Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

RECRUITING

Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835 · View on ClinicalTrials.gov ↗

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.

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

FieldValue
Enrollment Target 50 participants
Start Date 2018-01-29
Est. Completion 2033-05

Sponsor

Ultragenyx Pharmaceutical

23 total trials

Interested in This Trial?

Always speak with your doctor before enrolling in a clinical trial.

Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03604835

The ClinicalTrials.gov registry entry for NCT03604835 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 50 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ultragenyx Pharmaceutical, which has 23 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03604835 reports 14 study locations spanning 11 distinct geographic areas — top geographies include Other, California, District of Columbia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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, Sly Syndrome, MPS VII, MPS 7. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03604835?

The interventions under investigation include: No Intervention (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03604835?

This trial is sponsored by Ultragenyx Pharmaceutical, which has 23 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial