CompletedNot Applicable

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

Italy57 participantsStarted 2021-10-14

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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). Exclusion Criteria: * Participation in another clinical trial with any investigational agent within the 12 weeks preceding enrolment. * 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of participants with treatment-emergent adverse events (TEAEs)

Timeframe: For at least 12 months starting from enrollment (day 0)

Number of participants with treatment-emergent adverse events (TEAEs) for each class of severity

Timeframe: For at least 12 months starting from enrollment (day 0)

Number of participants with serious treatment-emergent adverse events (TEAEs)

Timeframe: For at least 12 months starting from enrollment (day 0)

Number of participants with treatment-emergent adverse events (TEAEs) related to alglucosidase or laronidase

Timeframe: For at least 12 months starting from enrollment (day 0)

Number of participants with infusion associated reactions (IARs)

Timeframe: For at least 12 months starting from enrollment (day 0)

Number of participants with concomitant medications for each Anatomical Therapeutic Chemical (ATC) classification systems

Timeframe: At enrollment (day 0)

Trial details

NCT IDNCT05073783

SponsorSanofi

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2024-01-31

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