Mucopolysaccharidosis VII Disease Monitoring Program
Recruiting · NCT03604835
Conditions: Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
At a glance
- Research payments reported, 2025
- $79,380
- U.S. investigators named
- 6
- Sites paid
- 6
- Planned or actual enrollment
- 50
About the trial
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Tested
- No Intervention (other)
- Dates
- Started 2018-01-29; completion 2032-04
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2026-08-27. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ultragenyx Pharmaceutical Inc. reported $79,380 in research payments for this study in 2025 (12 payments).
Products named: Mepsevii
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Monica Penon Portmann, MD Pediatrics · San Francisco, CA
- Raymond Y. Wang, MD Pediatrics · Orange, CA
- Christina L. Grant, M.D., PH.D. Pediatrics · Washington, DC
- Erika R. Vucko, APN Nurse Practitioners · Chicago, IL
- Elizabeth G. Ames, MD, PHD Medical Genetics · Ann Arbor, MI
- Heather A. Lau, M.D. Child Neurology · New York, NY
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- NYU Grossman School of Medicine $20,579
- University of Michigan Health System, MI $19,453
- Children's Hospital of Orange County, CA $17,112
- Ann & Robert H Lurie Childrens Hospital of Chicago, IL $16,177
- Childrens Research Institute $3,258
- Seattle Children's Hospital, WA $2,801
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.