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

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05248594
• Other study ID #: 2021-13264
• Secondary ID: ML43506
• Status: Not yet recruiting
• Start date: August 2024
• Est. completion date: August 2025

Study information

• Verified date: June 2024
• Source: Montefiore Medical Center
• Contact: Beatriz Casiano, BA
• Phone: 718-741-2579
• Email: bcasiano[@]montefiore.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 50
• Est. completion date: August 2025
• Est. primary completion date: August 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 36 Months

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

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.

Locations

Country	Name	City	State
United States	Children's Hospital at Montefiore	Bronx	New York
United States	Northwell Health	Manhasset	New York
United States	SUNY Upstate	Syracuse	New York

Sponsors (2)

Lead Sponsor	Collaborator
Montefiore Medical Center	Genentech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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.
Primary	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	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.