Mucopolysaccharidosis VII Disease Monitoring Program

Ultragenyx

Status: Enrolling

Conditions: MPS 7; Sly Syndrome; MPS VII; Mucopolysaccharidosis VII

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT03604835

UX003-CL401

EUPAS25082 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

50 participants in 2 patient groups

Patients with MPS VII receiving vestronidase-alfa

Description:

via prescription, or early access/ compassionate use program

Treatment:

Other: No Intervention

Patients with MPS VII not receiving vestronidase-alfa

Description:

no treatment or treatment other than vestronidase alfa

Treatment:

Other: No Intervention