NCT03604835 Mucopolysaccharidosis VII Disease Monitoring Program
| NCT ID | NCT03604835 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Ultragenyx Pharmaceutical Inc |
| Condition | Mucopolysaccharidosis VII |
| Study Type | OBSERVATIONAL |
| Enrollment | 50 participants |
| Start Date | 2018-01-29 |
| Primary Completion | 2032-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 50 participants in total. It began in 2018-01-29 with a primary completion date of 2032-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
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.
Contact & Investigator
Medical Director
STUDY DIRECTOR
Ultragenyx Pharmaceuticals Inc.
Frequently Asked Questions
Who can join the NCT03604835 clinical trial?
This trial is open to participants of all sexes, studying Mucopolysaccharidosis VII. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT03604835 currently recruiting?
Yes, NCT03604835 is actively recruiting participants. Contact the research team at trialrecruitment@ultragenyx.com for enrollment information.
Where is the NCT03604835 trial being conducted?
This trial is being conducted at Orange, United States, Washington D.C., United States, Chicago, United States, Ann Arbor, United States and 10 additional locations.
Who is sponsoring the NCT03604835 clinical trial?
NCT03604835 is sponsored by Ultragenyx Pharmaceutical Inc. The principal investigator is Medical Director at Ultragenyx Pharmaceuticals Inc.. The trial plans to enroll 50 participants.