Hemophilia AEMICARENCT04472169
UPLC-MS/MS Monitoring of Emicizumab Therapy
An observational study of Hemophilia A, run by University Hospital, Lille at 2 sites in France.
Trial summary
Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- How many people
- 100 participants
- Where
- 2 sitesFrance
- Study dates
- 13 Apr 2022to Nov 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 100 patients with severe hemophilia A will be included in the study on 2 french Hemophilia Treatement Centers (CHU Lille and CHU Caen)
Who may be able to join
- Adult or child with Clinical diagnosis of severe hemophilia A (FVIII activity < 1%) with or without inhibitor
- Clinical indication to emicizumab therapy
Who cannot join
- Refusal to give informed consent
- acquired hemophilia A
- other inherited or acquired bleeding disorder
- bodyweight < 10 kgs
Trial locations and contacts
2 sites in 1 country.
France2 sites
CHU de Caen
Caen
Institut Coeur-Poumon, Pôle d'Hématologie-Transfusion, CHU
Lille, 59037
About this study
Official title: Value of Emicizumab Monitoring With UPLC-MS/MS for Bleeding Risk Prediction in Severe Hemophilia A
Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Some cases of neutralizing antidrug antibodies have been also reported. Whether monitoring emicizumab plasma concentration could predict the residual bleeding risk under emicizumab is unknown. As conventional coagulation assays are not adapted for emicizumab monitoring, this study aims to assess the value of monitoring residual emicizumab plasma concentration by UPLC-MS/MS in bleeding risk prediction.
- Conditions
- Hemophilia A
- Keywords
- Drug monitoring, Severe Hemophilia A, Emicizumab
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Severe haemophila A patients with or without inhibitors | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area under the curve ROC of Residual plasma level of emicizumab
At least one clinically significant bleeding (defined as any bleeding treated with FVIII, rFVIIa or aPCC) from loading period completion (week 5) to the end of study, an average of 1 year
Measured: At Week 5 (end of emicizumab loading period)
Secondary outcomes (3)
Residual plasma level of emicizumab measured by UPLC-MS/MS
At least one hemarthrosis from loading period completion (week 5) to the end of study, an average of 1 year
Measured: At Week 5 (end of emicizumab loading period)
Residual plasma level of emicizumab measured by UPLC-MS/MS
Post-traumatic or spontaneous nature of bleeding event
Measured: At each breakthrough bleeding until end of study
Residual plasma level of emicizumab (UPLC-MS/MS dosing)
Emicizumab FVIII-like activity (chromogenic FVIII BIOPHEN™assay system with emicizumab calibration)
Measured: At Week 5 and at each breakthrough bleeding until end of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jul 2020
- Study start13 Apr 2022
- Primary completionNov 2026 (estimated)
- Study completionNov 2026 (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.
- More trials run by University Hospital, LilleThe 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
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Lille
- Sponsor type
- University, hospital or other
- Collaborators
- Groupement Interrégional de Recherche Clinique et d'Innovation
- Responsible party
- Sponsor
- Investigators
- Antoine Rauch, MD,PhD, Principal investigator, University Hospital, Lille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04472169
- Sponsor's protocol ID
- 2019_75
- Other IDs
- 2020-A00584-35
- Acronym
- EMICARE
- Status last verified
- May 2026
- First submitted
- 10 Jul 2020
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Value of Emicizumab Monitoring With UPLC-MS/MS for Bleeding Risk Prediction in Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT04472169
- Acronym / sponsor ID
- EMICARE / 2019_75
- Conditions
- Hemophilia A
- Keywords
- Drug monitoring, Severe Hemophilia A, Emicizumab
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- University Hospital, LilleUniversity, hospital or other
- Collaborators
- Groupement Interrégional de Recherche Clinique et d'Innovation
- Study start
- 13 Apr 2022 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Nov 2026 (estimated)
- First posted
- 15 Jul 2020 (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.
Emicizumab is a monoclonal bispecific antibody with a terminal half-life of 28 days which is now licensed in the treatment of severe haemophilia A with or without inhibitors. Some heterogeneity in residual emicizumab concentrations have been reported according to age, body mass index or drug therapeutic regimen. Some cases of neutralizing antidrug antibodies have been also reported. Whether monitoring emicizumab plasma concentration could predict the residual bleeding risk under emicizumab is unknown. As conventional coagulation assays are not adapted for emicizumab monitoring, this study aims to assess the value of monitoring residual emicizumab plasma concentration by UPLC-MS/MS in bleeding risk prediction.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Severe haemophila A patients with or without inhibitors | – | – |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 100 patients with severe hemophilia A will be included in the study on 2 french Hemophilia Treatement Centers (CHU Lille and CHU Caen)
Key inclusion criteria (2 listed)
- Adult or child with Clinical diagnosis of severe hemophilia A (FVIII activity < 1%) with or without inhibitor
- Clinical indication to emicizumab therapy
Key exclusion criteria (4 listed)
- Refusal to give informed consent
- acquired hemophilia A
- other inherited or acquired bleeding disorder
- bodyweight < 10 kgs
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Area under the curve ROC of Residual plasma level of emicizumab
Time frame: At Week 5 (end of emicizumab loading period)
At least one clinically significant bleeding (defined as any bleeding treated with FVIII, rFVIIa or aPCC) from loading period completion (week 5) to the end of study, an average of 1 year
3 secondary outcomes
Residual plasma level of emicizumab measured by UPLC-MS/MS
Time frame: At Week 5 (end of emicizumab loading period)
Residual plasma level of emicizumab measured by UPLC-MS/MS
Time frame: At each breakthrough bleeding until end of study
Residual plasma level of emicizumab (UPLC-MS/MS dosing)
Time frame: At Week 5 and at each breakthrough bleeding until end of study
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2026.
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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.
Antoine Rauch, MD,PhD principal investigator
University Hospital, Lille
2 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 University Hospital, Lille at 2 sites in France.
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 2 inclusion and 4 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: under way, not looking for participants, as of the sponsor's last update on 20 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. 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?
This study is still under way. Results are not available in the source record yet.
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?
Area under the curve ROC of Residual plasma level of emicizumab (time frame: At Week 5 (end of emicizumab loading period)). 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 20 May 2026.