Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835 · tracked via the Priya Life Science USA tracker

Sponsor

Ultragenyx Pharmaceutical Inc

Phase

Observational

Started

2018-01-29

Last updated

2026-08-27

Condition(s) studied

Mucopolysaccharidosis VIIMPS VIIMPS 7Sly Syndrome

Investigational drug(s) / intervention(s)

No Intervention

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.

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.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Clinical Course of MPS VII Disease — 10 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 Alfa — 10 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 Alfa — 10 years
Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.

Trial sites (14)

Facility	City	Region	Status
Children's Hospital of Orange County	Orange	California	Recruiting
Children's National Health System	Washington D.C.	District of Columbia	Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	Recruiting
University of Michigan	Ann Arbor	Michigan	Recruiting
New York University Langone Medical Center	New York	New York	Terminated
University of Utah Medical Center	Salt Lake City	Utah	Recruiting
Seattle Children's Hospital	Seattle	Washington	Recruiting
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.	Buenos Aires	Buenos Aires	Recruiting
Hospital de Clínicas de Porto Alegre	Porto Alegre	Rio Grande do Sul	Recruiting
Centre Hospitalier Universitaire La Timone	Marseille	Provence-Alpes-Côte d'Azur Region	Recruiting
Universitätsmedizin der Johannes Gutenberg-Universität Mainz	Mainz	Germany	Recruiting
Erasmus University Medical Center Rotterdam	Rotterdam	South Holland	Terminated
Centro Hospitalar do Porto	Porto	Portugal	Recruiting
Hospital Universitario Virgen del Rocío Pabellón Infantil	Seville	Sevilla	Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03604835 on ClinicalTrials.gov ↗ ← All trials in the USA