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Small Molecule

Cohort A1 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

Study 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.Check my eligibility

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.See study design

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 97 weeks

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence and severity of infusion-related reactions (IRRs)

Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)

Number of participants with clinically significant treatment emergent abnormalities in 12-lead ECG results.

+4 more

Secondary outcome measures

Change from baseline in liver volume

Change from baseline in spleen volume

DNL126 serum PK parameters

+12 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort A2Experimental Treatment1 Intervention

Participants with MPS IIIA

Group II: Cohort A1Experimental Treatment1 Intervention

Participants with MPS IIIA

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Denali Therapeutics Inc.Lead Sponsor

22 Previous Clinical Trials

1,859 Total Patients Enrolled

Steven Chessler, MD, PhDStudy DirectorDenali Therapeutics

Frequently Asked Questions

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

Are there any available slots for patients to participate in this research study?

"Indeed, according to the information available on clinicaltrials.gov, this research study is presently seeking eligible participants. The trial was initially posted on December 7th, 2023 and has been recently updated on December 24th, 2023."

Answered by AI

Is the enrollment for this clinical study open to individuals who are below 18 years of age?

"Prospective participants who are aged between 6 months and 18 years old meet the inclusion criteria for this trial. It is worth noting that there are currently 30 clinical trials available specifically for individuals under the age of 18, while there are 15 trials targeting those over the age of 65."

Answered by AI

What is the current number of individuals receiving medical care within this clinical trial?

"Indeed, the details on clinicaltrials.gov affirm that this ongoing clinical trial is actively seeking eligible participants. The initial posting of the trial took place on December 7th, 2023, and it was last updated on December 24th, 2023. The study aims to enroll a total of 16 patients at a single designated location."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

2023. "Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06181136.

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