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Monitoring Program for Mucopolysaccharidosis VII (Sly Syndrome)

This is an observational study, called a Disease Monitoring Program, for people with Mucopolysaccharidosis VII (MPS VII), also known as Sly Syndrome. The study aims to understand how MPS VII progresses over time and to evaluate the long-term effectiveness and safety of vestronidase alfa, including any allergic reactions or immune responses. You can join if you have a laboratory diagnosis of MPS VII and are willing to provide consent. Both patients receiving vestronidase alfa and those not receiving treatment will be included. The study will track the clinical course of MPS VII and the effects of vestronidase alfa for 10 years. The current status of this study is unclear, and it plans to enroll 50 participants.

Study design
This is a global, prospective, multicenter, longitudinal observational study designed to collect data from 50 participants with MPS VII. It is not a randomized study and includes both treated and untreated patients.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will monitor the clinical course, effectiveness, and safety of vestronidase alfa for 10 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03604835

Mucopolysaccharidosis VII Disease Monitoring Program

Recruiting
Not specifiedAll AgesObservational
Ultragenyx Pharmaceutical Inc
~50 participants
Updated 2026-08-27 on ClinicalTrials.gov
What's tested:No Intervention

At a glance

Recruiting sites
12 of 14 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Clinical Course of MPS VII Disease
Measured over 10 years
+2 more outcomes measured

Conditions

Mucopolysaccharidosis VII
MPS VII
MPS 7
Sly Syndrome

Explore related trials

Sly Syndrome

Where it's being run

14 sites across 14 states
California1
District of Columbia1
Illinois1
Michigan1
New York1
Utah1
Washington1
Buenos Aires1

Study leadership

  • Medical Director · STUDY_DIRECTOR · Ultragenyx Pharmaceuticals Inc.

Who to contact

Patients Contact: Trial Recruitment
[email protected]
1-888-756-8657
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Eligibility criteria

Inclusion

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

Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.

What this trial measures

  • Clinical Course of MPS VII Disease10 years

    To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa

  • Long-term Effectiveness of Vestronidase Alfa10 years

    To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa

  • Long-term Safety of Vestronidase Alfa10 years

    Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.