Mucopolysaccharidosis VII Disease Monitoring Program

Recruiting now

Conditions studied: Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome

In brief

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.

Key facts

Study ID

NCT03604835

Run by

Ultragenyx Pharmaceutical Inc

People needed

50

Starts

2018-01-29

Expected to finish

2032-04-01

Last updated by the study team

2026-06-11

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

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

Where it is running

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

Full record on ClinicalTrials.gov

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