Clinical trial NCT03604835: Mucopolysaccharidosis VII Disease Monitoring Program
Official title: Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Mucopolysaccharidosis VII
MPS VII
MPS 7
Sly Syndrome
Keywords: UX003
Mepsevii
vestronidase alfa-vjbk
vestronidase alfa
recombinant human beta-glucuronidase
Lead sponsor: Ultragenyx Pharmaceutical Inc
Sponsor class: INDUSTRY
First posted: 2018-07-30
Enrollment: 50
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
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.

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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Clinical Course of MPS VII Disease
timeFrame: 10 years
measure: Long-term Effectiveness of Vestronidase Alfa
timeFrame: 10 years
measure: Long-term Safety of Vestronidase Alfa
timeFrame: 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Washington
country: United States
facility: Children's National Health System
state: District of Columbia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: New York
country: United States
facility: New York University Langone Medical Center
state: New York
city: Salt Lake City
country: United States
facility: University of Utah Medical Center
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Buenos Aires
country: Argentina
facility: Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
city: Porto Alegre
country: Brazil
facility: Hospital de Clínicas de Porto Alegre
state: Rio Grande Do Sul
city: Marseille
country: France
facility: Centre Hospitalier Universitaire La Timone
state: Provence Alpes Cote D'Azur
city: Mainz
country: Germany
facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz
city: Rotterdam
country: Netherlands
facility: Erasmus University Medical Center Rotterdam
state: Zuid-Holland
city: Porto
country: Portugal
facility: Centro Hospitalar do Porto
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío Pabellón Infantil
Study officials:
affiliation: Ultragenyx Pharmaceuticals Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: