A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
Completed
Conditions studied: Mucopolysaccharidosis II
In brief
This is a six-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers potentially related to disease severity and/or treatment response and prospectively assess the progression of disease in participants with MPS II who are aged ≤30 years at the time of enrollment.
Key facts
- Study ID
- NCT04007536
- Run by
- Denali Therapeutics Inc.
- People needed
- 18
- Starts
- 2019-10-23
- Expected to finish
- 2024-03-01
- Last updated by the study team
- 2024-06-10
Who can join
Age: any, up to 30. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCSF Benioff Children's Hospital — Oakland, California, United States
- UNC Children's Research Institute — Chapel Hill, North Carolina, United States
- UPMC | Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Erasmus Medical Center — Rotterdam, South Holland, Netherlands
- Birmingham Children's Hospital — Birmingham, United Kingdom
- Manchester Centre for Genomic Medicine — Manchester, United Kingdom
Full record on ClinicalTrials.gov
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