Hemophilia AEarly Phase 1NCT05281718
Factor IX as Adjunctive Therapy to Emicizumab (EMIX)
A Early Phase 1 study testing Factor IX in people with Hemophilia A, run by Tulane University at 2 sites in United States.
Trial summary
This trial will enroll patients with severe hemophilia A. Experiments will be run in vitro by spiking patients' blood with different molecules (currently used and theoretically proposed as adjunctive therapy to emicizumab), therefore no more than minimal direct risk to patients is expected. Read more about this study
- Who is it for?
- Ages 12 to 120 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Factor IX
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Aug 2022to 14 Jul 2024
- Phase
- Early Phase 1
- 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 12 to 120 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed Informed Consent/Assent Form
- Male patients, Age 12 years and older at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of severe hemophilia A and are receiving emicizumab prophylaxis, on a weekly maintenance regimen
Who cannot join
- Not able to sign consent
- Not willing to provide extra blood for the experiments.
- Patients carrying the diagnoses of other coagulopathies in addition to hemophilia A
- Patients that have received any hemostatic agent within 5 half-lives of the blood draw. This applies to any agent that might have been used for any reason prior to the blood draw and might affect the global hemostasis.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Tulane University Lakeside Hospital and Clinics
Metairie, Louisiana, 70001
University Medical Center New Orleans
New Orleans, Louisiana, 70112
About this study
Official title: An in Vitro Pilot Study of the Role of Factor IX as an Alternative to Bypassing Agents in the Treatment of Breakthrough Bleeds in Patients With Hemophilia A and Inhibitors Treated With Emicizumab.
This trial will enroll patients with severe hemophilia A. Experiments will be run in vitro by spiking patients' blood with different molecules (currently used and theoretically proposed as adjunctive therapy to emicizumab), therefore no more than minimal direct risk to patients is expected. This is a pilot preclinical study.
Read the detailed description
The study aims to determine the hemostatic effect of different Factor IX (FIX) concentrates when added to the plasma of patients on emicizumab prophylaxis and compare it to the hemostatic effect obtained with the addition of bypassing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII).
The investigators will study the hemostatic effect using Thrombin Generation assay (CAT) in the presence of different concentrations of FIX concentrates. The investigators will compare the hemostatic effect of added FIX to the effect of added bypassing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII)
Plasma will be collected from patients with hemophilia A and inhibitors treated with Emicizumab, a group of plasma samples will be spiked with different concentrations of Factor IX. Thrombin generation profiles will be obtained using CAT.
Another group of plasma samples will be spiked with different concentrations of by passing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII). Thrombin generation profiles will be obtained using CAT.
- Conditions
- Hemophilia A
- Being tested
- Factor IX
- Keywords
- Hemophilia A with inhibitors, In vitro, 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.
Factor IXDrug
Factor IX is on of the substrates of emicizumab.
| Study group | Role | What they receive |
|---|---|---|
| Patient Group | Experimental | In vitro spiking experiments will be realized in plasmas from patients with severe haemophilia A on emicizumab using increasing concentrations of factor IX (rFIX), Activated prothrombin complex (aPCC) and recombinant VIIa (rFVIIa). Receives: Drug: Factor IX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin
Peak thrombin is a thrombin generation profile. The investigators will compare the peak thrombin obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with aPCC and rFVIIa on emicizumab prophylaxis.
Measured: 1 Year
Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin
The investigators will compare the peak thrombin obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with recombinant porcine FVIII on emicizumab prophylaxis.
Measured: 1 Year
Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP)
Endogenous Thrombin Potential is a thrombin generation profile. The investigators will compare the Endogenous Thrombin Potential obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with aPCC and rFVIIa on emicizumab prophylaxis.
Measured: 1 Year
Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP)
The investigators will compare the Endogenous Thrombin Potential obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with recombinant porcine FVIII on emicizumab prophylaxis.
Measured: 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2022
- Study start1 Aug 2022
- Primary completion7 Mar 2024
- Study completion14 Jul 2024
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 Tulane UniversityThe 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
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open Label
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Tulane University
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Maissa Janbain, MD, MS, Principal investigator, Tulane University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05281718
- Sponsor's protocol ID
- 2022-200
- Status last verified
- Jan 2025
- First submitted
- 11 Feb 2022
- Last updated
- 20 Jan 2025
- Results posted
- No
- 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 20 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An in Vitro Pilot Study of the Role of Factor IX as an Alternative to Bypassing Agents in the Treatment of Breakthrough Bleeds in Patients With Hemophilia A and Inhibitors Treated With Emicizumab.
- ClinicalTrials.gov ID
- NCT05281718
- Acronym / sponsor ID
- 2022-200
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A with inhibitors, In vitro, Emicizumab
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Tulane UniversityUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- 7 Mar 2024 (actual)
- Study completion
- 14 Jul 2024 (actual)
- First posted
- 16 Mar 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.
This trial will enroll patients with severe hemophilia A. Experiments will be run in vitro by spiking patients' blood with different molecules (currently used and theoretically proposed as adjunctive therapy to emicizumab), therefore no more than minimal direct risk to patients is expected. This is a pilot preclinical study.
Detailed description
The study aims to determine the hemostatic effect of different Factor IX (FIX) concentrates when added to the plasma of patients on emicizumab prophylaxis and compare it to the hemostatic effect obtained with the addition of bypassing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII).
The investigators will study the hemostatic effect using Thrombin Generation assay (CAT) in the presence of different concentrations of FIX concentrates. The investigators will compare the hemostatic effect of added FIX to the effect of added bypassing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII)
Plasma will be collected from patients with hemophilia A and inhibitors treated with Emicizumab, a group of plasma samples will be spiked with different concentrations of Factor IX. Thrombin generation profiles will be obtained using CAT.
Another group of plasma samples will be spiked with different concentrations of by passing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII). Thrombin generation profiles will be obtained using CAT.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open Label
| Arm | Type | Receives |
|---|---|---|
| Patient Group | Experimental | Drug: Factor IXIn vitro spiking experiments will be realized in plasmas from patients with severe haemophilia A on emicizumab using increasing concentrations of factor IX (rFIX), Activated prothrombin complex (aPCC) and recombinant VIIa (rFVIIa). |
Who may qualify
- Ages
- 12 to 120 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Signed Informed Consent/Assent Form
- Male patients, Age 12 years and older at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Diagnosis of severe hemophilia A and are receiving emicizumab prophylaxis, on a weekly maintenance regimen
Key exclusion criteria (4 listed)
- Not able to sign consent
- Not willing to provide extra blood for the experiments.
- Patients carrying the diagnoses of other coagulopathies in addition to hemophilia A
- Patients that have received any hemostatic agent within 5 half-lives of the blood draw. This applies to any agent that might have been used for any reason prior to the blood draw and might affect the global hemostasis.
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 |
|---|---|---|
| Factor IXFactor IX is on of the substrates of emicizumab. | Drug | Patient Group |
Primary outcome
Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin
Time frame: 1 Year
Peak thrombin is a thrombin generation profile. The investigators will compare the peak thrombin obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with aPCC and rFVIIa on emicizumab prophylaxis.
Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin
Time frame: 1 Year
The investigators will compare the peak thrombin obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with recombinant porcine FVIII on emicizumab prophylaxis.
Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP)
Time frame: 1 Year
Endogenous Thrombin Potential is a thrombin generation profile. The investigators will compare the Endogenous Thrombin Potential obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with aPCC and rFVIIa on emicizumab prophylaxis.
Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP)
Time frame: 1 Year
The investigators will compare the Endogenous Thrombin Potential obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with recombinant porcine FVIII on emicizumab prophylaxis.
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 20 Jan 2025.
- 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.
Maissa Janbain, MD, MS principal investigator
Tulane University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Factor IX in people with Hemophilia A, run by Tulane University at 2 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 4 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: finished, as of the sponsor's last update on 20 Jan 2025. 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 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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin (time frame: 1 Year). The study lists 4 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 20 Jan 2025.