Use of Emicizumab in Young Children with Hemophilia A
Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
This study looks at how emicizumab is used in babies and young children with Hemophilia A to help understand the best ways to treat them.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 50 (estimated) |
| Ages | N/A to 36 Months |
| Sex | All |
| Sponsor | Montefiore Medical Center Academic / other |
| Drugs / interventions | Emicizumab |
| Locations | 10 sites (Hartford, Connecticut and 9 other locations) |
| Trial ID | NCT05248594 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather data on the use of emicizumab in infants and children aged 0-3 years with Hemophilia A. Researchers will collect information through a questionnaire regarding dosing, frequency, and indications for emicizumab, as well as surgical practices related to its use. The study will involve a multi-institutional, retrospective review of pediatric patients treated with emicizumab across several hemophilia treatment centers in the New England region. The goal is to better understand the current practices and considerations for using emicizumab as primary prophylaxis in this young population.
Who should consider this trial
Good fit: Ideal candidates for this study are infants and children aged 0-36 months diagnosed with congenital mild, moderate, or severe Hemophilia A who have been prescribed emicizumab.
Not a fit: Patients with acquired Hemophilia A or those with Hemophilia A and another bleeding disorder may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into optimizing treatment protocols for young children with Hemophilia A, potentially improving their health outcomes.
How similar studies have performed: While this study focuses on a specific age group and treatment, similar studies on emicizumab have shown promising results in older populations, indicating potential for success in this novel application.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Hartford, Connecticut and 9 other locations
- Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center — Hartford, Connecticut, United States (Recruiting)
- Yale Hemophilia Treatment Center — New Haven, Connecticut, United States (Recruiting)
- Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital — Lebanon, New Hampshire, United States (Recruiting)
- Newark Beth Israel — Newark, New Jersey, United States (Recruiting)
- Children's Hospital at Montefiore — Bronx, New York, United States (Recruiting)
- Western NY Blood Care - Research Foundation for SUNY — Buffalo, New York, United States (Recruiting)
- Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell) — New York, New York, United States (Recruiting)
- Mary M Gooley Hemophilia Center, Inc. — Rochester, New York, United States (Recruiting)
- Northwell Health Hemostasis and Thrombosis Center — Staten Island, New York, United States (Recruiting)
- SUNY Upstate Hemophilia Treatment Center - HTC 058 — Syracuse, New York, United States (Recruiting)
Study contacts
- Principal investigator: Jennifer Davila, MD — Children's Hospital at Montefiore
- Study coordinator: Beatriz Casiano, BA
- Email: bcasiano@montefiore.org
- Phone: 718-430-8839
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.