Monitoring program for Mucopolysaccharidosis VII disease
Official title: Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Run by Ultragenyx Pharmaceutical Inc · NCT03604835 · Data updated 2026-11-12
This study is trying to understand how Mucopolysaccharidosis VII affects people over time and see if a treatment called vestronidase alfa is safe and effective for both treated and untreated patients.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 50 (estimated) |
| Sponsor | Ultragenyx Pharmaceutical Inc Industry-sponsored |
| Locations | 14 sites (Orange, California and 13 other locations) |
| Status | Recruiting |
| Started | January 29, 2018 |
| Main results expected | April 2032 |
| Study ends | April 2032 |
| Registry record updated | August 27, 2026 |
| Trial ID | NCT03604835 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include individuals diagnosed with MPS VII through laboratory testing.
✕Probably not a fit
Patients currently participating in other interventional clinical trials may not benefit from this program.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
The Mucopolysaccharidosis VII Disease Monitoring Program is a global, prospective, multicenter initiative aimed at characterizing the presentation and progression of MPS VII. It assesses the long-term effectiveness and safety of vestronidase alfa, including monitoring for hypersensitivity reactions and immunogenicity. The program collects comprehensive data on clinical presentation, disease heterogeneity, and progression through various biomarker assessments and patient-reported outcomes. This observational study includes both treated and untreated patients to provide a robust dataset for future analysis.
Why it matters
Potential benefit: If successful, this program could enhance understanding of MPS VII and improve management strategies for patients.
How similar studies have gone: While this program is observational, similar approaches in monitoring rare diseases have shown promise in enhancing patient care and treatment outcomes.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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. Exclusion Criteria: * Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Where it's running
-
Children's Hospital of Orange County
Orange, California, United States Recruiting -
Children's National Health System
Washington D.C., District of Columbia, United States Recruiting -
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States Recruiting -
University of Michigan
Ann Arbor, Michigan, United States Recruiting -
University of Utah Medical Center
Salt Lake City, Utah, United States Recruiting -
Seattle Children's Hospital
Seattle, Washington, United States Recruiting
Show 8 more locations
-
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
Buenos Aires, Buenos Aires, Argentina Recruiting -
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil Recruiting -
Centre Hospitalier Universitaire La Timone
Marseille, Provence-Alpes-Côte d'Azur Region, France Recruiting -
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, Germany Recruiting -
Centro Hospitalar do Porto
Porto, Portugal Recruiting -
Hospital Universitario Virgen del Rocío Pabellón Infantil
Seville, Sevilla, Spain Recruiting -
New York University Langone Medical Center
New York, New York, United States Terminated -
Erasmus University Medical Center Rotterdam
Rotterdam, South Holland, Netherlands Terminated
Study contacts
- Study coordinator: Patients Contact: Trial Recruitment
- Email: trialrecruitment@ultragenyx.com
- Phone: 1-888-756-8657
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Observational study on Mucopolysaccharidosis diseases in France
This study is trying to learn more about Mucopolysaccharidosis diseases by collecting information from patients in France to understand how these rare conditions affect their lives.
-
Registry for patients with lysosomal storage diseases
This study is collecting information from patients with lysosomal storage diseases to see how these conditions progress and how treatments can improve care for them and their families.
-
In utero enzyme replacement therapy for lysosomal storage disorders
This study is testing whether giving enzyme replacement therapy to unborn babies with lysosomal storage disorders can improve their health and development before they are born.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.