A Study to Determine the Efficacy and Safety of Tividenofusp Alfa(DNL310) vs Idursulfase in Pediatric and Young Adult Participants WithNeuronopathic or Non Neuronopathic Mucopolysaccharidosis Type II

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-DE
• Lead Sponsor: Denali Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-04-27
• Last Update Posted: 29 July 2024

Recruitment & Eligibility

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

Inclusion Criteria

1. Participants aged =2 to <6 years (Cohort A) or =6 to <26 years (Cohort B)

2. Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)

3. For non-run-in Cohort A and Cohort B only: Be on maintenance enzyme replacement therapy (ERT) and have tolerated idursulfase therapy for a minimum of 4 months (ie, 16 weeks) prior to screening
Are the trial subjects under 18? yes
Number of subjects for this age range: 54
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Have a documented pathogenic or likely pathogenic variants that are knwon to cause developmental delay or decline, cognitivedysfunction, seizures, or other significant CNS discorders.

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