Glanzmann ThrombastheniaPhase 2STRENGTHNCT07136857
Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)
A Phase 2 study testing EPTACOG BETA in people with Glanzmann Thrombasthenia, run by Emory University at 1 site in United States.
Trial summary
This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. Eptacog beta (EB) is not currently approved by the U.S. Food and Drug Administration (FDA) for this condition. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Glanzmann Thrombasthenia
- What is being tested?
- EPTACOG BETA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Oct 2025to Dec 2026
- Phase
- Phase 2
- 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
Who may be able to join
- Adult or Pediatric persons with inherited Glanzmann thrombasthenia (see diagnostic criteria below)
- Severe bleeding phenotype
- Adequate hepatic function
- Adequate renal function
- Adults subject (≥18 years of age) or caregiver (parent or legally authorized representative) for minor subjects, subjects with cognitive impairment, or subjects with impaired decision-making capacity have provided written informed consent, and the participant has given consent/assent (if applicable)
- Ability to speak, read, and understand the English language
Who cannot join
- Thrombocytopenia (platelet count < 100k)
- Acquired Glanzmann thrombasthenia secondary to autoimmune disease, malignancy, or medication
- Inherited or acquired bleeding diathesis other than Glanzmann thrombasthenia
- Have a history of venous or arterial thrombotic event within 2 years of study enrollment
- Active malignancy
- Known or suspected hypersensitivity to rabbits, rabbit protein, other forms of rFVIIa, or to any of the EB excipients
Show all 11Show fewer
- Have received an investigational drug within 30 days or within 5 half-lives of that investigational drug (whichever is longer) or are expected to receive such a drug during participation in this study
- Be using aspirin, non-steroidal anti-inflammatory drugs (NSAIDS), herbs, natural medications, or other drugs with platelet inhibitor properties for the duration of the study
- Be using or administered anticoagulant agents for the duration of the study
- Have any life-threatening disease or other disease or condition which, according to the investigator's judgement, could imply a potential hazard to the patient, or interfere with the study participation or study outcome
- Use of systemic immunomodulators at enrollment or planned use during the study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Arthur M. Blank Hospital | Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
About this study
Official title: Study To Assess Response to Eptacog Beta iN Patients With Glanzmann THromboasthenia (STRENGTH )
This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. Eptacog beta (EB) is not currently approved by the U.S. Food and Drug Administration (FDA) for this condition.
The study will assess the effectiveness and safety of eptacog beta (EB) when used to treat serious bleeding events, and in an optional phase, when used routinely to prevent bleeding. During the first three (3) months, participants will manage any bleeding episodes with their standard treatment (e.g., factor products or platelet transfusions). After this initial period, they will use the study drug to treat serious bleeding events.
Read more
Participants will have approximately 4 to 5 visits with their hematologist over the 9-month study period. They will also be asked to complete a diary documenting bleeding episodes and treatments, and to answer questions about how bleeding affects their daily life. Blood samples will be collected to monitor their condition and any potential side effects of the study drug.
At the end of the main study, participants will have the option to enter an optional extension phase, where they will receive routine intravenous infusions of the study drug 2 to 3 times per week for 6 months to help prevent future bleeding episodes and complications.
Read the detailed description
This study aims to investigate eptacog beta (EB), a new form of recombinant activated factor VIIa, for bleeding management in persons with Glanzmann thrombasthenia regardless of platelet refractoriness status.
Investigators will enroll six (6) people (adult or pediatric) with Glanzmann thrombasthenia (GT) who have a severe bleeding phenotype (defined as ≥ 2 treated bleeding events in the past 12 months, ≥ 1 hospitalization for bleeding or severe anemia or requiring prophylactic therapy to prevent bleeding). Initially, a retrospective chart review will collect data regarding bleeding events and their management over the previous 6 months before enrollment.
The initial 3 months of this trial will be a non-interventional phase during which time, participants will receive standard-of-care on-demand therapy for acute bleeding events at the discretion of their hematologist. They will complete a diary logging their bleeding events and their management. Hemostatic efficacy following acute bleed treatment will be evaluated using a 4-point hemostasis efficacy score.
Following this initial non-interventional phase, participants will use eptacog beta (EB) 75 mcg/kg/dose every 3 Hours for the management of breakthrough bleeding episodes that are not responsive to local hemostatic control and anti-fibrinolytic therapies, with the precise frequency of infusions and duration of treatment at the discretion of the subject's treating physician. This on-demand interventional phase will last 6 months. During this time, participants will log their acute bleeding episodes and management. At varying time points following a dose of EB, participants will complete a 4-point hemostasis scale assessing the efficacy of EB in controlling bleeding.
Following the 6-month interventional on-demand phase, participants may choose to continue in an interventional prophylaxis arm.
- Conditions
- Glanzmann Thrombasthenia
- Being tested
- EPTACOG BETA
- Keywords
- Severe Bleeding, Prevention of Bleeding, Eptacog beta (EB)
- Medical subject headings
- Thrombasthenia, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPTACOG BETADrug
Also called SEVENTFACT®; EB
Eptacog beta (SEVENFACT®; EB) is a lyophilized powder in single-use vials (1, 2, or 5 mg) of coagulation factor VIIa (recombinant)-jncw. It is reconstituted with sterile water (provided in the kit) and administered intravenously.
SEVENFACT® may be administered by a healthcare provider (HCP) at the study site or home, or by a trained participant/caregiver. Participants will be monitored in a healthcare facility for 60 minutes after the first dose for hypersensitivity.
| Study group | Role | What they receive |
|---|---|---|
| Eptacog beta | Experimental | For the initial 3 months, participants will be in a non-interventional phase and receive standard-of-care on-demand therapy for acute bleeding at their hematologist's discretion. Following this non-interventional phase, they will use eptacog beta on-demand to treat acute bleeding for 6 months. Throughout the study, they will log bleeding events and management in a diary. Hemostatic efficacy after treatment will be assessed using a 4-point hemostasis efficacy scale.
Participants will have the option to re-consent for an exploratory phase, adding 6 months to their time on the study (total of 15 months). For those entering the optional extension phase, eptacog beta will be routinely administered for bleed prophylaxis. The treating hematologist will determine the best method/location for infusions. The number of bleeding events over time will be compared between the prophylaxis phase and the on-demand phase. Receives: Drug: EPTACOG BETA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of bleeding events successfully treated within 24 hours of the first Eptacog Beta (EB) administration
Proportion of acute bleeding events successfully treated with EB within 24 hours of the first EB administration based on an "excellent or good" response on a 4-point hemostatic efficacy scale and without rebleeding before 36 hours.
Hemostatic Efficacy Scale:
None = No effect; bleeding unchanged or worsened; continued treatment needed.
Moderate = Some effect (e.g., pain decreased, bleeding signs improved) but bleeding continued; further treatment needed.
Good = Bleeding symptoms are largely reduced but not completely resolved; no further EB infusions are needed.
Excellent = Full relief of bleeding symptoms; no additional EB infusion required.
Measured: 24 hours after the first EB dose administration
Number of bleeding events over the study period
The number of bleeding events occurring while receiving routine EB prophylaxis will be compared with the number of bleeding events that occurred during on-demand therapy.
*This outcome applies only to the optional interventional prophylaxis phase.
Measured: 6 months following the start of the interventional prophylaxis phase
Secondary outcomes (15)
Number of treatment-Emergent Adverse Events and Clinically Significant Laboratory Abnormalities
Safety of EB will be measured by the number of treatment-emergent adverse events, treatment-emergent serious adverse events, and treatment-emergent potentially clinically significant laboratory values reported during the interventional phases compared to those reported during the non-interventional observational phase.
Measured: 6 months following the start of EB administration for each study phase
EuroQol five-dimensional questionnaire for youth (EQ-5D-Y) score for participants 4-15 years of age.
The EQ-5D-Y-5L is a child-friendly, five-dimension, five-level instrument assessing: Mobility, Looking After Myself, Doing Usual Activities, Having Pain or Discomfort, and Feeling Worried, Sad, or Unhappy.
Each dimension has five response levels (1 = no problems to 5 = extreme problems/unable to do). Respondents select one statement per dimension describing their health on the assessment day, forming a 5-digit profile summarizing their health state. For example, code 11234 indicates no problems with mobility or looking after myself, slight problems doing usual activities, some pain or discomfort, and feeling very worried, sad, or unhappy. Higher scores reflect greater severity.
*15 months applies to the optional phase only.
Measured: Baseline, 9 and 15 months after baseline
EuroQol five-dimensional 5 Level (EQ-5D-5L) score for participants ≥16 years
The EuroQol 5 Dimension 5 Level (EQ-5D-5L) is a self-report survey that measures quality of life across 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. It captures how people feel across five dimensions, each of which has five levels: 1 = no problems to 5 = extreme problems/unable to do. The five answers produce a unique 5-digit health state, for example, 11234, that describes the young person's health state. For example, the code 11234 describes a health state where there are no problems with walking about (mobility) or looking after myself, a little bit of a problem with doing usual activities, some pain or discomfort, and feeling really worried, sad, or unhappy.
*15 months applies to the optional phase only.
Measured: Baseline, 9 and 15 months after baseline
Patient Reported Outcomes Measurement Information System (PROMIS) fatigue short form for adults
The PROMIS Fatigue item banks assess a range of self-reported symptoms, from mild subjective feelings of tiredness to an overwhelming, debilitating, and sustained sense of exhaustion that likely decreases one's ability to execute daily activities and function normally in family or social roles. Fatigue is divided into the experience of fatigue (frequency, duration, and intensity) and the impact of fatigue on physical, mental, and social activities. The fatigue short forms are universal and assess fatigue over the past seven days. PROMIS measures use a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. On the T-score metric:
A score of 40 is one SD lower than the mean of the reference population. A score of 60 is one SD higher than the mean of the reference population. Higher scores equal more fatigue.
*15 months applies to the optional phase only.
Measured: Baseline, 9 and 15 months after baseline
PROMIS fatigue short form (pediatric version)
The PROMIS Pediatric Fatigue Short Form will be used to assess fatigue based on age: self-report (ages 8-17) and parent-proxy (ages 5-17). It measures fatigue symptoms and their impact on physical, mental, and social activities over the past 7 days. Items reflect both the frequency and severity of fatigue. Scores are reported as T-scores (mean = 50, SD = 10) based on a reference population. A higher T-score indicates greater fatigue (e.g., 60 = 1 SD above the mean). This measure captures the participant's ability to carry out daily activities and participate in usual roles.
*15 months applies to the optional phase only.
Measured: Baseline, 9 and 15 months after baseline
Menstrual impact questionnaire (MIQ) score
The MIQ was developed to assess the effect of heavy menstrual bleeding (menorrhagia) on various aspects of a woman's quality of life (QoL). It is a brief, 6-item patient-reported outcome measure that focuses on: Perceived amount of blood loss, limitations in work, physical and social activities, and changes due to treatment. Each item is rated on a 6-point scale, indicating the severity of impact (e.g., 0 = no effect, up to 5 = severe effect).
Total Score = Sum of all 6 items (range 0-30), with higher scores reflecting a greater negative impact of menorrhagia on daily life.
Measured: Baseline, 9 and 15 months after baseline
Exogenous thrombin potential (ETP) following ex vivo EB
ETP concentration following ex vivo spiking in various concentrations of EB. Samples will be compared to baseline participants and healthy control samples.
Measured: Baseline (pre-infusion)
Lag time and time to peak thrombin following ex vivo EB
Lag time and time to peak thrombin following ex vivo spiking in various concentrations of EB. Samples will be compared to baseline participants and healthy control samples.
Measured: Baseline (pre-infusion)
Peak thrombin levels following ex vivo EB
Peak thrombin levels following ex vivo spiking in various concentrations of EB. Samples will be compared to baseline participants and healthy control samples.
Measured: Baseline (pre-infusion)
ETP levels following in vivo EB
ETP thrombin levels at 30 and 60 minutes following in vivo infusion of EB. Samples will be compared to pre-infusion baseline participant and healthy control samples.
Measured: Preinfusion, 30 and 60 minutes following the first EB infusion
Lag time and time to peak thrombin following in vivo EB infusion
Lag time and time to peak thrombin at 30 and 60 minutes following in vivo infusion of EB. Samples will be compared to pre-infusion baseline patient PRP and healthy control samples.
Measured: Preinfusion, 30 and 60 minutes following the first EB infusion
Peak thrombin following in vivo EB infusion
Peak thrombin at 30 and 60 minutes following in vivo infusion of EB. Samples will be compared to pre-infusion baseline patient platelet-rich plasma (PRP) and healthy control samples.
Measured: Preinfusion, 30 and 60 minutes following the first EB infusion
Clotting time (CT) and clotting formation time (CFT) following in vivo EB infusion
CT and CFT will be measured using rotational thromboelastometry following ex vivo spiking with various concentrations of EB. Samples will be compared to baseline samples and healthy control samples.
Measured: Preinfusion, 30 and 60 minutes following the first EB infusion
Maximum clot firmness (MCF) following in vivo EB infusion
MCF will be measured using rotational thromboelastometry following ex vivo spiking with various concentrations of EB. Samples will be compared to baseline samples and healthy control samples.
Measured: Preinfusion, 30 and 60 minutes following the first EB infusion
Alpha angle following ex vivo EB
The alpha angle will be measured using rotational thromboelastometry following ex vivo spiking with various concentrations of EB. Samples will be compared to baseline samples and healthy control samples.
Measured: Preinfusion, 30 and 60 minutes following the first EB infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2025
- Study start2 Oct 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glanzmann ThrombastheniaStudies of the same condition, open and under way.
- More trials run by Emory 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Laboratoire français de Fractionnement et de Biotechnologies
- Responsible party
- Principal investigator, Karen L. Zimowski, Emory University
- Investigators
- Karen Zimowski, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07136857
- Sponsor's protocol ID
- STUDY00009481
- Other IDs
- 2025P010364
- Acronym
- STRENGTH
- Status last verified
- Nov 2025
- First submitted
- 15 Aug 2025
- Last updated
- 1 Dec 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 1 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study To Assess Response to Eptacog Beta iN Patients With Glanzmann THromboasthenia (STRENGTH )
- ClinicalTrials.gov ID
- NCT07136857
- Acronym / sponsor ID
- STRENGTH / STUDY00009481
- Conditions
- Glanzmann Thrombasthenia
- Keywords
- Severe Bleeding, Prevention of Bleeding, Eptacog beta (EB)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Laboratoire français de Fractionnement et de Biotechnologies
- Study start
- 2 Oct 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 22 Aug 2025 (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 study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. Eptacog beta (EB) is not currently approved by the U.S. Food and Drug Administration (FDA) for this condition.
The study will assess the effectiveness and safety of eptacog beta (EB) when used to treat serious bleeding events, and in an optional phase, when used routinely to prevent bleeding. During the first three (3) months, participants will manage any bleeding episodes with their standard treatment (e.g., factor products or platelet transfusions). After this initial period, they will use the study drug to treat serious bleeding events.
Participants will have approximately 4 to 5 visits with their hematologist over the 9-month study period. They will also be asked to complete a diary documenting bleeding episodes and treatments, and to answer questions about how bleeding affects their daily life. Blood samples will be collected to monitor their condition and any potential side effects of the study drug.
At the end of the main study, participants will have the option to enter an optional extension phase, where they will receive routine intravenous infusions of the study drug 2 to 3 times per week for 6 months to help prevent future bleeding episodes and complications.
Detailed description
This study aims to investigate eptacog beta (EB), a new form of recombinant activated factor VIIa, for bleeding management in persons with Glanzmann thrombasthenia regardless of platelet refractoriness status.
Investigators will enroll six (6) people (adult or pediatric) with Glanzmann thrombasthenia (GT) who have a severe bleeding phenotype (defined as ≥ 2 treated bleeding events in the past 12 months, ≥ 1 hospitalization for bleeding or severe anemia or requiring prophylactic therapy to prevent bleeding). Initially, a retrospective chart review will collect data regarding bleeding events and their management over the previous 6 months before enrollment.
The initial 3 months of this trial will be a non-interventional phase during which time, participants will receive standard-of-care on-demand therapy for acute bleeding events at the discretion of their hematologist. They will complete a diary logging their bleeding events and their management. Hemostatic efficacy following acute bleed treatment will be evaluated using a 4-point hemostasis efficacy score.
Following this initial non-interventional phase, participants will use eptacog beta (EB) 75 mcg/kg/dose every 3 Hours for the management of breakthrough bleeding episodes that are not responsive to local hemostatic control and anti-fibrinolytic therapies, with the precise frequency of infusions and duration of treatment at the discretion of the subject's treating physician. This on-demand interventional phase will last 6 months. During this time, participants will log their acute bleeding episodes and management. At varying time points following a dose of EB, participants will complete a 4-point hemostasis scale assessing the efficacy of EB in controlling bleeding.
Following the 6-month interventional on-demand phase, participants may choose to continue in an interventional prophylaxis arm.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eptacog beta | Experimental | Drug: EPTACOG BETAFor the initial 3 months, participants will be in a non-interventional phase and receive standard-of-care on-demand therapy for acute bleeding at their hematologist's discretion. Following this non-interventional phase, they will use eptacog beta on-demand to treat acute bleeding for 6 months. Throughout the study, they will log bleeding events and management in a diary. Hemostatic efficacy after treatment will be assessed using a 4-point hemostasis efficacy scale. Participants will have the option to re-consent for an exploratory phase, adding 6 months to their time on the study (total of 15 months). For those entering the optional extension phase, eptacog beta will be routinely administered for bleed prophylaxis. The treating hematologist will determine the best method/location for infusions. The number of bleeding events over time will be compared between the prophylaxis phase and the on-demand phase. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Adult or Pediatric persons with inherited Glanzmann thrombasthenia (see diagnostic criteria below)
- Severe bleeding phenotype
- Adequate hepatic function
- Adequate renal function
- Adults subject (≥18 years of age) or caregiver (parent or legally authorized representative) for minor subjects, subjects with cognitive impairment, or subjects with impaired decision-making capacity have provided written informed consent, and the participant has given consent/assent (if applicable)
Key exclusion criteria (11 listed)
- Thrombocytopenia (platelet count < 100k)
- Acquired Glanzmann thrombasthenia secondary to autoimmune disease, malignancy, or medication
- Inherited or acquired bleeding diathesis other than Glanzmann thrombasthenia
- Have a history of venous or arterial thrombotic event within 2 years of study enrollment
- Active malignancy
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 |
|---|---|---|
| EPTACOG BETAAlso: SEVENTFACT®; EBEptacog beta (SEVENFACT®; EB) is a lyophilized powder in single-use vials (1, 2, or 5 mg) of coagulation factor VIIa (recombinant)-jncw. It is reconstituted with sterile water (provided in the kit) and administered intravenously. SEVENFACT® may be administered by a healthcare provider (HCP) at the study site or home, or by a trained participant/caregiver. Participants will be monitored in a healthcare facility for 60 minutes after the first dose for hypersensitivity. | Drug | Eptacog beta |
Primary outcome
Proportion of bleeding events successfully treated within 24 hours of the first Eptacog Beta (EB) administration
Time frame: 24 hours after the first EB dose administration
Proportion of acute bleeding events successfully treated with EB within 24 hours of the first EB administration based on an "excellent or good" response on a 4-point hemostatic efficacy scale and without rebleeding before 36 hours. Hemostatic Efficacy Scale: None = No effect; bleeding unchanged or worsened; continued treatment needed. Moderate = Some effect (e.g., pain decreased, bleeding signs improved) but bleeding continued; further treatment needed. Good = Bleeding symptoms are largely reduced but not completely resolved; no further EB infusions are needed. Excellent = Full relief of bleeding symptoms; no additional EB infusion required.
Number of bleeding events over the study period
Time frame: 6 months following the start of the interventional prophylaxis phase
The number of bleeding events occurring while receiving routine EB prophylaxis will be compared with the number of bleeding events that occurred during on-demand therapy. *This outcome applies only to the optional interventional prophylaxis phase.
15 secondary outcomes
Number of treatment-Emergent Adverse Events and Clinically Significant Laboratory Abnormalities
Time frame: 6 months following the start of EB administration for each study phase
EuroQol five-dimensional questionnaire for youth (EQ-5D-Y) score for participants 4-15 years of age.
Time frame: Baseline, 9 and 15 months after baseline
EuroQol five-dimensional 5 Level (EQ-5D-5L) score for participants ≥16 years
Time frame: Baseline, 9 and 15 months after baseline
Patient Reported Outcomes Measurement Information System (PROMIS) fatigue short form for adults
Time frame: Baseline, 9 and 15 months after baseline
PROMIS fatigue short form (pediatric version)
Time frame: Baseline, 9 and 15 months after baseline
Menstrual impact questionnaire (MIQ) score
Time frame: Baseline, 9 and 15 months after baseline
Exogenous thrombin potential (ETP) following ex vivo EB
Time frame: Baseline (pre-infusion)
Lag time and time to peak thrombin following ex vivo EB
Time frame: Baseline (pre-infusion)
Peak thrombin levels following ex vivo EB
Time frame: Baseline (pre-infusion)
ETP levels following in vivo EB
Time frame: Preinfusion, 30 and 60 minutes following the first EB infusion
Lag time and time to peak thrombin following in vivo EB infusion
Time frame: Preinfusion, 30 and 60 minutes following the first EB infusion
Peak thrombin following in vivo EB infusion
Time frame: Preinfusion, 30 and 60 minutes following the first EB infusion
Clotting time (CT) and clotting formation time (CFT) following in vivo EB infusion
Time frame: Preinfusion, 30 and 60 minutes following the first EB infusion
Maximum clot firmness (MCF) following in vivo EB infusion
Time frame: Preinfusion, 30 and 60 minutes following the first EB infusion
Alpha angle following ex vivo EB
Time frame: Preinfusion, 30 and 60 minutes following the first EB infusion
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 1 Dec 2025.
- Individual participant dataSharing planned: No
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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.
Karen Zimowski, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EPTACOG BETA in people with Glanzmann Thrombasthenia, run by Emory University 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 6 inclusion and 11 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: looking for participants now, as of the sponsor's last update on 1 Dec 2025. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Karen Zimowski, MD (404-785-1200, kzimows@emory.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of bleeding events successfully treated within 24 hours of the first Eptacog Beta (EB) administration (time frame: 24 hours after the first EB dose administration). The study lists 2 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 1 Dec 2025.