Study to determine effectiveness and safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Hunter Syndrome

Phase 1

• Conditions: Mucopolysaccharidosis Type II [MPS II]; MedDRA version: 20.1Level: PTClassification code 10056889Term: Mucopolysaccharidosis IISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2021-005200-35-IT
• Lead Sponsor: Denali Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-09-13
• Last Update Posted: 28 August 2023

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• 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
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 (MRIs)

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 clinical trial of medicinal product
• Study Design: Not specified