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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: Active Phase 1 study of Mucopolysaccharidosis Type IIIA, sponsored by Denali Therapeutics.
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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
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 |
What the registry record for NCT06181136 still lists
NCT06181136 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (67% lower).
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 a single indexed study location in 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
How this trial's enrollment target compares
Where NCT06181136's enrollment target sits among peer trials
20 1629th of 2000 higher than 285 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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