Alagille Syndrome
Genetic and congenital
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Gene therapy trial halted after second death

Posted on July 5, 2020

Two patients in a clinical trial investigating the gene therapy candidate AT132 for X-linked myotubular myopathy (XLMTM) have died, according to Audentes Therapeutics.

On May 6, Audentes alerted patient groups that a patient receiving a high dose of AT132 has died from sepsis and 2 other patients who had received high doses had serious side effects. On June 23, another letter said 1 of the 2 patients had suffered from progressive liver dysfunction; that patient died from a bacterial infection and sepsis.

“Prior to both of these deaths, Audentes, in consultation with the DMC, had halted further dosing of patients currently enrolled in the clinical trial. Subsequently, following interactions with the FDA (US Food and Drug Administration), the study was put on formal clinical hold. Per FDA’s guidance, we continue to follow and actively monitor those patients who are enrolled in the trial,” said Audentes in the letter. 

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