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NCT04251026 · ClinicalTrials.gov registry record · Phase 1
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
A Phase 1 study of Mucopolysaccharidosis II, sponsored by Denali Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 47
- Enrollment target
- 7
- Study locations
NCT04251026: Active Phase 1 study of Mucopolysaccharidosis II, sponsored by Denali Therapeutics.
NCT04251026 is a Phase 1 study of Mucopolysaccharidosis II that is active but no longer recruiting, run by Denali Therapeutics. The registered enrollment target is 47 participants, below the 67-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (30% lower). The trial reports 7 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04251026, a Phase 1 study of Mucopolysaccharidosis II, is active but no longer recruiting, sponsored by Denali Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 47 participants
- Enrollment target
- 7
- Study locations
Study Summary
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.
Conditions Studied
Interventions
- DRUG tividenofusp alfa
Study Locations (7)
California
- UCSF Benioff Children's Hospital - Oakland
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
North Carolina
- UNC Children's Research Institute - Chapel Hill
Pennsylvania
- UPMC | Children's Hospital of Pittsburgh - Pittsburgh
Quebec
- McGill University Health Centre - Royal Victoria Hospital - Montreal
South Holland
- Erasmus Medical Center - Rotterdam
Other
- St Mary's Hospital, Manchester Academic Health Science Centre - Manchester
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 47 participants |
| Start Date | 2020-07-16 |
| Est. Completion | 2031-02 |
| Phase | Phase 1 |
What the registry record for NCT04251026 still lists
NCT04251026 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 47 participants, a relatively small participant target, below the 67-participant average among 7 other Mucopolysaccharidosis II trials with a reported enrollment target (30% lower).
The record links to 1 condition, with Mucopolysaccharidosis II appearing as the primary indexed condition, and to 1 intervention - of which tividenofusp alfa is the first listed.
NCT04251026 lists 7 locations in 7 states (California, Illinois, North Carolina).
Frequently Asked Questions
What is clinical trial NCT04251026 about?
NCT04251026 is a clinical study titled "A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome". This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manif...
What is the current status of trial NCT04251026?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 47 participants. The study started on 2020-07-16. Estimated completion is 2031-02.
What conditions does trial NCT04251026 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis II.
What interventions are being tested in trial NCT04251026?
The interventions under investigation include: tividenofusp alfa (DRUG).
Who is sponsoring clinical trial NCT04251026?
This trial is sponsored by Denali Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04251026 being conducted?
This trial has 7 study locations across California, Illinois, North Carolina, Pennsylvania, Quebec. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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