Actively Recruiting
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)
Led by Denali Therapeutics Inc. · Updated on 2025-08-05
63
Participants Needed
32
Research Sites
279 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 2 to less than 6 years (Cohort A) or 6 to less than 26 years (Cohort B)
- Confirmed diagnosis of mucopolysaccharidosis type II (nMPS II for Cohort A; nnMPS II for Cohort B)
- No prior enzyme replacement therapy or not on continuous ERT for 4 months before screening or on maintenance ERT with tolerance to idursulfase for at least 4 months before screening
You will not qualify if you...
- Documented mutation of other genes or genetic diagnosis causing developmental delay
- Prior treatment with iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy
- Received any central nervous system-targeted MPS enzyme replacement therapy within 6 months before screening
- Contraindication for lumbar punctures or magnetic resonance imaging (MRI)
- Participation in another investigational drug study or use of investigational drug within 60 days before screening or planned use during the study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 32 locations
1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
Actively Recruiting
2
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
3
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Actively Recruiting
4
UNC Children's Research Institute
Chapel Hill, North Carolina, United States, 27514
Actively Recruiting
5
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
6
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
7
The University of Texas Medical School at Houston
Houston, Texas, United States, 77030
Actively Recruiting
8
University of Utah, PPDS
Salt Lake City, Utah, United States, 84132
Actively Recruiting
9
Sanatorio Mater Dei
Buenos Aires, Argentina
Actively Recruiting
10
Women's and Children's Hospital
North Adelaide, Australia
Withdrawn
11
UZ Antwerpen
Antwerp, Antwerpen, Belgium, 2650
Actively Recruiting
12
Universitair Ziekenhuis Brussel
Jette, Brussels Capital, Belgium, 1090
Actively Recruiting
13
Hospital de Clínicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Brazil
Actively Recruiting
14
Instituto Fernandes Figueira
Rio de Janeiro, Brazil
Withdrawn
15
University of Alberta - Faculty of Medicine & Dentistry
Edmonton, Alberta, Canada
Actively Recruiting
16
Hospital for Sick Children
Toronto, Ontario, Canada, M5G1X8
Actively Recruiting
17
McGill University Health Center
Montreal, Quebec, Canada, H4A3J1
Actively Recruiting
18
Vseobecna Fakultni Nemocnice V Praze
Prague, Czechia, 128 08
Actively Recruiting
19
Hôpital Jeanne de Flandre
Lille, France, 59000
Actively Recruiting
20
SphinCS
Höchheim, Hochheim, Germany, 65239
Actively Recruiting
21
Medizinische Universität Lausitz - Carl Thiem
Cottbus, Germany
Actively Recruiting
22
Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia
Udine, Italy, 33100
Actively Recruiting
23
Erasmus Medical Center - Sophia Children's Hospital
Rotterdam, Rotterdam, Netherlands, 3000
Actively Recruiting
24
Hospital Universitario Vall d'Hebron
Barcelona, Barcelona, Spain, 08035
Actively Recruiting
25
Hospital Infantil Universitario Niño Jesus
Madrid, Madrid, Spain, 28009
Actively Recruiting
26
Drottning Silvias Barn Och Ungdomssjukhus
Gothenburg, Sweden, 416 85
Actively Recruiting
27
Cukurova University Medical Faculty Balcali Hospital
Adana, Turkey (Türkiye), 1330
Actively Recruiting
28
Gazi Universitesi Tip Fakultesi
Çankaya, Turkey (Türkiye), 06500
Actively Recruiting
29
Great Ormond Street Hospital for Children
London, London, United Kingdom, WC1N 3JH
Actively Recruiting
30
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
Actively Recruiting
31
Royal Free Hospital
London, United Kingdom, NW3 2QG
Actively Recruiting
32
Salford Royal Hospital
Salford, United Kingdom, M6 8HD
Actively Recruiting
Research Team
C
Clinical Trials at Denali Therapeutics
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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