Overview

Rossella: A Study to Evaluate the Safety, PK, Efficacy, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Status:
Recruiting

Trial end date:
2025-02-01

Target enrollment:

Participant gender:

Summary

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with classic IOPD.

Phase:

Phase 3

Details

Lead Sponsor:

Amicus Therapeutics

Treatments:

Miglustat