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Trial record 1 of 1 for:    NCT05371613
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A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

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ClinicalTrials.gov Identifier: NCT05371613
Recruitment Status : Recruiting
First Posted : May 12, 2022
Last Update Posted : May 16, 2024
Sponsor:
Information provided by (Responsible Party):
Denali Therapeutics Inc.

Tracking Information
First Submitted Date  ICMJE May 9, 2022
First Posted Date  ICMJE May 12, 2022
Last Update Posted Date May 16, 2024
Actual Study Start Date  ICMJE July 21, 2022
Estimated Primary Completion Date December 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 9, 2022)
  • Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A only) [ Time Frame: 24 weeks ]
  • Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A only) [ Time Frame: 96 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 9, 2022)
  • Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only) [ Time Frame: 96 weeks ]
  • Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only) [ Time Frame: 48 weeks ]
  • Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations (Cohorts A and B) [ Time Frame: up to 48 weeks ]
  • Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) (Cohorts A and B) [ Time Frame: 48 weeks ]
  • Spleen volume within the normal range (normal vs abnormal) as measured by MRI (Cohorts A and B) [ Time Frame: 48 weeks ]
  • Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B) [ Time Frame: 48 weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Official Title  ICMJE A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Mucopolysaccharidosis II
Intervention  ICMJE
  • Drug: tividenofusp alfa
    Intravenous repeating dose
  • Drug: idursulfase
    Intravenous repeating dose
Study Arms  ICMJE
  • Experimental: Cohort A: Participants with nMPS II
    Interventions:
    • Drug: tividenofusp alfa
    • Drug: idursulfase
  • Experimental: Cohort B: Participants with nnMPS II
    Interventions:
    • Drug: tividenofusp alfa
    • Drug: idursulfase
  • Experimental: Open-label Treatment Phase
    Participants who meet pre-specified criteria may receive DNL310 or idursulfase
    Interventions:
    • Drug: tividenofusp alfa
    • Drug: idursulfase
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 9, 2022)		 54
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2025
Estimated Primary Completion Date December 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Participants aged ≥2 to <6 years (Cohort A) or ≥6 to <17 years (Cohort B)
  • Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
  • Be on maintenance enzyme replacement therapy (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 imagings (MRIs)
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 16 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Trials at Denali Therapeutics clinical-trials@dnli.com
Listed Location Countries  ICMJE Argentina,   Belgium,   Brazil,   Canada,   Colombia,   Czechia,   France,   Germany,   Italy,   Netherlands,   Spain,   Sweden,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05371613
Other Study ID Numbers  ICMJE DNLI-E-0007
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Denali Therapeutics Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Denali Therapeutics Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Lubica Trokan, MD, MPH Denali Therapeutics Inc.
PRS Account Denali Therapeutics Inc.
Verification Date May 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP