Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT06181136 · ClinicalTrials.gov registry record · Phase 1
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
A Phase 1 study of Mucopolysaccharidosis Type IIIA, sponsored by Denali Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 20
- Enrollment target
- 4
- Study locations
NCT06181136 is a Phase 1 study of Mucopolysaccharidosis Type IIIA that is active but no longer recruiting, run by Denali Therapeutics. The registered enrollment target is 20 participants. The trial reports 4 study locations across 4 states.
The verdict
NCT06181136, a Phase 1 study of Mucopolysaccharidosis Type IIIA, is active but no longer recruiting, sponsored by Denali Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 20 participants
- Enrollment target
- 4
- Study locations
Study Summary
This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). The core study period is 25 weeks (approximately 6 months); followed by an open-label extension (OLE), which extends through Week 97 (approximately 18 months); and a long-term extension (LTE), which extends through Week 193 (Year 4). Participants with MPS IIIA will be enrolled in two planned cohorts, and additional participants with MPS IIIA may be enrolled in three optional cohorts.
Conditions Studied
Interventions
- DRUG DNL126
Study Locations (4)
California
- UCSF Benioff Children's Hospital Oakland - Oakland
Iowa
- University of Iowa Stead Family Children's Hospital - Iowa City
North Carolina
- University of North Carolina at Chapel Hill - Chapel Hill
Texas
- Baylor College of Medicine and Texas Children's Hospital - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2023-12-07 |
| Est. Completion | 2028-08 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06181136
The ClinicalTrials.gov registry entry for NCT06181136 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Denali Therapeutics, which has 12 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Mucopolysaccharidosis Type IIIA appearing as the primary indexed condition, and to 1 intervention - of which DNL126 is the first listed.
NCT06181136 reports 4 study locations spanning 4 distinct geographic areas - top geographies include California, Iowa, North Carolina.
Frequently Asked Questions
What is clinical trial NCT06181136 about?
NCT06181136 is a clinical study titled "Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)". This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). The core study period is 25 weeks (approximately 6 months); followed by an o...
What is the current status of trial NCT06181136?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 20 participants. The study started on 2023-12-07. Estimated completion is 2028-08.
What conditions does trial NCT06181136 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis Type IIIA.
What interventions are being tested in trial NCT06181136?
The interventions under investigation include: DNL126 (DRUG).
Who is sponsoring clinical trial NCT06181136?
This trial is sponsored by Denali Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06181136 being conducted?
This trial has 4 study locations across California, Iowa, North Carolina, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.