Hemophilia AML43518NCT06104826
Health-Related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab
An observational study of Hemophilia A, run by Newark Beth Israel Medical Center at 1 site in United States.
Trial summary
The main purposes of this study are to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes, if it improves quality of life of individuals and if improves arthropathy in persons with hemophilia A. Read more about this study
- Who is it for?
- Ages 4 years and olderHealthy volunteers can take part
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Emicizumab
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2023to Dec 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 4 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: The study population will include children and adults who are males and females with hemophilia A for all parts of the study. Children and adults are equally at risk for bleeding and hence we would collect information about bleeding events in both children and adults on emicizumab or on factor concentrates to calculate ABR. There will be no exclusion based on race or ethnicity. For HRQoL part of the study, children and their families will be included.The Quality of life was found to be poor both among children affected with hemophilia and their parents and perceived impact on family. Hemophilic arthropathy can be seen in children and adults and therefore, this outcome must be studied in both populations as it can be disabling at any age.
Who may be able to join
- Signed Informed Consent/Assent Form
- Age 4 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of hemophilia A (any severity) on emicizumab or factor concentrate prophylaxis for at least 12 months each
- Plan to be adherent to emicizumab prophylaxis during the study
- Patient with inhibitors
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- Patient without inhibitors Part B: HRQoL and Arthropathy
- Signed Informed Consent/Assent Form
- Age 4 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of hemophilia A (any severity) who has decided to switch to emicizumab prophylaxis
- Plan to be adherent to emicizumab prophylaxis during the study
- Individuals of childbearing age will be included in this study. Their participation involves voluntary completion of a questionnaire. There are no additional precautions required for this population.
- Patient/parent must be able to read and write English/Spanish
Who cannot join
- Part A: ABR
- Patients who are on a clinical trial for prophylaxis
- Patients with other bleeding disorders needing any scheduled treatment
- No eligibility restrictions will be based on gender, race, ethnic background or economic status
- Part B: HRQoL and Arthropathy
- Patients who are on a clinical trial for prophylaxis
Show all 9Show fewer
- Patients with other bleeding disorders needing any scheduled treatment
- Patients/parents who refuse to complete questionnaire or have joint health assessment by HJHS
- No eligibility restrictions will be based on gender, race, ethnic background or economic status
Trial locations and contacts
1 site in 1 country.
United States1 site
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Pari Sheerer, 973-803-7218, pari.sheerer2@rwjbh.org
Shalu Narang, MD
About this study
Official title: Health-related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab: Real World Data
The main purposes of this study are to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes, if it improves quality of life of individuals and if improves arthropathy in persons with hemophilia A.
Read the detailed description
The main purpose of this study is to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes and if it affects quality of life of individuals with hemophilia.
Patients with hemophilia A, especially those with severe disease, have recurrent bleeding in joints, muscles and deep tissue and recurrent bleeding in the same joint can result in irreversible damage in bone and cartilage and lead to disabling arthropathy. Treatment involves replacement with exogenous recombinant factor concentrates that can prevent further bleeding episodes but not the ongoing joint damage. In addition, treatment with recombinant factor concentrates can lead to inhibitory antibodies resulting in inability to use those for further treatment. The burden of this disease on patients and caregivers is well recognized.
Participant will be asked about bleeding episodes needing additional factor replacement in addition to checking bleeding log prior to starting emicizumab and at additional times after starting emicizumab. In addition, investigators would ask participant to complete questionnaire about participant's and/or their caregiver's quality of life prior to starting emicizumab and at additional times after starting emicizumab. Participant will also have joint scores measured before and at additional times after starting emicizumab. All the information collected will remain confidential and would only be shared with members of study team.
- Conditions
- Hemophilia A
- Being tested
- Emicizumab
- Keywords
- Bleeding Disorder, Hemophilia, Quality of Life Outcomes, Emicizumab
- Medical subject headings
- Hemophilia A, Hemostatic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmicizumabDrug
Emicizumab in Hemophilia A
What is being measured
The primary outcome is the main question the study is designed to answer.
To obtain and compare the annual bleeding rate in patients with hemophilia A on emicizumab before and after being on emicizumab.
Annualized Bleeding Rates (ABR)
Measured: 12 months
To evaluate the health related quality of life in patients with hemophilia A before and after being on emicizumab.
Quality of Life Questionaries
Measured: 12 months
To study any clinical changes in arthropathy.
Joint score measurements
Measured: 12 months
Secondary outcomes (1)
To study practitioner's preference for emicizumab schedule and how it affects compliance
ABR
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2023
- Study startDec 2023 (estimated)
- Primary completionDec 2024 (estimated)
- Study completionDec 2025 (estimated)
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.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Newark Beth Israel Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Principal investigator, Narang Shalu, Newark Beth Israel Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06104826
- Sponsor's protocol ID
- 2023.20
- Acronym
- ML43518
- Status last verified
- Oct 2023
- First submitted
- 23 Oct 2023
- Last updated
- 30 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Health-related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab: Real World Data
- ClinicalTrials.gov ID
- NCT06104826
- Acronym / sponsor ID
- ML43518 / 2023.20
- Conditions
- Hemophilia A
- Keywords
- Bleeding Disorder, Hemophilia, Quality of Life Outcomes, Emicizumab
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Newark Beth Israel Medical CenterUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Dec 2023 (estimated)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 27 Oct 2023 (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.
The main purposes of this study are to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes, if it improves quality of life of individuals and if improves arthropathy in persons with hemophilia A.
Detailed description
The main purpose of this study is to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes and if it affects quality of life of individuals with hemophilia.
Patients with hemophilia A, especially those with severe disease, have recurrent bleeding in joints, muscles and deep tissue and recurrent bleeding in the same joint can result in irreversible damage in bone and cartilage and lead to disabling arthropathy. Treatment involves replacement with exogenous recombinant factor concentrates that can prevent further bleeding episodes but not the ongoing joint damage. In addition, treatment with recombinant factor concentrates can lead to inhibitory antibodies resulting in inability to use those for further treatment. The burden of this disease on patients and caregivers is well recognized.
Participant will be asked about bleeding episodes needing additional factor replacement in addition to checking bleeding log prior to starting emicizumab and at additional times after starting emicizumab. In addition, investigators would ask participant to complete questionnaire about participant's and/or their caregiver's quality of life prior to starting emicizumab and at additional times after starting emicizumab. Participant will also have joint scores measured before and at additional times after starting emicizumab. All the information collected will remain confidential and would only be shared with members of study team.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population will include children and adults who are males and females with hemophilia A for all parts of the study. Children and adults are equally at risk for bleeding and hence we would collect information about bleeding events in both children and adults on emicizumab or on factor concentrates to calculate ABR. There will be no exclusion based on race or ethnicity. For HRQoL part of the study, children and their families will be included.The Quality of life was found to be poor both among children affected with hemophilia and their parents and perceived impact on family. Hemophilic arthropathy can be seen in children and adults and therefore, this outcome must be studied in both populations as it can be disabling at any age.
Key inclusion criteria (14 listed)
- Signed Informed Consent/Assent Form
- Age 4 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of hemophilia A (any severity) on emicizumab or factor concentrate prophylaxis for at least 12 months each
- Plan to be adherent to emicizumab prophylaxis during the study
Key exclusion criteria (9 listed)
- Part A: ABR
- Part B: HRQoL and Arthropathy
- Patients who are on a clinical trial for prophylaxis
- Patients with other bleeding disorders needing any scheduled treatment
- Patients/parents who refuse to complete questionnaire or have joint health assessment by HJHS
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 |
|---|---|---|
| EmicizumabEmicizumab in Hemophilia A | Drug | – |
Primary outcome
To obtain and compare the annual bleeding rate in patients with hemophilia A on emicizumab before and after being on emicizumab.
Time frame: 12 months
Annualized Bleeding Rates (ABR)
To evaluate the health related quality of life in patients with hemophilia A before and after being on emicizumab.
Time frame: 12 months
Quality of Life Questionaries
To study any clinical changes in arthropathy.
Time frame: 12 months
Joint score measurements
1 secondary outcome
To study practitioner's preference for emicizumab schedule and how it affects compliance
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2023.
- Individual participant dataSharing planned: Undecided
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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.
1 site 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 Newark Beth Israel Medical Center at 1 site 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 14 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 30 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Pari Sheerer (973-803-7218, pari.sheerer2@rwjbh.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To obtain and compare the annual bleeding rate in patients with hemophilia A on emicizumab before and after being on emicizumab. (time frame: 12 months). The study lists 3 primary outcomes in all. 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 30 Oct 2023.