COMPLETED

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

Conditions

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-08-01
Study Completion:2025-07-18
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:COMPLETED

Study ID

NCT05248594

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 36 Months
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Patients who have been prescribed Emicizumab
  2. * Patients who are 0-36 months of age at the time of starting treatment with Emicizumab
  3. * Diagnosis of congenital mild, moderate or severe hemophilia with or without an inhibitor
  1. * Patients with acquired Hemophilia A
  2. * Patients with Hemophilia A and another congenital or acquired bleeding disorder.

Contacts and Locations

Principal Investigator

Jennifer Davila, MD
PRINCIPAL_INVESTIGATOR
Children's Hospital at Montefiore

Study Locations (Sites)

Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center
Hartford, Connecticut, 06106
United States
Yale Hemophilia Treatment Center
New Haven, Connecticut, 06510
United States
Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital
Lebanon, New Hampshire, 03766
United States
Newark Beth Israel
Newark, New Jersey, 07112
United States
Western NY Blood Care - Research Foundation for SUNY
Buffalo, New York, 14202
United States
Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell)
New York, New York, 10065
United States
Mary M Gooley Hemophilia Center, Inc.
Rochester, New York, 14621
United States
Northwell Health Hemostasis and Thrombosis Center
Staten Island, New York, 11030
United States
SUNY Upstate Hemophilia Treatment Center - HTC 058
Syracuse, New York, 13210
United States
Children's Hospital at Montefiore
The Bronx, New York, 10467
United States

Collaborators and Investigators

Sponsor: Montefiore Medical Center

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-08-01
Study Completion Date2025-07-18

Study Record Updates

Study Start Date2024-08-01
Study Completion Date2025-07-18

Terms related to this study

Additional Relevant MeSH Terms

  • Hemophilia A