Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835 | Recruiting FDA Drug

• 2 other identifiers: UX003-CL401EUPAS25082
• Study Type: observational
• Target: 50
• Locations: 8 countries
• Sites: 14

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

MPS VII; MPS 7; Sly Syndrome; Mucopolysaccharidosis VII

Keywords

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

Outcome Measures

Primary Outcomes (3)

• 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

  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

  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.

  10 years

Study Arms (2)

Patients with MPS VII receiving vestronidase-alfa

via prescription, or early access/ compassionate use program

Other: No Intervention

Patients with MPS VII not receiving vestronidase-alfa

no treatment or treatment other than vestronidase alfa

Other: No Intervention

Interventions

No Intervention OTHER

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; Patients with MPS VII receiving vestronidase-alfa

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.

Sponsors & Collaborators

• Ultragenyx Pharmaceutical Inc: lead

Study Sites (14)

Children's Hospital of Orange County

Orange, California, 92868, United States

RECRUITING

Children's National Health System

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

RECRUITING

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

Chicago, Illinois, 60611, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

New York University Langone Medical Center

New York, New York, 10016, United States

TERMINATED

University of Utah Medical Center

Salt Lake City, Utah, 84112, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

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

Buenos Aires, C1425FNG, Argentina

RECRUITING

Hospital de Clínicas de Porto Alegre

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

RECRUITING

Centre Hospitalier Universitaire La Timone

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

RECRUITING

Universitätsmedizin der Johannes Gutenberg-Universität Mainz

Mainz, 55131, Germany

RECRUITING

Erasmus University Medical Center Rotterdam

Rotterdam, South Holland, 3015 CN, Netherlands

TERMINATED

Centro Hospitalar do Porto

Porto, 4050-651, Portugal

RECRUITING

Hospital Universitario Virgen del Rocío Pabellón Infantil

Seville, 41013, Spain

RECRUITING

Related Links

• Ultragenyx Patient Advocacy/MPS 7 Disease Information
• Ultragenyx Transparency Commitment

MeSH Terms

Conditions

Mucopolysaccharidosis VII

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Ultragenyx Pharmaceuticals Inc.

  STUDY DIRECTOR

Central Study Contacts

Patients Contact: Trial Recruitment

CONTACT

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

HCPs Contact: Medical Information

CONTACT

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

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 18, 2018
• First Posted: July 30, 2018
• Study Start: January 29, 2018
• Primary Completion (Estimated): May 1, 2033
• Study Completion (Estimated): May 1, 2033
• Last Updated: April 15, 2026

Record last verified: 2026-04

Locations

FR (1); US (7); ES (1); AR (1); BR (1); PT (1); DE (1); NL (1)