Clinical Hold for Rocket Pharmaceuticals’ Gene Therapy
Patient fatality reported in clinical trial for investigational gene therapy for Danon disease
Stephanie Vine | | 2 min read | News
The FDA has placed on hold a phase II trial of Rocket Pharmaceuticals’ RP-A501, an investigational gene therapy for Danon disease, following a patient fatality. The individual experienced a serious adverse event involving capillary leak syndrome, later complicated by an acute systemic infection that resulted in death.
Rocket is conducting a full root cause analysis, with a particular focus on a recently introduced immunosuppressive agent added to the pre-treatment protocol. The company says it is working with the FDA, an Independent Data Safety Monitoring Committee, trial investigators, and external experts to evaluate the situation and determine next steps.
The global, single-arm, multi-center phase II study was assessing the safety and efficacy of RP-A501 in 12 patients. Primary endpoints included LAMP2 protein expression and changes in left ventricular mass, with secondary outcomes measuring biomarkers and event-free survival over 24 months.
“We are heartbroken by this loss and are fully committed to our mission to develop gene therapies that address the underlying cause of devastating diseases like Danon. We are immensely grateful for the patients and families who participate in this important research,” said Gaurav Shah, CEO of Rocket Pharmaceuticals, in a statement.
Danon disease is a rare, X-linked genetic disorder caused by mutations in the LAMP2 gene, leading to the accumulation of cellular waste and resulting in a range of clinical symptoms. The condition primarily affects the heart, skeletal muscles, and, in some cases, the central nervous system. The most prominent and severe manifestation is cardiomyopathy, especially in males, who often present in childhood or adolescence with hypertrophic cardiomyopathy that can progress to heart failure or sudden cardiac death.
There is currently no cure. Disease management focuses on supportive care, including cardiac medications, the use of implantable cardioverter-defibrillators, and, in severe cases, heart transplantation.
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