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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Arthur M. Blank Hospital | Children's Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30329, United States