Health Trials

MPS VII

3

Review clinical trials related to MPS VII. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

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.

Participants needed: 50
Trial details
Biological sex: AllType: ObservationalSponsor: Ultragenyx Pharmaceutical IncUpdated: Jun 11, 2026Locations: 14
Eligibility criteria

Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic o... [+2]

Concurrent participation in other pharmaceutical company-sponsored interventiona...

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Status: Available

Expanded Access to Mepsevii

Individual patient expanded access requests may be considered for patients who have no other treatment options

Trial details
Biological sex: AllType: Expanded AccessSponsor: Ultragenyx Pharmaceutical IncUpdated: Apr 7, 2026
Eligibility criteria

Not listed

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Status: Recruiting

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

Participants needed: 10
Trial details
Phase: Phase 1Age: 18-50Biological sex: FemaleType: InterventionalSponsor: University of California, San FranciscoUpdated: Mar 17, 2026Locations: 1
Eligibility criteria

Live male or female fetuses at 18 0/7 weeks to 34 6/7 weeks gestation [+4]

Fetuses with a concurrent severe structural anomaly [+10]

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