Clinical Trials

The purpose of this study is to evaluate pomalidomide vs. placebo in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate-to-severe epistaxis who require parenteral iron infusions or blood transfusions. A total of 159 patients will be randomized 2:1 to treatment with oral pomalidomide or matching placebo for 24 weeks. Mean change from baseline to 24 weeks in the Epistaxis Severity Score (ESS) will be compared between treatment groups to determine pomalidomide efficacy.

The purpose of this study is to develop a repository of blood and tissue from patients with epistaxis and GI bleeding secondary to the conditions described above (HHT, GAVE, SBA and OGIB).

The purpose of this study is to assess the efficacy of engasertib 40 mg QD in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in subjects with moderate to severe HHT.

The study aims to identify predictors of brain outcomes in Hereditary Hemorrhagic Telangiectasia (HHT) patients. The study will use the modified Rankin Score (mRS). Furthermore to define a severe bleeding phenotype in HHT for clinical trial readiness. 

The purpose of this study is to develop a resource (bank) of biospecimens and data collected from individuals with a vascular tumor or malformation to facilitate discovery and development of novel biomarkers for diagnosis and treatment of disease, better elucidate etiologic factors relating to vascular anomalies, and novel targeted therapeutic strategies for vascular anomalies.

This study aims to develop a novel ultra-low dose chest CT technology for use in HHT patients and to determine the lowest possible radiation dose that is achievable without sacrificing the diagnostic quality.