An observational study in Mucopolysaccharidosis VII, MPS VII and MPS 7, sponsored by Ultragenyx Pharmaceutical Inc. Recruiting at 14 sites in 8 countries. Per ClinicalTrials.gov, last updated 2026-08-27.
Sponsored by Ultragenyx Pharmaceutical Inc · Observational
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.
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.
Patients with a confirmed diagnosis of MPS VII, including patients who already received vestronidase alfa in an Ultragenyx clinical trial or early access/ compassionate use program, and patients not receiving vestronidase alfa.
Patients who were previously enrolled in an Ultragenyx-sponsored clinical trial may participate in the DMP if they have completed or discontinued from the clinical trial.
Exclusion Criteria:
via prescription, or early access/ compassionate use program
Other: No Intervention
no treatment or treatment other than vestronidase alfa
Other: No Intervention
Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.
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
Time frame: 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
Time frame: 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.
Time frame: 10 years
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Mucopolysaccharidosis VII
Ultragenyx Pharmaceutical Inc