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Completed

NCT Number: NCT05248594

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

Up to 36 month

Sex eligibility

All sexes

Study type

Observational

Primary location

Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center, Hartford, Connecticut, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

HEMLIBRA

Drug

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.

Other names: Emicizumab-kxwh

Primary outcomes

  1. The proportion of patients that are being treated with emicizumab

    Time frame: 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 outcomes

  1. Patients who receive additional doses of factor concentrate while on emicizumab

    Time frame: 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.

Other outcomes

  1. Patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data

    Time frame: Within a year.

    Additionally, we will characterize the 37+ patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data (indication for emicizumab ie. primary vs secondary prophylaxis.

Sponsors and collaborators

Lead sponsor

Montefiore Medical Center

Other

Collaborators

  • Genentech, Inc.
  • Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center
  • Regional Comprehensive Hemophilia Treatment Center (Mount Sinai)
  • Rutgers Robert Wood Johnson Medical School

Registry information

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Feb 21, 2022
Registry last updated
Sep 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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