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

DNLI-E-0007

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.

2022-07-21

2025-12-01

2025-12-01

Recruiting

Early phase I

54

Key Inclusion Criteria: * Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<17 years (Cohort B) * Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II) * Be on maintenance enzyme replacement therapy (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 imagings (MRIs) * Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2', 'PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'DOUBLE', 'whoMasked': ['PARTICIPANT', 'INVESTIGATOR']}}, 'enrollmentInfo': {'count': 54, 'type': 'ESTIMATED'}}

2024-05-01