Mucopolysaccharidosis VII Disease Monitoring Program
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorUltragenyx Pharmaceutical Inc
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.
Trial 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.
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
No Intervention
Other interventionAccess to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.
Treatment groups
50 Participants
are divided into 2 treatment groupsGroup A: Patients with MPS VII receiving vestronidase-alfa1 intervention
Group B: Patients with MPS VII not receiving vestronidase-alfa1 intervention
Trial outcomes
Primary outcomes
1
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
Time frame
10 years2
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
Time frame
10 years3
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.
Time frame
10 yearsSponsors and contacts
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Source ClinicalTrials.gov