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Not recruiting
Phase 2
Phase 3

Tividenofusp Alfa vs. Idursulfase

Sponsor:

Denali Therapeutics Inc.

Code:

NCT05371613

Conditions

Mucopolysaccharidosis II

Eligibility Criteria

Sex: All

Age: 2 - 25

Healthy Volunteers: Not accepted

Interventions

tividenofusp alfa

idursulfase

Study Details

Brief summary:

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

Study ID

NCT05371613

Start date

Jul 21, 2022

Status verified date

Sep, 2026

Completion date

Dec, 2027

Anticipated

Primary completion date

Dec, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 25

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

65 participants

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort A: Participants with nMPS II

experimental: Cohort B: Participants with nnMPS II

experimental: Open-label Treatment Phase

Participants who meet pre-specified criteria may receive DNL310 or idursulfase

Interventions

tividenofusp alfa

Intravenous repeating dose

idursulfase

Intravenous repeating dose

Primary outcome measure

  • Ratio to baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A) [ Time Frame: 24 weeks ]
  • Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A) [ Time Frame: 96 weeks ]

Central Contacts and Locations

More Information

Sponsor

Denali Therapeutics Inc.

Last update posted

Sep 9, 2026

Last verified

Sep, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-11-10. This information was provided to ClinicalTrials.gov by Denali Therapeutics Inc. on 2026-09-09. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.