Mucopolysaccharidosis Type II [MPS II]
Conditions
Brief summary
1. Incidence and intensity of treatment emergent adverse events (TEAEs) throughout the treatment period [Time Frame: 5 years] 2. Clinically significant changes in urine total glycosaminoglycan (GAG) concentrations throughout the treatment period [Time Frame: 5 years] 3. Incidence and intensity of infusion related reactions (IRRs) throughout the treatment period [Time Frame: 5 years]
Detailed description
1.Percent change from baseline in CSF HS concentration [Time Frame: 5 years], 2.Change from baseline in the Vineland-3 ABC [Time Frame: 5 years], 3.Change from baseline in the BSID III cognitive raw score [Time Frame: 5 years], 4.Change from baseline in distance walked (meters) in the 6MWT [Time Frame: 5 years], 5.Percent change from baseline in the sum of urine HS and dermatan sulfate concentrations [Time Frame: 5 years], 6.Liver volume within the normal range (normal vs abnormal) as measured by MRI [Time Frame: 5 years], 7.Spleen volume within the normal range (normal vs abnormal) as measured by MRI [Time Frame: 5 years], 8.Improvement in CaGI C Overall MPS II (defined as much improved or a little improved) [Time Frame: 5 years]
Interventions
None listed
Sponsors
Denali Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Incidence and intensity of treatment emergent adverse events (TEAEs) throughout the treatment period [Time Frame: 5 years] 2. Clinically significant changes in urine total glycosaminoglycan (GAG) concentrations throughout the treatment period [Time Frame: 5 years] 3. Incidence and intensity of infusion related reactions (IRRs) throughout the treatment period [Time Frame: 5 years] | — |
Secondary
| Measure | Time frame |
|---|---|
| 1.Percent change from baseline in CSF HS concentration [Time Frame: 5 years], 2.Change from baseline in the Vineland-3 ABC [Time Frame: 5 years], 3.Change from baseline in the BSID III cognitive raw score [Time Frame: 5 years], 4.Change from baseline in distance walked (meters) in the 6MWT [Time Frame: 5 years], 5.Percent change from baseline in the sum of urine HS and dermatan sulfate concentrations [Time Frame: 5 years], 6.Liver volume within the normal range (normal vs abnormal) as measured by MRI [Time Frame: 5 years], 7.Spleen volume within the normal range (normal vs abnormal) as measured by MRI [Time Frame: 5 years], 8.Improvement in CaGI C Overall MPS II (defined as much improved or a little improved) [Time Frame: 5 years] | — |
Countries
Belgium, Czechia, France, Germany, Italy, Netherlands, Spain, Sweden
Outcome results
None listed