Study To Assess Response to Eptacog Beta iN Patients With Glanzmann THromboasthenia (STRENGTH )
Emory University
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. 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.
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 an…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
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: * Thrombo…
Interventions
- DrugEPTACOG BETA
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.
Location
- Arthur M. Blank Hospital | Children's Healthcare of AtlantaAtlanta, Georgia