Danon Disease
Conditions
Brief summary
Co-Primary endpoint including myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI). Evaluation of efficacy associated with RP-A501.
Detailed description
LAMP2 protein expression, LVMI, hsTnI, NT-proBNP, KCCQ, and NYHA class. Evaluation of efficacy associated with RP-A501., Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization. Evaluation of efficacy associated with RP-A501., Incidence, severity and duration of treatment emergent safety events. Evaluation of safety.
Interventions
DRUGEpysqli 300 mg concentrate for solution for infusion
DRUGRITUXIMAB
DRUGBEKEMV 300 mg concentrate for solution for infusion
DRUGSIROLIMUS
DRUGPARACETAMOL
DRUGMETHYLPREDNISOLONE SODIUM SUCCINATE
DRUGPREDNISONE
DRUGAAV9.LAMP2B
DRUGPEGCETACOPLAN
DRUGECULIZUMAB
Sponsors
Rocket Pharmaceuticals Inc.
Eligibility
Sex/Gender
Male
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Co-Primary endpoint including myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI). Evaluation of efficacy associated with RP-A501. | — |
Secondary
| Measure | Time frame |
|---|---|
| LAMP2 protein expression, LVMI, hsTnI, NT-proBNP, KCCQ, and NYHA class. Evaluation of efficacy associated with RP-A501., Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization. Evaluation of efficacy associated with RP-A501., Incidence, severity and duration of treatment emergent safety events. Evaluation of safety. | — |
Countries
Germany, Italy
Outcome results
None listed