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NCT03604835 · ClinicalTrials.gov registry record
Mucopolysaccharidosis VII Disease Monitoring Program
A clinical trial of Mucopolysaccharidosis VII and MPS VII, sponsored by Ultragenyx Pharmaceutical.
- Recruiting
- Registry status
- 50
- Enrollment target
- 14
- Study locations
NCT03604835: Recruiting study of Mucopolysaccharidosis VII and MPS VII, sponsored by Ultragenyx Pharmaceutical.
NCT03604835 is a study of Mucopolysaccharidosis VII and MPS VII that is actively recruiting participants, run by Ultragenyx Pharmaceutical. The registered enrollment target is 50 participants. The trial reports 14 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03604835, a study of Mucopolysaccharidosis VII and MPS VII, is actively recruiting participants, sponsored by Ultragenyx Pharmaceutical.
- RECRUITING
- Registry status
- 50 participants
- Enrollment target
- 14
- Study locations
Study Summary
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.
Primary Outcome
To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa
Conditions Studied
Interventions
- OTHER No Intervention
Study Locations (14)
Other
- Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L. - Buenos Aires
- Universitätsmedizin der Johannes Gutenberg-Universität Mainz - Mainz
- Centro Hospitalar do Porto - Porto
- Hospital Universitario Virgen del Rocío Pabellón Infantil - Seville
California
- Children's Hospital of Orange County - Orange
District of Columbia
- Children's National Health System - Washington D.C.
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Michigan
- University of Michigan - Ann Arbor
New York
- New York University Langone Medical Center - New York
Utah
- University of Utah Medical Center - Salt Lake City
Washington
- Seattle Children's Hospital - Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2018-01-29 |
| Est. Completion | 2033-05 |
What NCT03604835 shows while recruiting
NCT03604835 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 50 participants, a relatively small participant target.
The record links to 4 conditions, with Mucopolysaccharidosis VII appearing as the primary indexed condition, and to 1 intervention - of which No Intervention is the first listed.
NCT03604835 lists 14 locations in 11 states (Other, California, District of Columbia).
Frequently Asked Questions
What is clinical trial NCT03604835 about?
NCT03604835 is a clinical study titled "Mucopolysaccharidosis VII Disease Monitoring Program". 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.
What is the current status of trial NCT03604835?
This trial is currently recruiting. The enrollment target is 50 participants. The study started on 2018-01-29. Estimated completion is 2033-05.
What conditions does trial NCT03604835 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis VII, MPS VII, Sly Syndrome, MPS 7.
What interventions are being tested in trial NCT03604835?
The interventions under investigation include: No Intervention (OTHER).
Who is sponsoring clinical trial NCT03604835?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03604835 being conducted?
This trial has 14 study locations across California, District of Columbia, Illinois, Michigan, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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