Mucopolysaccharidosis VIINCT03604835
Mucopolysaccharidosis VII Disease Monitoring Program
An observational study of Mucopolysaccharidosis VII and MPS VII, run by Ultragenyx Pharmaceutical Inc at 14 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis VII, MPS VII, MPS 7
- How many people
- 50 participants
- Where
- 14 sitesin 8 countries
- Study dates
- 29 Jan 2018to Apr 2032
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Trial locations and contacts
14 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 8 countries
United States7 sites, 6 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
- Terminated
New York University Langone Medical Center
New York, New York, 10016
- Recruiting
Children's Hospital of Orange County
Orange, California, 92868
- Recruiting
University of Utah Medical Center
Salt Lake City, Utah, 84112
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
- Recruiting
Children's National Health System
Washington D.C., District of Columbia, 20010
Argentina1 site, 1 recruiting
- Recruiting
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
Buenos Aires, Buenos Aires, C1425FNG
Brazil1 site, 1 recruiting
- Recruiting
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-003
France1 site, 1 recruiting
- Recruiting
Centre Hospitalier Universitaire La Timone
Marseille, Provence-Alpes-Côte d'Azur Region, 13005
Germany1 site, 1 recruiting
- Recruiting
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, 55131
Netherlands1 site
- Terminated
Erasmus University Medical Center Rotterdam
Rotterdam, South Holland, 3015 CN
Portugal1 site, 1 recruiting
- Recruiting
Centro Hospitalar do Porto
Porto, 4050-651
Spain1 site, 1 recruiting
- Recruiting
Hospital Universitario Virgen del Rocío Pabellón Infantil
Seville, Sevilla, 41013
About this study
Official title: Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
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.
Read the 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.
- Conditions
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Being tested
- No Intervention
- Keywords
- UX003, Mepsevii, vestronidase alfa-vjbk, vestronidase alfa, recombinant human beta-glucuronidase
- Medical subject headings
- Mucopolysaccharidosis VII
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.
| Study group | Role | What they receive |
|---|---|---|
| Patients with MPS VII receiving vestronidase-alfa | via prescription, or early access/ compassionate use program Receives: Other: No Intervention | |
| Patients with MPS VII not receiving vestronidase-alfa | no treatment or treatment other than vestronidase alfa Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Course of MPS VII Disease
To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa
Measured: 10 years
Long-term Effectiveness of Vestronidase Alfa
To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa
Measured: 10 years
Long-term Safety of Vestronidase Alfa
Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2018
- Study start29 Jan 2018
- Primary completionApr 2032 (estimated)
- Study completionApr 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis VIIStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceuticals Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03604835
- Sponsor's protocol ID
- UX003-CL401
- Other IDs
- EUPAS25082
- Status last verified
- Aug 2026
- First submitted
- 18 Jul 2018
- Last updated
- 27 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
- ClinicalTrials.gov ID
- NCT03604835
- Acronym / sponsor ID
- UX003-CL401
- Conditions
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Keywords
- UX003, Mepsevii, vestronidase alfa-vjbk, vestronidase alfa, recombinant human beta-glucuronidase
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 29 Jan 2018 (actual)
- Primary completion
- Apr 2032 (estimated)
- Study completion
- Apr 2032 (estimated)
- First posted
- 30 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with MPS VII receiving vestronidase-alfa | – | Other: No Interventionvia prescription, or early access/ compassionate use program |
| Patients with MPS VII not receiving vestronidase-alfa | – | Other: No Interventionno treatment or treatment other than vestronidase alfa |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionAccess to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP. | Other | Patients with MPS VII not receiving vestronidase-alfa, Patients with MPS VII receiving vestronidase-alfa |
Primary outcome
Clinical Course of MPS VII Disease
Time frame: 10 years
To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa
Long-term Effectiveness of Vestronidase Alfa
Time frame: 10 years
To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa
Long-term Safety of Vestronidase Alfa
Time frame: 10 years
Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2026.
- Ultragenyx Patient Advocacy/MPS 7 Disease InformationLink provided by the sponsor.
- Ultragenyx Transparency CommitmentLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Ultragenyx Pharmaceuticals Inc.
14 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis VII and MPS VII, run by Ultragenyx Pharmaceutical Inc at 14 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Argentina, Brazil, France, Germany and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patients Contact: Trial Recruitment (1-888-756-8657, trialrecruitment@ultragenyx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical Course of MPS VII Disease (time frame: 10 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2026.