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A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
N/A
Waitlist Available
Research Sponsored by Denali Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 18 months
Awards & highlights
No Placebo-Only Group
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.
Eligible Conditions
- Hunter Syndrome
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 18 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 18 months
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Adverse events related to study procedures
Exploratory biomarkers, such as neurofilament light chain, lysosomal lipids/proteins, and cytokines, in patient CSF, urine, and/or blood
Cerebrospinal fluid shunts procedure
+4 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
6Treatment groups
Experimental Treatment
Group I: Part 6Experimental Treatment1 Intervention
Participants from 1 to 17 years of age with nMPS II. Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted. Part 6 will also include a single collection of CSF.
Group II: Part 5Experimental Treatment1 Intervention
Participants ≤3 years of age with an undetermined MPS II phenotype. Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted.
Group III: Part 4Experimental Treatment1 Intervention
Participants 6 to 17 years of age with the non-neuronopathic form of mucopolysaccharidosis type II (nnMPS II). Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted.
Group IV: Part 3Experimental Treatment1 Intervention
Participants \<8 years of age who have the neuronopathic form of mucopolysaccharidosis type II (nMPS II). Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted.
Group V: Part 2Experimental Treatment1 Intervention
Participants from 2 through 30 years of age who have MPS II; Part 2 will entail a single collection of cerebrospinal fluid (CSF), urine, and blood. Clinical outcome assessments are optional in Part 2 for participants aged 18 years or younger; no clinical assessments are planned for participants older than 18 years.
Group VI: Part 1Experimental Treatment1 Intervention
Participants from 2 through 10 years of age who have MPS II. Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted.
Find a Location
Who is running the clinical trial?
Denali Therapeutics Inc.Lead Sponsor
23 Previous Clinical Trials
1,912 Total Patients Enrolled
Anna Bakardjiev, MDStudy DirectorDenali Therapeutics
1 Previous Clinical Trials
47 Total Patients Enrolled
Katia Meirelles, MDStudy DirectorDenali Therapeutics
1 Previous Clinical Trials
47 Total Patients Enrolled