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New hope for rare bleeding disorder: experimental drug aims to stop bleeding episodes
NCT ID NCT07136857
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests an investigational drug called eptacog beta (SEVENFACT®) in 6 people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. For the first 3 months, participants use their standard treatments for bleeding; then they switch to the study drug to treat serious bleeds. An optional extension phase tests the drug as a routine preventive infusion 2-3 times per week for 6 months. The goal is to see if the drug can safely and effectively control bleeding.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- eptacog beta (SEVENFACT®)
- What this could lead to
- If successful, this could provide a new option to control bleeding episodes in people with Glanzmann Thrombasthenia, potentially reducing the need for platelet transfusions.
- What could go wrong
- This is a very small early-phase trial with only 6 participants, so results may not apply to everyone. The drug is not yet approved for this condition, and side effects or lack of effectiveness are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 6 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2025
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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 Exclusion Criteria: * 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 * 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Arthur M. Blank Hospital | Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Tongue camera could spot bleeding danger in rare blood disorders
- Massive blood disorder registry aims to transform care
- New blood test could unmask hidden bleeding disorders
- New antibody aims to stop bleeding episodes in rare clotting disorder