Mucopolysaccharidosis VII Disease Monitoring Program

Study Overview
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Brief Summary
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The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

Detailed Description
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The Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) is a global, prospective, multicenter, longitudinal protocol designed to characterize MPS VII disease presentation and progression, assess long-term effectiveness and safety of vestronidase alfa, including hypersensitivity reactions and immunogenicity , as well as prospectively investigate longitudinal change across biomarker(s), clinical assessments, and patient/ caregiver-reported outcome measures in a representative population. The aim of this DMP is to collect data on patients with MPS VII to provide a comprehensive dataset on the clinical presentation, heterogeneity, and disease progression, and meaningful standardized ICH GCP-quality data collected in-clinic across multiple sites globally. The DMP is not a randomized study and both treated and untreated patients will be enrolled.

Official Title
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Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Conditions
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MPS VII, Mucopolysaccharidosis VII, MPS 7, Sly Syndrome

Intervention / Treatment
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* Other: No Intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis. Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or below adult ages as defined by local laws and regulations) or patients >18 years of age who have cognitive deficiencies, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the DMP has been explained, and prior to any research-related procedures. Willing to comply with DMP visit schedule. Exclusion Criteria: Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients with MPS VII receiving vestronidase-alfa<br>via prescription, or early access/ compassionate use program | Other: No Intervention<br>* Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.<br>|
| Patients with MPS VII not receiving vestronidase-alfa<br>no treatment or treatment other than vestronidase alfa | Other: No Intervention<br>* Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Course of MPS VII Disease | To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa | 10 years | 
| Long-term Effectiveness of Vestronidase Alfa | To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa | 10 years | 
| Long-term Safety of Vestronidase Alfa | Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa. | 10 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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UX003, Mepsevii, vestronidase alfa-vjbk, vestronidase alfa, recombinant human beta-glucuronidase