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)

Description

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

Mucopolysaccharidosis II

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Study Overview

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Study Details

Study overview

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.

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

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)

Condition
Mucopolysaccharidosis II
Intervention / Treatment

-

Contacts and Locations

Oakland

UCSF Benioff Children's Hospital Oakland, Oakland, California, United States, 94609

Chicago

Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Hackensack

Hackensack University Medical Center, Hackensack, New Jersey, United States, 07601

Chapel Hill

UNC Children's Research Institute, Chapel Hill, North Carolina, United States, 27514

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Houston

The University of Texas Medical School at Houston, Houston, Texas, United States, 77030

Salt Lake City

University of Utah, PPDS, Salt Lake City, Utah, United States, 84132

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

2 Years to 26 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Denali Therapeutics Inc.,

Jose Alcantara Rodriguez, PharmD, STUDY_DIRECTOR, Denali Therapeutics Inc.

Study Record Dates

2025-12