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A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non Neuronopathic Mucopolysaccharidosis Type II
EU CTIS ID: 2024-510990-21-00
What this study is testing
1. To evaluate the CNS activity of DNL310 vs idursulfase as measured by the CSF concentration of HS in nMPS II participants 2. To evaluate the clinical CNS efficacy of DNL310 vs idursulfase on adaptive behavior as assessed by the Vineland-3 in nMPS II participants
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participants aged ≥2 to <6 years (Cohort A) or ≥6 to <26 years (Cohort B)
- Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
- For non-run-in Cohort A and Cohort B only: Be on maintenance ERT and have tolerated a minimum of 4 months (ie, 16 weeks) of idursulfase therapy during the period immediately prior to screening.
You likely can't join if
- Have documented pathogenic or likely pathogenic variants that are known to cause developmental delay or decline, cognitive dysfunction, seizures, or other significant CNS disorders.
- 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 imaging (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
The study team makes the final eligibility decision.
Where it's taking place
- Brazil
- United States
- Turkey
- Mexico
- Canada
- Australia
- United Kingdom
- Argentina
- Colombia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Brazil; United States; Turkey; Mexico; Canada; Australia and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.