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)

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

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

Quick Facts

Study Start:2022-07-21
Study Completion:2025-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05371613

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.

Ages Eligible for Study:2 Years to 26 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<26 years (Cohort B)
  2. * Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
  3. * 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
  1. * Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay
  2. * Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy
  3. * Received any CNS-targeted MPS ERT within 6 months prior to screening
  4. * Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI)
  5. * 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

Contacts and Locations

Study Contact

Clinical Trials at Denali Therapeutics
CONTACT
clinical-trials@dnli.com

Principal Investigator

Jose Alcantara Rodriguez, PharmD
STUDY_DIRECTOR
Denali Therapeutics Inc.

Study Locations (Sites)

UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
United States
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601
United States
UNC Children's Research Institute
Chapel Hill, North Carolina, 27514
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
The University of Texas Medical School at Houston
Houston, Texas, 77030
United States
University of Utah, PPDS
Salt Lake City, Utah, 84132
United States

Collaborators and Investigators

Sponsor: Denali Therapeutics Inc.

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-07-21
Study Completion Date2025-12

Study Record Updates

Study Start Date2022-07-21
Study Completion Date2025-12

Terms related to this study

Keywords Provided by Researchers

  • Hunter Syndrome
  • MPS II
  • nMPS II
  • nnMPS II

Additional Relevant MeSH Terms

  • Mucopolysaccharidosis II