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McCrae, K., Thomas, S., Kuter, D., Pyeritz, R., Kasthuri, R., Merlo, C., Battaile, J., Whitehead, K., Iyer, NN., Conrad, M., Decker, J., Clancy, M., & Catellier, D. (2019). Pomalidomide in HHT: June 12-16, 2019, San Juan, Puerto Rico. Angiogenesis, 22(4), 590-591. https://doi.org/10.1007/s10456-019-09686-w
Oral Communications SESSION 2: ENG/ALK1 SIGNALING Poster Presentations: Drug Treatments of HHT
Objective: Based on the results of a pilot study, we have developed a randomized, placebo-controlled study to further examine the efficacy and safety of pomalidomide in HHT. This abstract describes the development and primary features of the study. Methods: With the support of a U34 award from NHLBI, a group of experienced HHT investigators was convened and met biweekly to develop this trial. Assistance in trial design was provided by Research Triangle Institute (RTI). Participating sites were selected with the guidance of CureHHT.
Results: One hundred fifty-nine study subjects will be randomized 2:1 to six four-week cycles of pomalidomide or placebo. The primary endpoint will be the difference in the mean change of the Epistaxis Severity Score (ESS) from baseline to week 24 between placebo- and pomalidomidetreated groups. Eligible patients ill have a clinical diagnosis of HHT, a requirement for parenteral infusion of at least 250 mg of iron or transfusion of 1 unit of blood over the preceding 24 weeks, and an ESS of C 3 measured over the preceding 3 months. All patients will undergo genotyping, the cost of which will be paid by the study in the absence of third-party coverage. Samples for exploratory mechanistic analyses and to define biomarkers of response will be obtained before and during treatment.
Conclusion: The study has been approved by NHLBI and milestones developed. Efforts are underway to finalize the protocol and obtain the IND. We expect this study to begin accrual in August, 2019.
