Mucopolysaccharidosis VII Disease Monitoring Program

Ultragenyx Pharmaceutical Inc50 enrolled

Overview

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.

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 Type

OBSERVATIONAL

Enrollment

Conditions

Mucopolysaccharidosis VII MPS VII MPS 7 Sly Syndrome

Interventions

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.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (14)

Children's Hospital of Orange County

Orange, California, United States

RECRUITING

Children's National Health System

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

Outcomes

Primary Outcomes

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

Time frame: 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

Time frame: 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.

Time frame: 10 years

Central 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

Data from ClinicalTrials.gov

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