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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05371613




Registration number
NCT05371613
Ethics application status
Date submitted
9/05/2022
Date registered
12/05/2022

Titles & IDs
Public title
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)
Scientific 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
Secondary ID [1] 0 0
DNLI-E-0007
Universal Trial Number (UTN)
Trial acronym
COMPASS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis II 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - tividenofusp alfa
Treatment: Drugs - idursulfase

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


Treatment: Drugs: tividenofusp alfa
Intravenous repeating dose

Treatment: Drugs: idursulfase
Intravenous repeating dose
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A)
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A)
Timepoint [2] 0 0
96 weeks
Secondary outcome [1] 0 0
Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only)
Timepoint [1] 0 0
96 weeks
Secondary outcome [2] 0 0
Change from baseline in the Vineland-3 Adaptive Behavior Composite (ABC; Cohort A only)
Timepoint [2] 0 0
96 weeks
Secondary outcome [3] 0 0
Change from baseline in serum neurofilament light chain (NfL) (Cohort A only)
Timepoint [3] 0 0
96 weeks
Secondary outcome [4] 0 0
Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only)
Timepoint [4] 0 0
48 weeks
Secondary outcome [5] 0 0
Percent change from baseline in the sum of urine HS and dermatan sulfate (DS) concentrations (Cohorts A and B)
Timepoint [5] 0 0
up to 48 weeks
Secondary outcome [6] 0 0
Liver volume within the normal range (normal vs abnormal) as measured by magnetic resonance imaging (MRI) (Cohorts A and B)
Timepoint [6] 0 0
48 weeks
Secondary outcome [7] 0 0
Spleen volume within the normal range (normal vs abnormal) as measured by MRI (Cohorts A and B)
Timepoint [7] 0 0
48 weeks
Secondary outcome [8] 0 0
Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B)
Timepoint [8] 0 0
48 weeks

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
2 Years
Maximum age
26 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/07/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
54
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment postcode(s) [1] 0 0
- North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
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United States of America
State/province [2] 0 0
Illinois
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United States of America
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New Jersey
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United States of America
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North Carolina
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United States of America
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Ohio
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United States of America
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Pennsylvania
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United States of America
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Texas
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United States of America
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Utah
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Argentina
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Buenos Aires
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Belgium
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Brussels
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Belgium
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Antwerpen
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Brazil
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Porto Alegre
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Brazil
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Rio De Janeiro
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Canada
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Alberta
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Canada
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Ontario
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Canada
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Quebec
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Colombia
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Bogotá
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Czechia
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Praha
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France
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Lille
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France
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Marseille
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Germany
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Giessen
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Germany
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Hamburg
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Germany
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Hochheim
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Italy
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Napoli
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Italy
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Udine
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Netherlands
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Rotterdam
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Spain
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Barcelona
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Spain
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Madrid
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Sweden
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Göteborg
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Turkey
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Adana
Country [31] 0 0
Turkey
State/province [31] 0 0
Çankaya
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Birmingham
Country [33] 0 0
United Kingdom
State/province [33] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Denali Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Lubica Trokan, MD, MPH
Address 0 0
Denali Therapeutics Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clinical Trials at Denali Therapeutics
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
clinical-trials@dnli.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05371613