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

NCT05248594 | Completed DMC FDA Drug

• 2 other identifiers: 2021-13264ML43506
• Study Type: observational
• Target: 71
• Locations: 1 country
• Sites: 10

Brief Summary

Hemophilia A (HA) is a rare congenital bleeding disorder characterized by coagulation factor VIII deficiency. In severe HA, defined as plasma FVIII clotting activity \< 1%, bleeding may frequently occur spontaneously, most commonly in joints, leading to painful hemophilic arthropathy and loss of joint function. Patients with moderate or mild hemophilia A, defined as FVIII clotting activity between 1-\<5% and 5-40% respectively, are less likely to have spontaneous bleeding however can have significant bleeding with trauma or surgery. Perioperative management by a hematologist who specializes in hemophilia is needed to ensure hemostasis during surgery. Hemophilia is an X-linked recessive disorder affecting 1 in 5000 to 10,000 males.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Patients who receive additional doses of factor concentrate while on emicizumab

  Within a year.

Other Outcomes (1)

• Patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data

  Within a year.

Interventions

HEMLIBRA DRUG

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.

Also known as: Emicizumab-kxwh

Eligibility Criteria

• Age: Up to 36 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• 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

You may not qualify if:

• Patients with acquired Hemophilia A
• Patients with Hemophilia A and another congenital or acquired bleeding disorder.

Sponsors & Collaborators

• Montefiore Medical Center: lead
• Genentech, Inc.: collaborator
• Regional Comprehensive Hemophilia Treatment Center (Mount Sinai): collaborator
• Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center: collaborator
• Rutgers Robert Wood Johnson Medical School: collaborator

Study Sites (10)

Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

Location

Yale Hemophilia Treatment Center

New Haven, Connecticut, 06510, United States

Location

Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital

Lebanon, New Hampshire, 03766, United States

Location

Newark Beth Israel

Newark, New Jersey, 07112, United States

Location

Western NY Blood Care - Research Foundation for SUNY

Buffalo, New York, 14202, United States

Location

Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell)

New York, New York, 10065, United States

Location

Mary M Gooley Hemophilia Center, Inc.

Rochester, New York, 14621, United States

Location

Northwell Health Hemostasis and Thrombosis Center

Staten Island, New York, 11030, United States

Location

SUNY Upstate Hemophilia Treatment Center - HTC 058

Syracuse, New York, 13210, United States

Location

Children's Hospital at Montefiore

The Bronx, New York, 10467, United States

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

emicizumab

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jennifer Davila, MD

  Children's Hospital at Montefiore

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2022
• First Posted: February 21, 2022
• Study Start: August 1, 2024
• Primary Completion: July 18, 2025
• Study Completion: July 18, 2025
• Last Updated: September 8, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)