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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 : April 5, 2021
Sponsor:
Collaborator:
PRA Health Sciences
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Tracking Information
First Submitted Date July 18, 2018
First Posted Date July 30, 2018
Last Update Posted Date April 5, 2021
Actual Study Start Date January 29, 2018
Estimated Primary Completion Date May 2033   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 27, 2018)
  • 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
  • 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
  • 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.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Mucopolysaccharidosis VII Disease Monitoring Program
Official Title Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
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.
Study Type Observational
Study Design Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.

Condition
  • Mucopolysaccharidosis VII
  • MPS VII
  • MPS 7
  • 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.
Study Groups/Cohorts
  • Patients with MPS VII receiving vestronidase-alfa
    via prescription, or early access/ compassionate use program
    Intervention: Other: No Intervention
  • Patients with MPS VII not receiving vestronidase-alfa
    no treatment or treatment other than vestronidase alfa
    Intervention: Other: No Intervention
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 27, 2018)
35
Original Estimated Enrollment Same as current
Estimated Study Completion Date May 2033
Estimated Primary Completion Date May 2033   (Final data collection date for primary outcome measure)
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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Patients Contact: Patient Advocacy 1-415-483-8800 patientadvocacy@ultragenyx.com
Contact: HCPs Contact: Medical Information 1-888-756-8657 medinfo@ultragenyx.com
Listed Location Countries Argentina,   Brazil,   France,   Netherlands,   Portugal,   Spain,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03604835
Other Study ID Numbers UX003-CL401
EUPAS25082 ( Other Identifier: EU PAS Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Ultragenyx Pharmaceutical Inc
Study Sponsor Ultragenyx Pharmaceutical Inc
Collaborators PRA Health Sciences
Investigators
Study Director: Medical Director Ultragenyx Pharmaceuticals Inc.
PRS Account Ultragenyx Pharmaceutical Inc
Verification Date April 2021