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

We have developed 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. We are also collecting data on any pre-, peri and post surgical practices while on emicizumab. More importantly, we are asking if pediatricians are planning to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when they are planning to do so. We hope 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.

Study Overview

• Status: Not yet recruiting
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: HEMLIBRA

Detailed Description

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. Currently 11 out of the 23 regional centers have committed to collaborating on this study (Appendix A). 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.

• Study Type: Observational
• Enrollment (Estimated): 50

Contacts and Locations

• Study Contact: Name: Beatriz Casiano, BA; Phone Number: 718-741-2579; Email: bcasiano@montefiore.org
• Study Contact Backup: Name: Noelle Townsend, BS; Phone Number: 718-430-2377; Email: noelle.townsend@einsteinmed.edu

Study Locations

• United States
  • New York
    • Bronx, New York, United States, 10467
      • Children's Hospital at Montefiore
      • Principal Investigator: Jennifer Davila, MD
    • Manhasset, New York, United States, 11030
      • Northwell Health
    • Syracuse, New York, United States, 13210
      • SUNY Upstate

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second to 3 years (Child)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients 0 to 36 months of age, diagnosed with severe, moderate or mild Hemophlia A at the time of initiating treatment with Emicizumab.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of patients that are being treated with emicizumab	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.	Within a year.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patients who receive additional doses of factor concentrate while on emicizumab	Data collected will also include proportion of patients who receive additional doses of factor concentrate while on emicizumab for trauma or surgical interventions.	Within a year.

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data	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.	Within a year.

Collaborators and Investigators

• Sponsor: Montefiore Medical Center
• Collaborators: Genentech, Inc.
• Investigators: Principal Investigator: Jennifer Davila, MD, Children's Hospital at Montefiore

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2024
• Primary Completion (Estimated): April 1, 2025
• Study Completion (Estimated): April 1, 2025

Study Registration Dates

• First Submitted: February 10, 2022
• First Submitted That Met QC Criteria: February 10, 2022
• First Posted (Actual): February 21, 2022

Study Record Updates

• Last Update Posted (Estimated): February 9, 2024
• Last Update Submitted That Met QC Criteria: February 8, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: 2021-13264; ML43506 (Other Identifier: Sponsor)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No