Primary Objective: To determine efficacy of pomalidomide compared to placebo for the reduction in severity of epistaxis after 24 weeks of treatment.
Secondary Objectives: To determine the safety and tolerability of pomalidomide for the treatment of HHT; to determine if pomalidomide treatment improves quality of life in HHT; to determine whether a continued response to pomalidomide is evident 12 weeks after treatment discontinuation; to develop a biorepository for future studies to define biomarkers predictive of pomalidomide response and allow investigations into the biology of HHT and mechanisms of pomalidomide.
Primary endpoint: Change from baseline to 24 weeks in the Epistaxis Severity Score.
Secondary endpoints: Total parenteral iron infused and packed red blood cell units transfused during the 24 week treatment period, change from baseline to 24 weeks in total weekly duration of epistaxis, change in quality of life (QOL) scores assessed using NIH PROMIS Satisfaction with Social Roles and Activities, Emotional Distress-Depression and Fatigue instruments, and HHT-specific QOL questions, proportion of patients requiring no blood transfusions or iron infusions during the 24 week treatment period, change from baseline in the Epistaxis Severity Score averaged across weeks 16-24 and at each study visit, proportion of patients requiring endoscopic interventions for bleeding, safety assessed by adverse events and side effects.
PATH is registered with clinicaltrials.gov. More information on the study design and status can be found here: https://www.clinicaltrials.gov/ct2/show/NCT03910244