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 | Completed

• 2 other identifiers: OBS17128U1111-1266-7312
• Study Type: observational
• Target: 57
• Locations: 1 country
• Sites: 1

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.

Conditions

Pompe Disease; Mucopolysaccharidosis Type I (MPS I)

Outcome Measures

Primary Outcomes (7)

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

  Treatment emergent adverse events (TEAEs) are defined as any event which are not present prior to the initiation of the Enzyme replacement therapy (ERT) administration in homecare setting or any event already present that worsens in either intensity or frequency following initiation of ERT administration in home-care setting.

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

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

  TEAEs are defined as any event which are not present prior to the initiation of the ERT administration in homecare setting or any event already present that worsens in either intensity or frequency following initiation of ERT administration in home-care setting. An adverse event grading scale of mild, moderate and severe is used for grading of adverse event severity.

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

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

  A serious adverse event (SAE) is any untoward medical occurrence that at any dose: 1) results in death or 2) is life-threatening or 3) requires inpatient hospitalization or prolongation of existing hospitalization or 4) results in persistent or significant disability/incapacity or 5) is a congenital anomaly/birth defect or 6) is a medically important event.

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

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

  TEAEs are defined as any event which are not present prior to the initiation of the ERT administration in homecare setting or any event already present that worsens in either intensity or frequency following initiation of ERT administration in home-care setting. A TEAE is defined as treatment-related if it has a reasonable possibility that the event is related to alglucosidase or laronidase.

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

• Number of participants with infusion associated reactions (IARs)

  IARs are defined as AEs that occur during either the infusion or the observation period following the infusion which are deemed to be related or possibly related to Myozyme® and Aldurazyme®. At the discretion of the Investigator, AEs occurring after completion of the post-infusion observation period that are assessed as related may also be considered IARs.

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

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

  Participants will be asked about their use of concomitant medication at enrollment.

  At enrollment (day 0)

• Number of participants with change in the use of concomitant medications in case of non-tolerated infusion

  Participants will be asked about their perception regarding any additional medications or treatments or any changes in regimen or dosages compared to their baseline (day 0) state. Any change in the therapy (increased therapy, decrease therapy, no change in therapy) during the study will be reported.

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

Secondary Outcomes (2)

• Patient satisfaction

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

• Patient compliance

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

Study Arms (2)

Cohort A

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

Cohort B

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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).

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (1)

Investigational Site Italy

Italy, Italy

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II; Mucopolysaccharidosis I

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Mucopolysaccharidoses; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2021
• First Posted: October 11, 2021
• Study Start: October 14, 2021
• Primary Completion: January 31, 2024
• Study Completion: January 31, 2024
• Last Updated: February 14, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will share

Locations

IT (1)