Actively Recruiting
Mucopolysaccharidosis VII Disease Monitoring Program
Led by Ultragenyx Pharmaceutical Inc · Updated on 2026-04-15
50
Participants Needed
14
Research Sites
795 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Mucopolysaccharidosis VII Disease Monitoring Program
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.
- Willing and able to provide written informed consent or, for patients under 18 or those over 18 with cognitive deficiencies, provide written assent (if required) and consent by a legally authorized representative.
- Willing to comply with the Disease Monitoring Program visit schedule.
You will not qualify if you...
- Concurrent participation in other pharmaceutical company-sponsored interventional clinical trials unless approved by Ultragenyx.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
2
Children's National Health System
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
4
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
5
New York University Langone Medical Center
New York, New York, United States, 10016
Terminated
6
University of Utah Medical Center
Salt Lake City, Utah, United States, 84112
Actively Recruiting
7
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
8
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
Buenos Aires, Argentina, C1425FNG
Actively Recruiting
9
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil, 90035-003
Actively Recruiting
10
Centre Hospitalier Universitaire La Timone
Marseille, Provence-Alpes-Côte d'Azur Region, France, 13005
Actively Recruiting
11
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, Germany, 55131
Actively Recruiting
12
Erasmus University Medical Center Rotterdam
Rotterdam, South Holland, Netherlands, 3015 CN
Terminated
13
Centro Hospitalar do Porto
Porto, Portugal, 4050-651
Actively Recruiting
14
Hospital Universitario Virgen del Rocío Pabellón Infantil
Seville, Spain, 41013
Actively Recruiting
Research Team
P
Patients Contact: Trial Recruitment
CONTACT
H
HCPs Contact: Medical Information
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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