NCT06181136

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

A Phase 1/2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Denali Therapeutics Inc. · Industry
• Sex: All
• Age: 0 Years – 18 Years
• Healthy volunteers: Not accepted

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

• Mucopolysaccharidosis Type IIIA

Interventions

Type	Name	Description
DRUG	DNL126	intravenous repeating dose

Timeline

• Start date: 2023-12-07
• Primary completion: 2028-08-01
• Completion: 2028-08-01
• First posted: 2023-12-26
• Last updated: 2025-12-05

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study