Clinical Research Directory
Browse clinical research sites, groups, and studies.
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)
Sponsor: Denali Therapeutics Inc.
Summary
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.
Official title: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II
Key Details
Gender
All
Age Range
2 Years - 25 Years
Study Type
INTERVENTIONAL
Enrollment
63
Start Date
2022-07-21
Completion Date
2027-12
Last Updated
2025-08-05
Healthy Volunteers
No
Conditions
Interventions
tividenofusp alfa
Intravenous repeating dose
idursulfase
Intravenous repeating dose
Locations (32)
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
University of Utah, PPDS
Salt Lake City, Utah, United States
Sanatorio Mater Dei
Buenos Aires, Argentina
Women's and Children's Hospital
North Adelaide, Australia
UZ Antwerpen
Antwerp, Antwerpen, Belgium
Universitair Ziekenhuis Brussel
Jette, Brussels Capital, Belgium
Hospital de Clínicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Brazil
Instituto Fernandes Figueira
Rio de Janeiro, Brazil
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
Hôpital Jeanne de Flandre
Lille, France
SphinCS
Höchheim, Hochheim, Germany
Medizinische Universität Lausitz - Carl Thiem
Cottbus, Germany
Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia
Udine, Italy
Erasmus Medical Center - Sophia Children's Hospital
Rotterdam, Rotterdam, Netherlands
Hospital Universitario Vall d'Hebron
Barcelona, Barcelona, Spain
Hospital Infantil Universitario Niño Jesus
Madrid, Madrid, Spain
Drottning Silvias Barn Och Ungdomssjukhus
Gothenburg, Sweden
Cukurova University Medical Faculty Balcali Hospital
Adana, Turkey (Türkiye)
Gazi Universitesi Tip Fakultesi
Çankaya, Turkey (Türkiye)
Great Ormond Street Hospital for Children
London, London, United Kingdom
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
Royal Free Hospital
London, United Kingdom
Salford Royal Hospital
Salford, United Kingdom