Rare disease study tracks MPS VII over time

NCT ID NCT03604835

Recruiting now Knowledge-focused Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study monitors up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. It also checks the long-term safety and effectiveness of the drug vestronidase alfa. Participants may or may not be taking the drug, and the study involves regular check-ups and tests.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 7

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MPS 7 MPS VII MUCOPOLYSACCHARIDOSIS VII SLY SYNDROME

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Centre Hospitalier Universitaire La Timone
RECRUITING

Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France
Centro Hospitalar do Porto
RECRUITING

Porto, 4050-651, Portugal
Children's Hospital of Orange County
RECRUITING

Orange, California, 92868, United States
Children's National Health System
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Erasmus University Medical Center Rotterdam
TERMINATED

Rotterdam, South Holland, 3015 CN, Netherlands
Hospital Universitario Virgen del Rocío Pabellón Infantil
RECRUITING

Seville, 41013, Spain
Hospital de Clínicas de Porto Alegre
RECRUITING

Porto Alegre, Rio Grande do Sul, 90035-003, Brazil
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
RECRUITING

Buenos Aires, C1425FNG, Argentina
New York University Langone Medical Center
TERMINATED

New York, New York, 10016, United States
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Utah Medical Center
RECRUITING

Salt Lake City, Utah, 84112, United States
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
RECRUITING

Mainz, 55131, Germany

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