Hemophilia ANCT05248594
Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
An observational study of Hemophilia A, run by Montefiore Medical Center at 10 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 36 months
- Conditions the study is for
- Hemophilia A
- What is being tested?
- HEMLIBRA
- How many people
- 71 participants
- Where
- 10 sitesUnited States
- Study dates
- 1 Aug 2024to 18 Jul 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 36 months
- All sexes
- No healthy volunteers
- Child
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.
Who may be able to join
None listed.
Who cannot join
- Patients with acquired Hemophilia A
- Patients with Hemophilia A and another congenital or acquired bleeding disorder.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Western NY Blood Care - Research Foundation for SUNY
Buffalo, New York, 14202
Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital
Lebanon, New Hampshire, 03766
Yale Hemophilia Treatment Center
New Haven, Connecticut, 06510
Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell)
New York, New York, 10065
Newark Beth Israel
Newark, New Jersey, 07112
Mary M Gooley Hemophilia Center, Inc.
Rochester, New York, 14621
Northwell Health Hemostasis and Thrombosis Center
Staten Island, New York, 11030
SUNY Upstate Hemophilia Treatment Center - HTC 058
Syracuse, New York, 13210
Children's Hospital at Montefiore
The Bronx, New York, 10467
About this study
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.
Read the detailed description
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 has been developed. Data on any pre-, peri and post-surgical practices while on emicizumab is being collected. Pediatricians are being asked if there are plans to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when this is planned on being done. The hope is 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.
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.
- Conditions
- Hemophilia A
- Being tested
- HEMLIBRA
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HEMLIBRADrug
Also called Emicizumab-kxwh
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Within a year.
Secondary outcomes (1)
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.
Measured: Within a year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2022
- Study start1 Aug 2024
- Primary completion18 Jul 2025
- Study completion18 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Montefiore Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Montefiore Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc., Regional Comprehensive Hemophilia Treatment Center (Mount Sinai), Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center, Rutgers Robert Wood Johnson Medical School
- Responsible party
- Sponsor
- Investigators
- Jennifer Davila, MD, Principal investigator, Children's Hospital at Montefiore
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05248594
- Sponsor's protocol ID
- 2021-13264
- Other IDs
- ML43506
- Status last verified
- Sep 2025
- First submitted
- 10 Feb 2022
- Last updated
- 8 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A
- ClinicalTrials.gov ID
- NCT05248594
- Acronym / sponsor ID
- 2021-13264
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 71 participants (actual)
- Lead sponsor
- Montefiore Medical CenterUniversity, hospital or other
- Collaborators
- Genentech, Inc., Regional Comprehensive Hemophilia Treatment Center (Mount Sinai), Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center, Rutgers Robert Wood Johnson Medical School
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 18 Jul 2025 (actual)
- Study completion
- 18 Jul 2025 (actual)
- First posted
- 21 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 has been developed. Data on any pre-, peri and post-surgical practices while on emicizumab is being collected. Pediatricians are being asked if there are plans to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when this is planned on being done. The hope is 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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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.
Key inclusion criteria (0 listed)
Key exclusion criteria (2 listed)
- Patients with acquired Hemophilia A
- Patients with Hemophilia A and another congenital or acquired bleeding disorder.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HEMLIBRAAlso: Emicizumab-kxwhEmicizumab (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. | Drug | – |
Primary outcome
The proportion of patients that are being treated with emicizumab
Time frame: Within a year.
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.
1 secondary outcome
Patients who receive additional doses of factor concentrate while on emicizumab
Time frame: Within a year.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2025.
- Individual participant dataSharing planned: No
Related trials for Hemophilia A
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jennifer Davila, MD principal investigator
Children's Hospital at Montefiore
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Montefiore Medical Center at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of patients that are being treated with emicizumab (time frame: Within a year.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2025.