NCT05073783

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

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 57 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed description

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

Conditions

• Pompe Disease
• Mucopolysaccharidosis Type I (MPS I)

Timeline

• Start date: 2021-10-14
• Primary completion: 2024-01-31
• Completion: 2024-01-31
• First posted: 2021-10-11
• Last updated: 2024-02-14

Locations

1 site across 1 country: Italy