Your session is about to expire
← Back to Search
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 belowSelect...
I have been diagnosed with MPS II.
Select...
I am between 2 and 17 years old.
COMPASS Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 96 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~96 weeks
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 moreCOMPASS 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
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Hunter Syndrome treatments primarily involve enzyme replacement therapies (ERTs) like idursulfase, which supplement the deficient enzyme to reduce glycosaminoglycan (GAG) accumulation in tissues. Tividenofusp alfa, a CNS-penetrant ERT, is under investigation for its ability to cross the blood-brain barrier and address neurological symptoms, a critical advancement since traditional ERTs do not effectively treat CNS manifestations.
This matters greatly for patients with neuronopathic Hunter Syndrome, as it offers hope for mitigating cognitive decline alongside peripheral symptom management.
Further experience with the use of 6-thioguanine in patients with Crohn's disease.A PRISMA-compliant systematic review and network meta-analysis on the efficacy between different regimens based on Tripterygium wilfordii Hook F in patients with primary nephrotic syndrome.Enzyme replacement therapy for mucopolysaccharidosis VI--experience in Taiwan.
Further experience with the use of 6-thioguanine in patients with Crohn's disease.A PRISMA-compliant systematic review and network meta-analysis on the efficacy between different regimens based on Tripterygium wilfordii Hook F in patients with primary nephrotic syndrome.Enzyme replacement therapy for mucopolysaccharidosis VI--experience in Taiwan.
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
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Share this study with friends
Copy Link
Messenger