Mucopolysaccharidosis VII Disease Monitoring Program

Launched by ULTRAGENYX PHARMACEUTICAL INC · Jul 27, 2018

Keywords

ClinConnect Summary

The Mucopolysaccharidosis VII Disease Monitoring Program is a clinical trial aimed at understanding how MPS VII, also known as Sly Syndrome, develops and changes over time. The study will also evaluate the long-term safety and effectiveness of a treatment called vestronidase alfa, which is designed to help manage this condition. Researchers are particularly interested in how patients respond to the treatment, including any allergic reactions that might occur.

To participate in this trial, individuals must have a confirmed diagnosis of MPS VII through specific laboratory tests. They should also be willing to follow the study schedule and provide consent, or have a guardian provide consent if they are under 18 years old. It's important to note that participants cannot be involved in other clinical trials sponsored by different companies at the same time without special permission. Those who join can expect regular check-ups and monitoring throughout the study to help improve understanding and treatment of this rare disease.

Gender

ALL

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.