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Study identifier: NCT03604835 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

Condition: Mucopolysaccharidosis VII · MPS VII · MPS 7  ·  Sponsor: Ultragenyx Pharmaceutical Inc

PhaseN/A
Planned participants50
Who can joinAll sexes, N/A to no upper limit
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Patients Contact: Trial Recruitment  ·  1-888-756-8657  ·  trialrecruitment@ultragenyx.com

HCPs Contact: Medical Information  ·  1-888-756-8657  ·  medinfo@ultragenyx.com

Always discuss trial participation with your own doctor first.

Locations (14)

Children's Hospital of Orange CountyOrange, California, United StatesRecruiting
Children's National Health SystemWashington D.C., District of Columbia, United StatesRecruiting
Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois, United StatesRecruiting
University of MichiganAnn Arbor, Michigan, United StatesRecruiting
New York University Langone Medical CenterNew York, New York, United StatesTerminated
University of Utah Medical CenterSalt Lake City, Utah, United StatesRecruiting
Seattle Children's HospitalSeattle, Washington, United StatesRecruiting
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.Buenos Aires, ArgentinaRecruiting
Hospital de Clínicas de Porto AlegrePorto Alegre, Rio Grande do Sul, BrazilRecruiting
Centre Hospitalier Universitaire La TimoneMarseille, Provence-Alpes-Côte d'Azur Region, FranceRecruiting
Universitätsmedizin der Johannes Gutenberg-Universität MainzMainz, GermanyRecruiting
Erasmus University Medical Center RotterdamRotterdam, South Holland, NetherlandsTerminated

+ 2 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT03604835