Despite encouraging results from a phase I trial of the RP-A501 gene therapy for cardiomyopathy in Danon disease, the phase II trial which had started with 12 participants has been put on hold.
- This follows the appearance of complications linked to capillary leak syndrome in one of the participants, resulting in his death from an acute systemic infection.
- Rocket Pharmaceuticals, the company developing the product, is working with the Food and Drug Administration (FDA) to better understand the causes of this complication.
Rocket Pharmaceuticals Provides Update on Phase 2 Clinical Trial of RP-A501 for Danon Disease. Rocket Pharmaceuticals presse release, 27 Mayi 2025.
