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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1266-7312 | Registry Identifier | ICTRP |
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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:
Prospective observation duration for each patient: at least 12 months (from enrollment)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Patient satisfaction | Patient satisfaction data will be collected through a satisfaction questionnaire, including potential benefits in terms of stress, time and costs. The questionnaire aims to evaluate patient satisfaction about home infusion: 1) where do you prefer to receive ERT, home or hospital; 2) why (list of reasons); 3) how do you feel now; 4) if you are in home infusion, how do you rate your health than when you were treated in hospital. |
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Inclusion Criteria:
Exclusion Criteria:
The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
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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).
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational Site Italy | Italy | Italy |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41272769 | Derived | Toscano A, Musumeci O, Sacchini M, Ravaglia S, Siciliano G, Fiumara A, Verrecchia E, Fischetto R, Crescimanno G, Taurisano R, Sechi A, Gasperini S, Cianci V, Maggi L, Brighina F, Barone R, Cianflone A, Balzarini M, Parini R, Scarpa M. Long-term safety outcomes and patient preferences for home-based intravenous enzyme replacement therapy (ERT) in Pompe disease and Mucopolysaccharidosis Type I (MPS-I): final results of two-year observation. Orphanet J Rare Dis. 2025 Nov 21;20(1):639. doi: 10.1186/s13023-025-04108-1. |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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| 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) |
| For at least 12 months starting from enrollment (day 0) |
| Patient compliance | Patient compliance is assessed as number of missed infusions versus planned and/or return to hospital setting (with reasons). | For at least 12 months starting from enrollment (day 0) |
| ID | Term |
|---|---|
| D006009 | Glycogen Storage Disease Type II |
| D008059 | Mucopolysaccharidosis I |
| ID | Term |
|---|---|
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D009083 | Mucopolysaccharidoses |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
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