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Rare disease study tracks MPS VII over time

NCT ID NCT03604835

Knowledge-focused Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 2 times

Summary

This study monitors up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. It also checks the long-term safety and effectiveness of the drug vestronidase alfa. Participants may or may not be taking the drug, and the study involves regular check-ups and tests.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2018

Expected to finish

Apr 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients with a confirmed diagnosis of MPS VII, including patients who already received vestronidase alfa in an Ultragenyx clinical trial or early access/ compassionate use program, and patients not receiving vestronidase alfa. Patients who were previously enrolled in an Ultragenyx-sponsored clinical trial may participate in the DMP if they have completed or discontinued from the clinical trial.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 7

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MPS 7 MPS vii Mucopolysaccharidosis vii Sly syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites in 7 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Centre Hospitalier Universitaire La Timone

    RECRUITING

    Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France

  • Centro Hospitalar do Porto

    RECRUITING

    Porto, 4050-651, Portugal

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Children's National Health System

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Erasmus University Medical Center Rotterdam

    TERMINATED

    Rotterdam, South Holland, 3015 CN, Netherlands

  • Hospital Universitario Virgen del Rocío Pabellón Infantil

    RECRUITING

    Seville, Sevilla, 41013, Spain

  • Hospital de Clínicas de Porto Alegre

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90035-003, Brazil

  • Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.

    RECRUITING

    Buenos Aires, Buenos Aires, C1425FNG, Argentina

  • New York University Langone Medical Center

    TERMINATED

    New York, New York, 10016, United States

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Utah Medical Center

    RECRUITING

    Salt Lake City, Utah, 84112, United States

  • Universitätsmedizin der Johannes Gutenberg-Universität Mainz

    RECRUITING

    Mainz, 55131, Germany

More trials for these conditions

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