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Small Molecule

DNL126 for Sanfilippo Syndrome

Phase 1 & 2
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 IIIA
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 97 weeks
Awards & highlights
No Placebo-Only Group

Summary

This trial is researching a drug called DNL126 for people with Sanfilippo syndrome Type A. It will study the safety, how well the drug is tolerated, how the body processes the drug,

Who is the study for?
This trial is for children with Sanfilippo Syndrome Type A (MPS IIIA), specifically those who have a confirmed diagnosis. If they have an older sibling with the same severe condition, they may also qualify to participate in this study.
What is being tested?
The trial is testing DNL126, which is being studied for safety, how well it's tolerated by patients, its movement and actions within the body (PK/PD), and potential benefits on symptoms of MPS IIIA over a core period of 6 months followed by an extension of approximately 18 months.
What are the potential side effects?
As this is a Phase 1/2 study primarily focused on safety and tolerability, specific side effects are not listed but will be closely monitored throughout the trial duration.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with MPS IIIA.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 97 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 97 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
DNL126 serum PK parameters
Maximum concentration (Cmax)
apparent terminal elimination half-life (t½)
+3 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

5Treatment groups
Experimental Treatment
Group I: Cohort B2Experimental Treatment1 Intervention
Participants with MPS IIIA
Group II: Cohort B1Experimental Treatment1 Intervention
Participants with MPS IIIA
Group III: Cohort A3Experimental Treatment1 Intervention
Participants with MPS IIIA
Group IV: Cohort A2Experimental Treatment1 Intervention
Participants with MPS IIIA
Group V: Cohort A1Experimental Treatment1 Intervention
Participants with MPS IIIA

Find a Location

Who is running the clinical trial?

Denali Therapeutics Inc.Lead Sponsor
23 Previous Clinical Trials
1,910 Total Patients Enrolled
Ana-Claire Meyer, MDStudy DirectorDenali Therapeutics
2 Previous Clinical Trials
54 Total Patients Enrolled
Steven Chessler, MD, PhDStudy DirectorDenali Therapeutics
~13 spots leftby Aug 2028
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