Mucopolysaccharidosis VII Disease Monitoring Program

Mucopolysaccharidosis VII Clinical Trial

Official title:

Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03604835
• Other study ID #: UX003-CL401
• Secondary ID: EUPAS25082
• Status: Recruiting
• Start date: January 29, 2018
• Est. completion date: May 2033

Study information

• Verified date: May 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: Patients Contact: Trial Recruitment
• Phone: 1-888-756-8657
• Email: trialrecruitment[@]ultragenyx.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: May 2033
• Est. primary completion date: May 2033
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• MPS 7
• MPS VII
• Mucopolysaccharidoses
• Mucopolysaccharidosis VII
• Sly Syndrome

Intervention

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.

Locations

Country	Name	City	State
Argentina	Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.	Buenos Aires
Brazil	Hospital de Clínicas de Porto Alegre	Porto Alegre	Rio Grande Do Sul
France	Centre Hospitalier Universitaire La Timone	Marseille	Provence Alpes Cote D'Azur
Germany	Universitätsmedizin der Johannes Gutenberg-Universität Mainz	Mainz
Netherlands	Erasmus University Medical Center Rotterdam	Rotterdam	Zuid-Holland
Portugal	Centro Hospitalar do Porto	Porto
Spain	Hospital Universitario Virgen del Rocío Pabellón Infantil	Sevilla
Turkey	Gazi University	Ankara
United States	University of Michigan	Ann Arbor	Michigan
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	New York University Langone Medical Center	New York	New York
United States	Children's Hospital of Orange County	Orange	California
United States	University of Utah Medical Center	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	Children's National Health System	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Argentina,  Brazil,  France,  Germany,  Netherlands,  Portugal,  Spain,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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

External Links

•   Ultragenyx Patient Advocacy/MPS 7 Disease Information