Rocket Pharmaceuticals has started dosing patients in a Phase I clinical trial of its adeno-associated viral vector (AAV)-based gene therapy, RP-A501, to treat Danon disease.
University of California San Diego (UCSD) Health is serving as the initial and lead centre for the non-randomised, open-label Phase I trial, which is expected to enrol 12-24 pediatric and young adult male patients.
The trial has been designed to evaluate the safety and tolerability of a single infusion of RP-A501.
Rocket Pharmaceuticals CEO and president Gaurav Shah said: “Danon disease is a rapidly progressive cardiomyopathy where we believe gene therapy can make a meaningful difference in patient outcomes. Current standards of care for Danon disease, including heart transplant, are not curative and are associated with considerable morbidity and mortality.
“As a result, median survival for male Danon disease patients has been reported at age 19, caused by progressive heart failure.
“This underscores the urgent need for new treatment options like RP-A501 gene therapy for the patients and families contending with this debilitating, fatal disease.”
Four patient cohorts will be separated by pediatric and adult age groups and two dose levels will be investigated.
A low dose level of 6.7x1013 genome copies/kg will be given to the first cohort, and the cohort will receive a higher dose upon completion.
Estimated to affect between 15,000 and 30,000 people in the US and EU, Danon disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2.
Cardiac transplantation is the only therapy currently available for Danon disease.