DNL310 for Mucopolysaccharidosis II

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
UCSF Benioff Children's Hospital Oakland, Oakland, CA
Mucopolysaccharidosis II+1 More
DNL310 - Drug
Eligibility
< 18
All Sexes
What conditions do you have?
Select

Study 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 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.

Eligible Conditions

  • Mucopolysaccharidosis II

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Mucopolysaccharidosis II

Study Objectives

2 Primary · 6 Secondary · Reporting Duration: 96 weeks

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

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Mucopolysaccharidosis II

Trial Design

5 Treatment Groups

Cohort B
1 of 5
Cohort A
1 of 5
Open-label Treatment Phase
1 of 5
Cohort A: Participants with nMPS II
1 of 5
Cohort B: Participants with nnMPS II
1 of 5
Experimental Treatment

54 Total Participants · 5 Treatment Groups

Primary Treatment: DNL310 · No Placebo Group · Phase 2 & 3

Cohort BExperimental Group · 2 Interventions: Idursulfase, DNL310 · Intervention Types: Drug, Drug
Cohort AExperimental Group · 2 Interventions: Idursulfase, DNL310 · Intervention Types: Drug, Drug
Open-label Treatment PhaseExperimental Group · 2 Interventions: Idursulfase, DNL310 · Intervention Types: Drug, Drug
Cohort A: Participants with nMPS IIExperimental Group · 2 Interventions: Idursulfase, DNL310 · Intervention Types: Drug, Drug
Cohort B: Participants with nnMPS IIExperimental Group · 2 Interventions: Idursulfase, DNL310 · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Idursulfase
2012
Completed Phase 4
~160

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 96 weeks
Closest Location: UCSF Benioff Children's Hospital Oakland · Oakland, CA
Photo of california 1Photo of california 2Photo of california 3
2021First Recorded Clinical Trial
4 TrialsResearching Mucopolysaccharidosis II
38 CompletedClinical Trials

Who is running the clinical trial?

Denali Therapeutics Inc.Lead Sponsor
18 Previous Clinical Trials
1,904 Total Patients Enrolled
2 Trials studying Mucopolysaccharidosis II
82 Patients Enrolled for Mucopolysaccharidosis II
Jose Alcantara Rodriguez, PharmDStudy DirectorDenali Therapeutics Inc.

Eligibility Criteria

Age < 18 · All Participants · 4 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are aged 2 to <17 years or ≥17 to <6 years.
You have a confirmed diagnosis of MPS II.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.