NHLBI Lifts Administrative Hold on SCD Gene Therapy Trial, Resumes Enrollment

January 05, 2022

The National Heart, Lung and Blood Institute (NHLBI) resumed enrolling patients in an NHLBI-funded gene therapy trial taking place at Boston Children’s Hospital (BCH) after lifting an administrative hold on Dec. 1, 2021. Earlier in February 2021, the institute placed a hold on the Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease out of an abundance of caution. This action came after to investigate newly reported cancers in two patients who had received their investigational gene therapy for sickle cell disease (SCD) that used a different, yet related, lentiviral vector.

During the administrative hold, NHLBI performed a comprehensive assessment of available safety information, including convening and soliciting input from multidisciplinary scientific and clinical perspectives, as well as patient perspectives. According to the agency, the insights gained through these deliberations enabled the investigators to supplement their existing risk mitigation plans to further minimize patient risk. Following a review of this panel’s report and other data, the NHLBI Gene and Cell Therapy Data and Safety Monitoring Board (DSMB) recommended lifting the administrative hold.