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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)
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)

NCT05371613

Active Not RecruitingPhase 2, Phase 3

Sponsor: Denali Therapeutics Inc.

Conditions: Mucopolysaccharidosis II

Interventions: tividenofusp alfa, idursulfase

Countries: United States, Argentina, Belgium, Brazil, Canada, Czechia, France, Germany

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.

Eligibility overview

Sex: ALL

Age: 2 Years to 25 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Key Inclusion Criteria:

* Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<26 years (Cohort B)
* 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

Key Exclusion Criteria:

* Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
* 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
Locations (30)
  • Oakland, California, United States
  • Chicago, Illinois, United States
  • Hackensack, New Jersey, United States
  • Chapel Hill, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Philadelphia, Pennsylvania, United States
  • Houston, Texas, United States
  • Salt Lake City, Utah, United States
  • Buenos Aires, Argentina
  • Antwerp, Antwerpen, Belgium
  • Jette, Brussels Capital, Belgium
  • Porto Alegre, Brazil
  • Edmonton, Alberta, Canada
  • Toronto, Ontario, Canada
  • Montreal, Quebec, Canada
  • Prague, Czechia
  • Lille, France
  • Höchheim, Hochheim, Germany
  • Cottbus, Germany
  • Udine, Italy
  • Rotterdam, Rotterdam, Netherlands
  • Barcelona, Barcelona, Spain
  • Madrid, Madrid, Spain
  • Gothenburg, Sweden
  • Adana, Turkey (Türkiye)
  • Çankaya, Turkey (Türkiye)
  • London, London, United Kingdom
  • Birmingham, United Kingdom
  • London, United Kingdom
  • Salford, United Kingdom