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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
Sponsor: Denali Therapeutics Inc.
Summary
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.
Official title: An Open-Label Extension to Investigate the Long-Term Safety, Tolerability, and Efficacy of DNL310 in Patients With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Key Details
Gender
All
Age Range
Any - 18 Years
Study Type
INTERVENTIONAL
Enrollment
99
Start Date
2023-09-20
Completion Date
2027-06
Last Updated
2026-03-11
Healthy Volunteers
No
Conditions
Interventions
tividenofusp alfa
Intravenous repeating dose
Locations (25)
UCSF Benioff Children's Hospital Oakland
Oakland, California, 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)
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
Great Ormond Street Hospital
London, United Kingdom