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Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2026-04-15

No results posted yet for this study

Summary

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.

Conditions

  • Mucopolysaccharidosis VII
  • MPS VII
  • MPS 7
  • Sly Syndrome

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceuticals Inc.

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-01-29
Primary Completion
2033-05-31
Completion
2033-05-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Brazil
  • France
  • Germany
  • Netherlands
  • Portugal
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03604835 on ClinicalTrials.gov