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NIH-supported clinical trial shows cancer drug has ability to treat hereditary hemorrhagic telangiectasia
RESEARCH TRIANGLE PARK, N.C. – Researchers at RTI International, a nonprofit research institute, contributed significantly to an NIH-supported clinical trial that has found sufficient evidence that the cancer drug pomalidomide is safe and effective in treating hereditary hemorrhagic telangiectasia (HHT).
The trial, which took place at 11 medical centers across the U.S during 2019-2023, was stopped early because of the positive findings, now published in the New England Journal of Medicine.
“The trial data showed patients with HHT who took the oral medication experienced a reduction in nosebleeds, needed fewer blood transfusions or iron infusions and showed improved quality of life,” said Sonia Thomas, DrPH, the study’s lead statistician at RTI and a lead author.
HHT, also known as Osler-Weber-Rendu syndrome, is a rare genetic disorder that impacts one in 5,000 people worldwide. It is characterized by abnormal blood vessels that cause excessive nosebleeds or bleeding along the gastrointestinal tract which can result in anemia and reduced quality of life. In severe cases, bleeding can be life-threatening.
“Finding a therapeutic agent that works in a rare disorder is highly uncommon, so this is a real success story,” said Andrei Kindzelski, M.D., Ph.D., program officer in the division of blood diseases and resources at the National Heart, Lung, and Blood Institute, part of NIH.
RTI worked in partnership with researchers at the Cleveland Clinic in the development and implementation of the trial. As the Data Coordinating Center, RTI experts provided biostatistical leadership, data management and logistical/operational oversight for the study from protocol design to trial implementation, clinical site monitoring and data analysis.
Thomas added that since the study took place during the COVID-19 pandemic, accommodations were made to allow for virtual participant study visits, off-site lab testing and direct drug shipments to participants’ home. “With these changes, the study continued successfully during the pandemic and accommodated participants who lived farther away from their clinic,” said Thomas.
This study was supported by NHLBI grants 1U24HL140090-01A1 and 1UG3HL140097-01A1.
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