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

NCT05248594 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 71

Last updated 2025-09-08

No results posted yet for this study

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.

Conditions

Interventions

DRUG

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.

Sponsors & Collaborators

  • Genentech, Inc.

    collaborator INDUSTRY

  • Regional Comprehensive Hemophilia Treatment Center (Mount Sinai)

    collaborator UNKNOWN

  • Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center

    collaborator UNKNOWN

  • Rutgers Robert Wood Johnson Medical School

    collaborator OTHER

  • Montefiore Medical Center

    lead OTHER

Principal Investigators

  • Jennifer Davila, MD · Children's Hospital at Montefiore

Eligibility

Max Age
36 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-08-01
Primary Completion
2025-07-18
Completion
2025-07-18
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05248594 on ClinicalTrials.gov