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A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting — HomERT  

Pompe Disease Clinical Trial

Official title:
Multi-Centre, Non-Interventional, Double Cohort Study to Assess the Safety of Myozyme® and of Aldurazyme® in Real-World Home Infusion Setting

Clinical Trial Summary

Primary objective: To obtain data pertaining to the safety and tolerability of alglucosidase alfa and laronidase treatments administered in a home-care infusion setting. Secondary objectives: - To evaluate personal satisfaction of both cohorts of patients treated in a home-care infusion setting. - To evaluate the infusion compliance in both cohorts of patients treated in a home-care infusion setting.

Clinical Trial Description

Prospective observation duration for each patient: at least 12 months (from enrollment) ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05073783
Study type Observational
Source Sanofi
Contact
Status Completed
Phase
Start date October 14, 2021
Completion date January 31, 2024
View Details
See also
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