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Compares treatment options for Mucopolysaccharidosis II
Official title A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
ClinicalTrials.gov ID: NCT05371613
What this study is testing
What is tividenofusp alfa?
tividenofusp alfa is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for mucopolysaccharidosis ii.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 25
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)
- Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on...
You likely can't join if
- Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
- Previously received an iduronate 2-sulfatase (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 (MRI)
- Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another...
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Chicago, Illinois, United States
- Hackensack, New Jersey, United States
- Chapel Hill, North Carolina, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
- Buenos Aires, Argentina
- North Adelaide, Australia
- Antwerp, Antwerpen, Belgium
- Jette, Brussels Capital, Belgium
- Porto Alegre, Brazil
- Rio de Janeiro, Brazil
- Edmonton, Alberta, Canada
- Toronto, Ontario, Canada
- Montreal, Quebec, Canada
- Prague, Czechia
- Lille, France
- Höchheim, Hochheim, Germany
+ 12 more site(s).
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, 2 years to 25 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Chicago, Illinois, United States; Hackensack, New Jersey, United States; Chapel Hill, North Carolina, United States; Cincinnati, Ohio, United States; Philadelphia, Pennsylvania, United States and 26 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 ClinicalTrials.gov; the study team decides eligibility.