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Recruiting NCT03604835

Mucopolysaccharidosis VII Disease Monitoring Program

— Observational Mucopolysaccharidosis VII MPS VII MPS 7 Sly Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: No Intervention.
Who it may be relevant to
Registry conditions: Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Argentina, Brazil, France, Germany +3
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Overview

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.

Detailed description

The Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) is a global, prospective, multicenter, longitudinal protocol designed to characterize MPS VII disease presentation and progression, assess long-term effectiveness and safety of vestronidase alfa, including hypersensitivity reactions and immunogenicity , as well as prospectively investigate longitudinal change across biomarker(s), clinical assessments, and patient/ caregiver-reported outcome measures in a representative population. The aim of this DMP is to collect data on patients with MPS VII to provide a comprehensive dataset on the clinical presentation, heterogeneity, and disease progression, and meaningful standardized ICH GCP-quality data collected in-clinic across multiple sites globally. The DMP is not a randomized study and both treated and untreated patients will be enrolled.

Interventions

  • Other No Intervention
    Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.

Primary outcome measures

  • Clinical Course of MPS VII Disease [Time frame: 10 years]
  • Long-term Effectiveness of Vestronidase Alfa [Time frame: 10 years]
  • Long-term Safety of Vestronidase Alfa [Time frame: 10 years]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Other

Study locations

United States · 7 centers
  • Children's Hospital of Orange County — Orange
  • Children's National Health System — Washington D.C.
  • Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
  • University of Michigan — Ann Arbor
  • New York University Langone Medical Center — New York
  • University of Utah Medical Center — Salt Lake City
  • Seattle Children's Hospital — Seattle
Argentina · 1 center
  • Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L. — Buenos Aires
Brazil · 1 center
  • Hospital de Clínicas de Porto Alegre — Porto Alegre
France · 1 center
  • Centre Hospitalier Universitaire La Timone — Marseille
Germany · 1 center
  • Universitätsmedizin der Johannes Gutenberg-Universität Mainz — Mainz
Netherlands · 1 center
  • Erasmus University Medical Center Rotterdam — Rotterdam
Portugal · 1 center
  • Centro Hospitalar do Porto — Porto
Spain · 1 center
  • Hospital Universitario Virgen del Rocío Pabellón Infantil — Seville

Identifiers

NCT: NCT03604835 · UX003-CL401 · EUPAS25082

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗