Rocket Pharmaceuticals Announces Completion of Enrollment in Phase 2 Pivotal Trial of RP ...
Rocket Pharmaceuticals, Inc. announces full enrollment in global Phase 2 trial evaluating RP-A501 for Danon disease, aiming for regulatory filings in the U.S. and ex-U.S.
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Rocket Pharmaceuticals completes enrollment in global Phase 2 pivotal trial of RP-A501 for Danon disease, aiming to evaluate efficacy and safety, with co-primary endpoints of LAMP2 protein expression and left ventricular mass reduction.
Rocket Pharmaceuticals, Inc. announced full enrollment in its global Phase 2 trial evaluating RP-A501 for Danon disease, a rare genetic disorder. The trial aims to assess the efficacy and safety of RP-A501, a gene therapy designed to restore cardiac function by delivering functional LAMP2B genes to heart tissue.
Rocket Pharmaceuticals completed enrollment in a phase 2 trial (NCT06092034) evaluating RP-A501, a gene therapy for Danon disease, with 12 male patients receiving a dose of 6.7x10^13 GC/kg. The trial's primary end points include LAMP2 expression and left ventricular mass reduction, with secondary end points including troponin levels and natriuretic peptide levels. Rocket plans to seek regulatory filings for RP-A501 both inside and outside the US.
Rocket Pharmaceuticals, Inc. announces full enrollment in global Phase 2 trial evaluating RP-A501 for Danon disease, aiming for regulatory filings in the U.S. and ex-U.S.