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 (HomERT)

Sanofi

Status: Completed

Conditions: Pompe Disease; Mucopolysaccharidosis Type I (MPS I)

Study type: Observational

Funder types: Industry

Identifiers

NCT05073783

OBS17128

U1111-1266-7312 (Registry Identifier)

Details and patient eligibility

About

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.

Full description

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

Enrollment: 57 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

57 participants in 2 patient groups

Cohort A

Description:

Pompe disease patients receiving Myozyme® (alglucosidase alfa) in a home-care setting.

Cohort B

Description:

MPS I patients receiving Aldurazyme® (laronidase) in a home-care setting.