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.
Ultragenyx Pharmaceutical Inc is the lead sponsor of 63 studies on the registry; 8 are open to participants now.
Of its 16 completed or terminated interventional studies of FDA-regulated products, 12 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Not enrolling at this site.
Not enrolling at this site.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Mucopolysaccharidosis VII
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