A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

Conditions

Mucopolysaccharidosis Type II [MPS II]

Brief summary

Percent change from baseline in CSF HS concentration at Week 24 (Cohort A), Change from baseline in the Vineland-3 at Week 96 (Cohort A)

Detailed description

Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) at Week 96 (Cohort A only), Change from baseline in the Vineland-3 ABC at Week 96 (Cohort A only), Change from baseline in serum NfL at Week 96 (Cohort A only), Change from baseline in distance walked (meters) in the 6MWT at Week 48 (Cohort B only), Percent change from baseline in the sum of urine HS and DS concentrations at Week 24 (Cohorts A and B), Percent change from baseline in the sum of urine HS and DS concentrations at Week 48 (Cohorts A and B), Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) at Week 48 (Cohorts A and B), Spleen volume within the normal range (normal vs abnormal) as measured by MRI at Week 48 (Cohorts A and B), Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II at Week 48 (Cohorts A and B)

Related papers

No related papers found yet.

Interventions

DRUGElaprase 2 mg/ml concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Percent change from baseline in CSF HS concentration at Week 24 (Cohort A), Change from baseline in the Vineland-3 at Week 96 (Cohort A)	—

Secondary

Measure	Time frame
Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) at Week 96 (Cohort A only), Change from baseline in the Vineland-3 ABC at Week 96 (Cohort A only), Change from baseline in serum NfL at Week 96 (Cohort A only), Change from baseline in distance walked (meters) in the 6MWT at Week 48 (Cohort B only), Percent change from baseline in the sum of urine HS and DS concentrations at Week 24 (Cohorts A and B), Percent change from baseline in the sum of urine HS and DS concentrations at Week 48 (Cohorts A and B), Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) at Week 48 (Cohorts A and B), Spleen volume within the normal range (normal vs abnormal) as measured by MRI at Week 48 (Cohorts A and B), Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II at Week 48 (Cohorts A and B)	—

Measure

Time frame

Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) at Week 96 (Cohort A only), Change from baseline in the Vineland-3 ABC at Week 96 (Cohort A only), Change from baseline in serum NfL at Week 96 (Cohort A only), Change from baseline in distance walked (meters) in the 6MWT at Week 48 (Cohort B only), Percent change from baseline in the sum of urine HS and DS concentrations at Week 24 (Cohorts A and B), Percent change from baseline in the sum of urine HS and DS concentrations at Week 48 (Cohorts A and B), Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) at Week 48 (Cohorts A and B), Spleen volume within the normal range (normal vs abnormal) as measured by MRI at Week 48 (Cohorts A and B), Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II at Week 48 (Cohorts A and B)

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Countries

Belgium, Czechia, France, Germany, Italy, Netherlands, Spain, Sweden

Outcome results

None listed