Clinical trial 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
Official title: Multi-Centre, Non-Interventional, Double Cohort Study to Assess the Safety of Myozyme® and of Aldurazyme® in Real-World Home Infusion Setting
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pompe Disease
Mucopolysaccharidosis Type I (MPS I)
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2021-10-11
Enrollment: 57
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pompe disease patients with confirmed 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).

Brief 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)

Eligibility criteria: 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.

Primary outcomes:
measure: Number of participants with treatment-emergent adverse events (TEAEs)
timeFrame: For at least 12 months starting from enrollment (day 0)
measure: 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)
measure: Number of participants with serious treatment-emergent adverse events (TEAEs)
timeFrame: For at least 12 months starting from enrollment (day 0)
measure: 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)
measure: Number of participants with infusion associated reactions (IARs)
timeFrame: For at least 12 months starting from enrollment (day 0)
measure: Number of participants with concomitant medications for each Anatomical Therapeutic Chemical (ATC) classification systems
timeFrame: At enrollment (day 0)
measure: Number of participants with change in the use of concomitant medications in case of non-tolerated infusion
timeFrame: For at least 12 months starting from enrollment (day 0)
Secondary outcomes:
measure: Patient satisfaction
timeFrame: For at least 12 months starting from enrollment (day 0)
measure: Patient compliance
timeFrame: For at least 12 months starting from enrollment (day 0)
Other outcomes:
Locations:
city: Italy
country: Italy
facility: Investigational Site Italy
Study officials:
affiliation: Sanofi
name: Clinical Sciences & Operations
role: STUDY_DIRECTOR
References: