An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Enrolling by invitation · Phase 2/Phase 3
Conditions studied: Mucopolysaccharidosis II
In brief
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant intravenous (IV) enzyme replacement therapy (ERT) for Hunter syndrome (MPS II). Participants who complete at least through the Week 49 visit in Study DNLI-E-0002 and do not discontinue study intervention early and participants who complete Study DNLI-E-0007 will be enrolled in this OLE. All participants will receive DNL310 for up to 5 years from the time of entry in this OLE. Participants, site staff, and the Sponsor will remain blinded to the original treatment assignment for participants entering this OLE from Study DNLI-E-0007.
Key facts
- Study ID
- NCT06075537
- Run by
- Denali Therapeutics Inc.
- People needed
- 99
- Starts
- 2023-09-20
- Expected to finish
- 2027-06-01
- Last updated by the study team
- 2026-06-11
Who can join
Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
- Ann and Robert H Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Hackensack University Medical Center — Hackensack, New Jersey, United States
- UNC Children's Research Institute — Chapel Hill, North Carolina, United States
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- The University of Texas Medical School at Houston — Houston, Texas, United States
- Huntsman Cancer Hospital — Salt Lake City, Utah, United States
- Sanatorio Mater Dei — Buenos Aires, Argentina
- Universitair Ziekenhuis Antwerpen — Edegem, Antwerpen, Belgium
- UZ Brussel — Jette, Belgium
- University of Alberta - Faculty of Medicine & Dentistry — Edmonton, Alberta, Canada
- Hospital for Sick Children — Toronto, Ontario, Canada
- McGill University Health Center — Montreal, Quebec, Canada
- Vseobecna Fakultni Nemocnice V Praze — Prague, Czechia
- Hopital Jeanne De Flandre - Metabolic Diseases Unit — Lille, France
- Universitätsklinikum Hamburg-Eppendorf — Hamburg, Germany
- SpinCS GmbH — Höchheim, Germany
- ASST di Cremona — Cremona, Italy
- Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia — Udine, Italy
- Erasmus Medical Center - Sophia Children's Hospital — Rotterdam, Netherlands
- Hospit U. Vall d'Hebron - PPDS — Barcelona, Spain
- Drottning Silvias Barn Och Ungdomssjukhus — Gothenburg, Sweden
- University Medical Faculty Balcali Hospital — Adana, Turkey (Türkiye)
- Gazi Universitesi Tip Fakultes — Ankara, Turkey (Türkiye)
Full record on ClinicalTrials.gov
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