Brief Title
Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
Official Title
Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
Brief 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.
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
Primary Outcome
The proportion of patients that are being treated with emicizumab
Secondary Outcome
Patients who receive additional doses of factor concentrate while on emicizumab
Condition
Hemophilia A
Intervention
HEMLIBRA
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
50
Start Date
April 2023
Completion Date
April 1, 2024
Primary Completion Date
April 1, 2024
Eligibility Criteria
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.
Gender
All
Ages
0 Months - 36 Months
Accepts Healthy Volunteers
No
Contacts
Jennifer Davila, MD, 718-741-2579, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT05248594
Organization ID
2021-13264
Secondary IDs
ML43506
Responsible Party
Sponsor
Study Sponsor
Montefiore Medical Center
Collaborators
Genentech, Inc.
Study Sponsor
Jennifer Davila, MD, Principal Investigator, Children's Hospital at Montefiore
Verification Date
March 2023