NCT04007536

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

A Prospective, Longitudinal Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 18 (actual)
• Sponsor: Denali Therapeutics Inc. · Industry
• Sex: All
• Age: 30 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

Conditions

• Mucopolysaccharidosis II

Interventions

Type	Name	Description
OTHER	No Intervention	No Intervention

Timeline

• Start date: 2019-10-23
• Primary completion: 2024-03-01
• Completion: 2024-03-01
• First posted: 2019-07-05
• Last updated: 2024-06-10

Locations

6 sites across 3 countries: United States, Netherlands, United Kingdom