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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 1, 2024
Sponsor:
Information provided by (Responsible Party):
Denali Therapeutics Inc.

Study Description
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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.


Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis II Drug: tividenofusp alfa Drug: idursulfase Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : July 21, 2022
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Idursulfase

Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A only) [ Time Frame: 24 weeks ]
  2. Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A only) [ Time Frame: 96 weeks ]

Secondary Outcome Measures :
  1. Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only) [ Time Frame: 96 weeks ]
  2. Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only) [ Time Frame: 48 weeks ]
  3. 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 ]
  4. Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) (Cohorts A and B) [ Time Frame: 48 weeks ]
  5. Spleen volume within the normal range (normal vs abnormal) as measured by MRI (Cohorts A and B) [ Time Frame: 48 weeks ]
  6. Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B) [ Time Frame: 48 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05371613


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

Locations
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Sponsors and Collaborators
Denali Therapeutics Inc.
Investigators
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Study Director: Lubica Trokan, MD, MPH Denali Therapeutics Inc.
More Information
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Additional Information:

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Responsible Party: Denali Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT05371613    
Other Study ID Numbers: DNLI-E-0007
First Posted: May 12, 2022    Key Record Dates
Last Update Posted: May 1, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Denali Therapeutics Inc.:
Hunter Syndrome
MPS II
nMPS II
nnMPS II
Additional relevant MeSH terms:
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Mucopolysaccharidosis II
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
 Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System