Rocket Pharmaceuticals is set to resume participant enrolment in its Phase I clinical trial of RP-A501 to treat Danon disease, after the US Food and Drug Administration (FDA) lifted the trial’s clinical hold.

The FDA decision comes after the company addressed the agency’s requests to change the trial protocol and other supporting documents with updated subject selection and management guidelines.

Rocket Pharma is currently working to restart the programme soon and anticipates beginning dosing in the 6.7e13vg/kg low-dose paediatric patient cohort in the third quarter of this year.

Long-term results from the low-dose and 1.1e14vg/kg higher-dose young adult cohorts are expected in the fourth quarter.

Rocket Pharmaceuticals CEO Gaurav Shah said: “We are moving as quickly as possible to resume dosing and commence treatment this quarter.

“Additionally, given the activity observed among young adults in our low-dose cohort, in agreement with the FDA, we are now proceeding with the paediatric cohort.”

Danon disease is a rare inherited condition that develops due to mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2). The disorder can result in heart failure and even death.

RP-A501 is an experimental gene therapy being developed to treat Danon disease and the first gene therapy with the potential to treat monogenic heart failure.

The therapeutic contains a recombinant adeno-associated serotype 9 (AAV9) capsid with a functional form of the human LAMP2B transgene (AAV9.LAMP2B).

FDA placed RP-A501’s Danon disease programme on clinical hold in May 2021 to alter the trial protocol and other supporting documents.

As part of or during the hold, no new safety events associated with the gene therapy were reported in the low-dose or higher-dose young adult cohorts.

The non-randomised, open-label Phase I trial is intended to recruit paediatric and young adult male patients to evaluate the safety and tolerability of RP-A501’s single intravenous infusion.

Other outcome measures in the trial include cardiomyocyte and skeletal muscle transduction by gene expression and histologic correction using endomyocardial biopsy.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.