Actively Recruiting
Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Led by Ultragenyx Pharmaceutical Inc · Updated on 2026-04-15
50
Participants Needed
14
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying Mucopolysaccharidosis VII (MPS VII), also known as Sly Syndrome, to understand how the disease presents and progresses over time. This global, prospective, multicenter, and longitudinal program aims to assess the long-term effectiveness and safety of the treatment vestronidase alfa, including monitoring for hypersensitivity reactions and immune responses. Both treated and untreated patients with MPS VII are included to gather a comprehensive understanding of the disease. The program includes patients receiving vestronidase alfa either through prescription or early access/compassionate use, as well as patients who are untreated or receiving treatments other than vestronidase alfa. This is an observational study without randomization, designed to collect standardized high-quality data from multiple global sites. Treatments are not assigned by the study but observed as part of routine care or compassionate use. Participants will be monitored over a period of 10 years to track the clinical course of MPS VII, the long-term effectiveness of vestronidase alfa, and its safety profile. Data collection includes biomarker analysis, clinical assessments, and patient or caregiver-reported outcomes. The study aims to provide detailed insights into disease heterogeneity and progression by gathering consistent and meaningful data during routine clinical visits.
CONDITIONS
Brief 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, if under 18 or cognitively impaired, provide written assent 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 trial unless approved by Ultragenyx
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 10 years
Participants with MPS VII are observed over time to characterize disease presentation, progression, and long-term effects of treatments or lack thereof.
Regular visits at multiple global sites as per disease monitoring program schedule
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
H
HCPs Contact: Medical Information
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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