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)
NCT ID: NCT05371613
Last Updated: 2025-08-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
63 participants
INTERVENTIONAL
2022-07-21
2027-12-31
Brief Summary
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Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Cohort A: Participants with nMPS II
tividenofusp alfa
Intravenous repeating dose
idursulfase
Intravenous repeating dose
Cohort B: Participants with nnMPS II
tividenofusp alfa
Intravenous repeating dose
idursulfase
Intravenous repeating dose
Open-label Treatment Phase
Participants who meet pre-specified criteria may receive DNL310 or idursulfase
tividenofusp alfa
Intravenous repeating dose
idursulfase
Intravenous repeating dose
Interventions
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tividenofusp alfa
Intravenous repeating dose
idursulfase
Intravenous repeating dose
Eligibility Criteria
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Inclusion Criteria
* Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
* Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening
Exclusion Criteria
* Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy
* Received any CNS-targeted MPS ERT within 6 months prior to screening
* Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI)
* Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study
2 Years
25 Years
ALL
No
Sponsors
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Denali Therapeutics Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Jose Alcantara Rodriguez, PharmD
Role: STUDY_DIRECTOR
Denali Therapeutics Inc.
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Role: backup
Related Links
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Denali Therapeutics
Other Identifiers
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2024-510990-21-00
Identifier Type: CTIS
Identifier Source: secondary_id
2021-005200-35
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
DNLI-E-0007
Identifier Type: -
Identifier Source: org_study_id
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