A Study to the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

• Status: Not yet recruiting
• Trial end date: 2025-12-01
• Target enrollment: 0
• Participant gender: All

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 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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Denali Therapeutics Inc.

Criteria

Key Inclusion Criteria:

• Parrticipants 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 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