The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT). The study will also look at whether TX002100 reduces the need for blood or iron treatments, and whether it improves blood counts, such as hemoglobin levels.
Adults with HHT who need regular intravenous iron or blood transfusions.
Females of childbearing potential and male participants must agree to comply with the contraception requirements.
Able to understand and is willing and able to comply with the study requirements and study procedures and agrees to participate in the study by giving written informed consent.
History of anemia of any cause other than HHT-related bleeding, or any other clinically significant inherited bleeding disorder aside from HHT.
Has undergone or is anticipated to undergo liver transplantation.
Medical conditions or comorbidities that, in the opinion of the investigator, would interfere with study compliance or data interpretation; or, in the opinion of the Investigator, taking part in the study would jeopardize the safety of the patient.
History of hemorrhage from untreated pulmonary arteriovenous malformations (AVMs) manifesting as hemoptysis or hemothorax within 12 weeks before screening.
Single group
TX002100
Biological/VaccineSubcutaneous injection
Incidence of adverse events (AEs), including serious adverse events (SAEs)
Incidence of clinically significant changes from baseline in safety laboratory assessments and other safety assessments
To assess hematologic support score change from baseline calculated over the 12 weeks preceding the time point
To assess hematologic impact score
To assess hemoglobin change from baseline
Change from baseline in the following calculated over the 12 weeks preceding the time point: - Total parenteral iron (mg) infused - Number of pRBCs transfused
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This trial is not recruiting at the moment. You can still explore other options:
