

Identifier Source: secondary_id

2021-13264

Identifier Type: -

Identifier Source: org_study_id