Status:

COMPLETED

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

Lead Sponsor:

Montefiore Medical Center

Collaborating Sponsors:

Genentech, Inc.

Regional Comprehensive Hemophilia Treatment Center (Mount Sinai)

Conditions:

Hemophilia A

Eligibility:

All Genders

Up to 36 years

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 occ...

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-...

Eligibility Criteria

Inclusion

  • 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

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

Key Trial Info

Start Date :

August 1 2024

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

July 18 2025

Estimated Enrollment :

71 Patients enrolled

Trial Details

Trial ID

NCT05248594

Start Date

August 1 2024

End Date

July 18 2025

Last Update

September 8 2025

Active Locations (10)

Enter a location and click search to find clinical trials sorted by distance.

Page 1 of 3 (10 locations)

1

Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center

Hartford, Connecticut, United States, 06106

2

Yale Hemophilia Treatment Center

New Haven, Connecticut, United States, 06510

3

Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital

Lebanon, New Hampshire, United States, 03766

4

Newark Beth Israel

Newark, New Jersey, United States, 07112