A New Jersey biotech company has paused a gene therapy trial after a participant had an unexpected adverse event and later died from a sudden, severe infection.
Rocket Pharmaceuticals said the patient experienced “clinical complications related to a capillary leak syndrome,” a rare disorder in which fluids escape blood vessels and enter surrounding tissue. The company voluntarily paused further dosing in the 12-person trial before the FDA placed a clinical hold on May 23.
Since then, the patient died after an acute systemic infection, according to a May 27 news release from the company.
The phase 2 trial is investigating Rocket’s gene therapy candidate for Danon disease, a rare inherited disorder that causes heart failure and is estimated to affect 15,000 to 30,000 people in the U.S. and Europe. The disease leads to “frequent death during adolescence and early adulthood” in male patients, according to the company.
Rocket Pharmaceuticals said it is conducting a root cause analysis with a focus on “the recent introduction of a novel immune suppression agent to the pre-treatment regimen.”
