Clinical Trial

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

Active, Not Recruiting Phase 1/2
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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.
Protocol Amendment History 13 amendments
This ClinicalTrials.gov record has been amended 13 times since 2023-12-22; most recent amendment 2026-07-10.
Status change: Recruiting → Active, Not Recruiting 2025-09-17
Status change: Not Yet Recruiting → Recruiting 2023-12-24
Trial Details
NCT Number NCT06181136
Lead Sponsor Denali Therapeutics Inc.
Conditions Mucopolysaccharidosis Type IIIA
Enrollment 20 participants
Start Date 2023-12-07
Primary Completion 2028-08 (estimated)
Study Completion 2028-08 (estimated)
Updated on ClinicalTrials.gov 2026-07-13