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)
Recruiting now · Phase 2/Phase 3
Conditions studied: Mucopolysaccharidosis II
In brief
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.
Key facts
- Study ID
- NCT05371613
- Run by
- Denali Therapeutics Inc.
- People needed
- 63
- Starts
- 2022-07-21
- Expected to finish
- 2027-12-01
- Last updated by the study team
- 2025-08-05
Who can join
Age: 2 and older, up to 25. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Salford Royal Hospital — Salford, United Kingdom (enrolling)
- Ann and Robert H Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
- Hackensack University Medical Center — Hackensack, New Jersey, United States (enrolling)
- UNC Children's Research Institute — Chapel Hill, North Carolina, United States (enrolling)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
- The University of Texas Medical School at Houston — Houston, Texas, United States (enrolling)
- University of Utah, PPDS — Salt Lake City, Utah, United States (enrolling)
- Sanatorio Mater Dei — Buenos Aires, Argentina (enrolling)
- Royal Free Hospital — London, United Kingdom (enrolling)
- UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (enrolling)
- UZ Antwerpen — Antwerp, Antwerpen, Belgium (enrolling)
- Universitair Ziekenhuis Brussel — Jette, Brussels Capital, Belgium (enrolling)
- Hospital de Clínicas de Porto Alegre (HCPA) - PPDS — Porto Alegre, Brazil (enrolling)
- University of Alberta - Faculty of Medicine & Dentistry — Edmonton, Alberta, Canada (enrolling)
- Hospital for Sick Children — Toronto, Ontario, Canada (enrolling)
- McGill University Health Center — Montreal, Quebec, Canada (enrolling)
- Vseobecna Fakultni Nemocnice V Praze — Prague, Czechia (enrolling)
- Hôpital Jeanne de Flandre — Lille, France (enrolling)
- SphinCS — Höchheim, Hochheim, Germany (enrolling)
- Medizinische Universität Lausitz - Carl Thiem — Cottbus, Germany (enrolling)
- Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia — Udine, Italy (enrolling)
- Erasmus Medical Center - Sophia Children's Hospital — Rotterdam, Rotterdam, Netherlands (enrolling)
- Hospital Universitario Vall d'Hebron — Barcelona, Barcelona, Spain (enrolling)
- Hospital Infantil Universitario Niño Jesus — Madrid, Madrid, Spain (enrolling)
Full record on ClinicalTrials.gov
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