Recruiting

Disease Monitoring

Sponsor:

Ultragenyx Pharmaceutical Inc

Code:

NCT03604835

Conditions

Mucopolysaccharidosis VII

MPS VII

MPS 7

Sly Syndrome

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

No Intervention

Study Details

Brief 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.

Conditions

Mucopolysaccharidosis VII

MPS VII

MPS 7

Sly Syndrome

Study ID

NCT03604835

Start date

Jan 29, 2018

Status verified date

Aug, 2026

Completion date

Apr, 2032

Anticipated

Primary completion date

Apr, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

50 participants

Anticipated

Interventions and Outcome Measures

Arms

Patients with MPS VII receiving vestronidase-alfa

via prescription, or early access/ compassionate use program

Patients with MPS VII not receiving vestronidase-alfa

no treatment or treatment other than vestronidase alfa

Interventions

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.

Primary outcome measure

  • Clinical Course of MPS VII Disease [ Time Frame: 10 years ]
  • Long-term Effectiveness of Vestronidase Alfa [ Time Frame: 10 years ]
  • Long-term Safety of Vestronidase Alfa [ Time Frame: 10 years ]

Central Contacts and Locations

Central contacts

Patients Contact: Trial Recruitment

1-888-756-8657trialrecruitment@ultragenyx.com

HCPs Contact: Medical Information

1-888-756-8657medinfo@ultragenyx.com

Locations

Children's Hospital of Orange County

Recruiting

Orange, California, United States, 92868

Children's National Health System

Recruiting

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

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

Recruiting

Chicago, Illinois, United States, 60611

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

University of Utah Medical Center

Recruiting

Salt Lake City, Utah, United States, 84112

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

More Information

Sponsor

Ultragenyx Pharmaceutical Inc

Last update posted

Aug 27, 2026

Last verified

Aug, 2026

Keywords

  • UX003
  • Mepsevii
  • vestronidase alfa-vjbk
  • vestronidase alfa
  • recombinant human beta-glucuronidase

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Ultragenyx Pharmaceutical Inc on 2026-08-27.