A Study to Determine the Efficacy and Safety of Tividenofusp Alfa(DNL310) vs Idursulfase in Pediatric and Young Adult ParticipantsWith Neuronopathic or Non Neuronopathic Mucopolysaccharidosis Type II
- Conditions
- Mucopolysaccharidosis Type II [MPS II]Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2021-005200-35-BE
- Lead Sponsor
- Denali Therapeutics Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 54
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 for a minimum of 4 months (ie, 16 weeks) of idursulfase therapy during the period immediately 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
1. Have a documented pathogenic or likely pathogenic variants that are known to cause developmental delay or decline, cognitive dysfunction, seizures, or other significant CNS disorders.
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
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method