NCT05073783Completed
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
Source: ClinicalTrials.gov · Last updated 14 Feb 2024
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 57 (actual)
- First submitted
- 16 Sept 2021
- Start date
- Oct 2021
- Primary completion date
- Jan 2024
- Lead sponsor
- Sanofi
- Diseases
- Pompe disease
- NCT number
- NCT05073783
What you commit to
- Study type: Observational
- Participants enrolled: 57
- From Oct 2021 to the primary completion date, Jan 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Participation in another clinical trial with any investigational agent within the 12 weeks preceding enrolment.
Other illness
- Any condition (e.g. medical concern) which, in the opinion of the Investigator, would make the participant unsuitable for the study.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Who the study is looking for
- Signed, informed consent obtained prior to being enrolled into the study and prior to starting any data collection. Consent of a legally authorized guardian is required for legally minor patients as defined by local regulation. If the patient is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from the patient, if applicable.
- Pompe disease patients with confirmed acid alpha-glucosidase (GAA) enzyme deficiency treated with Myozyme® in home infusion setting according to authorized clinical practice and the approved risk management plan document (Cohort A) or
- MPS I patients with confirmed deficiency of the lysosomal enzyme, alpha-L-iduronidase treated with Aldurazyme® in home infusion setting according to authorized clinical practice and the approved risk management plan document (Cohort B).
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Investigational Site Italy Coordinator of this site? Confirm its status | Italy | Italy | Unknown status |
Related publications
This page is information, not medical advice. Discuss any trial with your doctor.