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Enzyme-replacement Therapy

Tividenofusp Alfa for Hunter Syndrome (COMPASS Trial)

Phase 2 & 3
Recruiting
Research Sponsored by Denali Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II)
Participants aged ≥2 to <6 years (Cohort A) or ≥6 to <17 years (Cohort B)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 96 weeks
Awards & highlights

COMPASS Trial Summary

This trial is testing an investigational enzyme-replacement therapy for people with mucopolysaccharidosis type II. The trial has two parts: a double-blind part, where participants are randomly assigned to receive either the investigational therapy or a standard therapy, and an open-label part, where participants may receive either the investigational therapy or a standard therapy.

Who is the study for?
This trial is for kids with Hunter Syndrome (MPS II), aged 2-6 or 6-17, depending on the group. They must have been treated with idursulfase for at least 4 months. Kids can't join if they've had gene or stem cell therapy for MPS, are unable to undergo lumbar punctures/MRIs, received CNS-targeted ERT recently, or participated in other drug trials within the last 60 days.Check my eligibility
What is being tested?
The study compares Tividenofusp Alfa (DNL310), a new treatment that reaches the brain better than current therapies, against Idursulfase, which is standard care. It's randomized and double-blind meaning neither participants nor researchers know who gets which treatment until after results are collected.See study design
What are the potential side effects?
Potential side effects aren't specified here but may include reactions similar to those seen with enzyme replacement therapies such as allergic reactions, pain at injection site, headache, fever and chills.

COMPASS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with MPS II.
Select...
I am between 2 and 17 years old.

COMPASS Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~96 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 96 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from baseline in the Vineland Adaptive Behavior Scale, Third Edition (Vineland-3)(Cohort A only)
Percent change from baseline in cerebrospinal fluid (CSF) heparan sulfate (HS) concentration (Cohort A only)
Secondary outcome measures
Change from baseline in distance walked in the Six-Minute Walk Test (6MWT; Cohort B only)
Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III)(Cohort A only)
Improvement in Parent/Caregiver Global Impression of Change (CaGI-C) Overall MPS II (Cohorts A and B)
+3 more

COMPASS Trial Design

3Treatment groups
Experimental Treatment
Group I: Open-label Treatment PhaseExperimental Treatment2 Interventions
Participants who meet pre-specified criteria may receive DNL310 or idursulfase
Group II: Cohort B: Participants with nnMPS IIExperimental Treatment2 Interventions
Group III: Cohort A: Participants with nMPS IIExperimental Treatment2 Interventions

Find a Location

Who is running the clinical trial?

Denali Therapeutics Inc.Lead Sponsor
22 Previous Clinical Trials
1,815 Total Patients Enrolled
Jose Alcantara Rodriguez, PharmDStudy DirectorDenali Therapeutics Inc.
1 Previous Clinical Trials
99 Total Patients Enrolled
Lubica Trokan, MD, MPHStudy DirectorDenali Therapeutics Inc.

Media Library

DNL310 (Enzyme-replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05371613 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

If an elderly patient is not yet 85 years old, can they still join the trial?

"Children as young as 2 and no older than 16 years old may participate in this clinical trial, based on the provided age requirements."

Answered by AI

Are there specific requirements to participate in this research?

"Up to 54 children and adolescents with mucopolysaccharidosis ii will be accepted into this clinical trial. To be eligible, candidates must meet the following requirements: 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."

Answered by AI

Are people with the required qualifications able to enroll in this research project at this time?

"Yes, the trial is still open and actively recruiting patients. The original posting was on July 21st, 2022 and the most recent edit was on October 10th, 2022."

Answered by AI

How many people are being allowed to participate in this trial?

"In order for this study to commence, 54 patients that meet the pre-specified inclusion criteria must enroll. Some of the hospitals where patients can participate in this study include UCSF Benioff Children's Hospital Oakland located in Oakland, California and Cincinnati Children's Hospital Medical Center situated in Cincinnati, Ohio."

Answered by AI

Who else is applying?

What state do they live in?
North Carolina
California
British Columbia
What site did they apply to?
UCSF Benioff Children's Hospital Oakland
What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
2
Recent research and studies
clinicaltrials.govStudy
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)
2022. "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)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05371613.
~24 spots leftby Dec 2025
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