Trials / Active Not Recruiting
Active Not RecruitingNCT04251026
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants With Hunter Syndrome
- Status
- Active Not Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 47 (actual)
- Sponsor
- Denali Therapeutics Inc. · Industry
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | tividenofusp alfa | Intravenous repeating dose |
Timeline
- Start date
- 2020-07-16
- Primary completion
- 2031-02-01
- Completion
- 2031-02-01
- First posted
- 2020-01-31
- Last updated
- 2025-08-07
Locations
7 sites across 4 countries: United States, Canada, Netherlands, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04251026. Inclusion in this directory is not an endorsement.