A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II
Denali Therapeutics Inc.
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.
Eligibility
- Age range
- 2–25 years
- Sex
- All
- Healthy volunteers
- No
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)…
Interventions
- Drugtividenofusp alfa
Intravenous repeating dose
- Drugidursulfase
Intravenous repeating dose
Locations (32)
- UCSF Benioff Children's Hospital OaklandOakland, California
- Ann and Robert H Lurie Children's Hospital of ChicagoChicago, Illinois
- Hackensack University Medical CenterHackensack, New Jersey
- UNC Children's Research InstituteChapel Hill, North Carolina
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania