Study to determine the effectiveness and safety of DNL310 vs idursulfase in pediatric participants with neuronopathic or non-neuronopathic Hunter Syndrome

Phase 2

• Conditions: Mucopolysaccharidosis Type II [MPS II]; Nutritional, Metabolic, Endocrine; Mucopolysaccharidosis, type II

• Registration Number: ISRCTN11652897
• Lead Sponsor: Denali Therapeutics (United States)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-06-30
• Last Update Posted: 28 November 2022
• Study Completion: 2025-09-01

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 54

Inclusion Criteria

1. Participants aged =2 to <6 years (Cohort A) or =6 to <17 years (Cohort B)
2. Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
3. Be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase for a minimum of 4 months prior to screening

Exclusion Criteria

1. Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
2. Previously received an IDS gene therapy or stem cell therapy
3. Received any CNS-targeted MPS ERT within 6 months prior to screening
4. Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI)
5. 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified