A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase
This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants
| Condition(s) | Mucopolysaccharidosis II |
|---|---|
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Study type | Interventional |
| Summary | This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria. |
| Who can participate | Key Inclusion Criteria: * Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<26 years (Cohort B) * Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II) * Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening Key Exclusion Criteria: * Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay * Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy * Received any CNS-targeted MPS ERT within 6 months prior to screening * Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI) * Participa |
| Ages | 2 Years to 25 Years |
| Sex | All |
| Lead sponsor | Denali Therapeutics Inc. |
| Locations | Oakland, California, United States; Chicago, Illinois, United States; Hackensack, New Jersey, United States; Chapel Hill, North Carolina, United States; Cincinnati, Ohio, United States; Philadelphia, Pennsylvania, United States (+26 more sites) |
| Start date | 2022-07-21 |
| NCT ID | NCT05371613 |
| Official listing | https://clinicaltrials.gov/study/NCT05371613 |