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Purpose

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.

Condition

Eligibility

Eligible Ages
Between 2 Years and 26 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

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

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Cohort A: Participants with nMPS II
  • Drug: tividenofusp alfa
    Intravenous repeating dose
  • Drug: idursulfase
    Intravenous repeating dose
Experimental
Cohort B: Participants with nnMPS II
  • Drug: tividenofusp alfa
    Intravenous repeating dose
  • Drug: idursulfase
    Intravenous repeating dose
Experimental
Open-label Treatment Phase 		Participants who meet pre-specified criteria may receive DNL310 or idursulfase
  • Drug: tividenofusp alfa
    Intravenous repeating dose
  • Drug: idursulfase
    Intravenous repeating dose

Recruiting Locations

The University of Texas Medical School at Houston
Houston, Texas 77030
Contact:
Marilyn Garcia
marilyn.garcia@uth.tmc.edu

More Details

Status
Recruiting
Sponsor
Denali Therapeutics Inc.

Study Contact

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.