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Mucopolysaccharidosis VII Disease Monitoring Program

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ClinicalTrials.gov Identifier: NCT03604835
Recruitment Status : Recruiting
First Posted : July 30, 2018
Last Update Posted : August 5, 2019
Sponsor:
Collaborator:
PRA Health Sciences
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Study Description
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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.

Condition or disease Intervention/treatment
Mucopolysaccharidosis VII MPS VII MPS 7 Sly Syndrome Other: No Intervention

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.

Study Design
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Study Type : Observational
Estimated Enrollment : 35 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Actual Study Start Date : January 29, 2018
Estimated Primary Completion Date : May 2033
Estimated Study Completion Date : May 2033

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Patients with MPS VII receiving vestronidase-alfa
via prescription, or early access/ compassionate use program
 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.
Patients with MPS VII not receiving vestronidase-alfa
no treatment or treatment other than vestronidase alfa
 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.


Outcome Measures
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Primary Outcome Measures :
  1. Clinical Course of MPS VII Disease [ Time Frame: 10 years ]
    To characterize MPS VII disease presentation and progression over time in patients treated and not treated with vestronidase alfa

  2. Long-term Effectiveness of Vestronidase Alfa [ Time Frame: 10 years ]
    To evaluate longitudinal change in biomarker(s), clinical assessments and patient/caregiver reported outcomes to examine the effectiveness of vestronidase alfa

  3. Long-term Safety of Vestronidase Alfa [ Time Frame: 10 years ]
    Hypersensitivity reactions, immunogenicity and other safety outcomes will be assessed to examine the long-term safety of vestronidase alfa.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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 16 years, depending on the region) 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 enrollment in an Ultragenyx-sponsored clinical trial.
  • Participation in any other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03604835


Contacts
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Contact: Patients Contact: Patient Advocacy 1-415-483-8800 patientadvocacy@ultragenyx.com
Contact: HCPs Contact: Medical Information 1-888-756-8657 medinfo@ultragenyx.com

Locations
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United States, California
Children's Hospital of Orange County Recruiting
Orange, California, United States, 92868
United States, New York
New York University Langone Medical Center Recruiting
New York, New York, United States, 10016
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
PRA Health Sciences
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceuticals Inc.
More Information
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03604835     History of Changes
Other Study ID Numbers: UX003-CL401
First Posted: July 30, 2018    Key Record Dates
Last Update Posted: August 5, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ultragenyx Pharmaceutical Inc:
UX003
Mepsevii
vestronidase alfa-vjbk
vestronidase alfa
recombinant human beta-glucuronidase

Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis VII
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
 Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases