Clinical Trials Logo

Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT03604835
Other study ID # UX003-CL401
Secondary ID EUPAS25082
Status Recruiting
Phase
First received
Last updated
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
Health authority
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.

Study Design


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
See also
  Status Clinical Trial Phase
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT02432144 - A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Phase 3
Recruiting NCT05619900 - Registry of Patients Diagnosed With Lysosomal Storage Diseases
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed NCT02418455 - Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age Phase 2
Recruiting NCT06036693 - MPS (RaDiCo Cohort) (RaDiCo-MPS)
Available NCT03775174 - Expanded Access to Mepsevii