Monitoring program for Mucopolysaccharidosis VII disease
Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
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 |
|---|---|
| Enrollment | 50 (estimated) |
| Sex | All |
| Sponsor | Ultragenyx Pharmaceutical Inc Industry-sponsored |
| Locations | 14 sites (Orange, California and 13 other locations) |
| Trial ID | NCT03604835 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include individuals diagnosed with MPS VII through laboratory testing.
Not a fit: Patients currently participating in other interventional clinical trials may not benefit from this program.
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 performed: While this program is observational, similar approaches in monitoring rare diseases have shown promise in enhancing patient care and treatment outcomes.
Eligibility criteria
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 this trial is running
Orange, California and 13 other locations
- 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)
- New York University Langone Medical Center — New York, New York, United States (Terminated)
- University of Utah Medical Center — Salt Lake City, Utah, United States (Recruiting)
- Seattle Children's Hospital — Seattle, Washington, United States (Recruiting)
- Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L. — 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)
- Erasmus University Medical Center Rotterdam — Rotterdam, South Holland, Netherlands (Terminated)
- Centro Hospitalar do Porto — Porto, Portugal (Recruiting)
- Hospital Universitario Virgen del Rocío Pabellón Infantil — Seville, Spain (Recruiting)
Study contacts
- Study coordinator: Patients Contact: Trial Recruitment
- Email: trialrecruitment@ultragenyx.com
- Phone: 1-888-756-8657
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.