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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

NCT05073783 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 57

Last updated 2024-02-14

No results posted yet for this study

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.

Conditions

  • Pompe Disease
  • Mucopolysaccharidosis Type I (MPS I)

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-14
Primary Completion
2024-01-31
Completion
2024-01-31

Countries

  • Italy

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05073783 on ClinicalTrials.gov