Clinical trial NCT05248594: 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophilia A
Lead sponsor: Montefiore Medical Center
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2022-02-21
Enrollment: 50
Sex: ALL
Maximum age (years): 36
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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.

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. 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.

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.

Primary outcomes:
measure: The proportion of patients that are being treated with emicizumab
timeFrame: Within a year.
Secondary outcomes:
measure: Patients who receive additional doses of factor concentrate while on emicizumab
timeFrame: Within a year.
Other outcomes:
measure: Patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data
timeFrame: Within a year.
Locations:
city: Hartford
country: United States
facility: Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center
state: Connecticut
city: New Haven
country: United States
facility: Yale Hemophilia Treatment Center
state: Connecticut
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital
state: New Hampshire
city: Newark
country: United States
facility: Newark Beth Israel
state: New Jersey
city: Bronx
country: United States
facility: Children's Hospital at Montefiore
state: New York
city: Buffalo
country: United States
facility: Western NY Blood Care - Research Foundation for SUNY
state: New York
city: New York
country: United States
facility: Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell)
state: New York
city: Rochester
country: United States
facility: Mary M Gooley Hemophilia Center, Inc.
state: New York
city: Staten Island
country: United States
facility: Northwell Health Hemostasis and Thrombosis Center
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Hemophilia Treatment Center - HTC 058
state: New York
Study officials:
affiliation: Children's Hospital at Montefiore
name: Jennifer Davila, MD
role: PRINCIPAL_INVESTIGATOR
References: