Mucopolysaccharidosis VII Disease Monitoring Program
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03604835 |
Recruitment Status :
Recruiting
First Posted : July 30, 2018
Last Update Posted : April 5, 2021
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Condition or disease | Intervention/treatment |
---|---|
Mucopolysaccharidosis VII MPS VII MPS 7 Sly Syndrome | Other: No Intervention |
Study Type : | Observational |
Estimated Enrollment : | 35 participants |
Observational Model: | Other |
Time Perspective: | Prospective |
Official Title: | Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP) |
Actual Study Start Date : | January 29, 2018 |
Estimated Primary Completion Date : | May 2033 |
Estimated Study Completion Date : | May 2033 |

Group/Cohort | Intervention/treatment |
---|---|
Patients with MPS VII receiving vestronidase-alfa
via prescription, or early access/ compassionate use program
|
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. |
Patients with MPS VII not receiving vestronidase-alfa
no treatment or treatment other than vestronidase alfa
|
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. |
- 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.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
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.
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03604835
Contact: Patients Contact: Patient Advocacy | 1-415-483-8800 | patientadvocacy@ultragenyx.com | |
Contact: HCPs Contact: Medical Information | 1-888-756-8657 | medinfo@ultragenyx.com |
United States, California | |
Children's Hospital of Orange County | Recruiting |
Orange, California, United States, 92868 | |
United States, District of Columbia | |
Children's National Health System | Recruiting |
Washington, District of Columbia, United States, 20010 | |
United States, Illinois | |
Ann & Robert H. Lurie Children's Hospital of Chicago | Recruiting |
Chicago, Illinois, United States, 60611 | |
United States, New York | |
New York University Langone Medical Center | Recruiting |
New York, New York, United States, 10016 | |
United States, Washington | |
Seattle Children's Hospital | Recruiting |
Seattle, Washington, United States, 98105 | |
Argentina | |
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L. | Recruiting |
Buenos Aires, Argentina, C1425FNG | |
Brazil | |
Hospital de Clínicas de Porto Alegre | Recruiting |
Porto Alegre, Rio Grande Do Sul, Brazil, 90035-003 | |
France | |
Centre Hospitalier Universitaire La Timone | Recruiting |
Marseille, Provence Alpes Cote D'Azur, France, 13005 | |
Netherlands | |
Erasmus University Medical Center Rotterdam | Recruiting |
Rotterdam, Zuid-Holland, Netherlands, 3015 CN | |
Portugal | |
Centro Hospitalar do Porto | Recruiting |
Porto, Portugal, 4050-651 | |
Spain | |
Hospital Universitario Virgen del Rocío Pabellón Infantil | Recruiting |
Sevilla, Spain, 41013 |
Study Director: | Medical Director | Ultragenyx Pharmaceuticals Inc. |
Responsible Party: | Ultragenyx Pharmaceutical Inc |
ClinicalTrials.gov Identifier: | NCT03604835 |
Other Study ID Numbers: |
UX003-CL401 EUPAS25082 ( Other Identifier: EU PAS Number ) |
First Posted: | July 30, 2018 Key Record Dates |
Last Update Posted: | April 5, 2021 |
Last Verified: | April 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
UX003 Mepsevii vestronidase alfa-vjbk vestronidase alfa recombinant human beta-glucuronidase |
Mucopolysaccharidoses Mucopolysaccharidosis VII Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |